Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Feb. 24, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | SRPT | ||
Entity Registrant Name | Sarepta Therapeutics, Inc. | ||
Entity Central Index Key | 0000873303 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | false | ||
Entity Emerging Growth Company | false | ||
Entity Shell Company | false | ||
ICFR Auditor Attestation Flag | false | ||
Entity File Number | 001-14895 | ||
Entity Tax Identification Number | 93-0797222 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Address, Address Line One | 215 First Street | ||
Entity Address, Address Line Two | Suite 415 | ||
Entity Address, City or Town | Cambridge | ||
Entity Address, State or Province | MA | ||
Entity Address, Postal Zip Code | 02142 | ||
City Area Code | 617 | ||
Local Phone Number | 274-4000 | ||
Entity Interactive Data Current | Yes | ||
Title of 12(b) Security | Common Stock, $0.0001 par value | ||
Security Exchange Name | NASDAQ | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Common Stock, Shares Outstanding | 87,149,589 | ||
Entity Public Float | $ 6,206,016,151 | ||
Documents Incorporated by Reference | The registrant has incorporated by reference into Part II and Part III of this Annual Report on Form 10-K portions of its definitive Proxy Statement for the 2022 Annual Meeting of Stockholders to be filed no later than 120 days after the end of the fiscal year covered by this Annual Report on Form 10-K. | ||
Auditor Name | KPMG LLP | ||
Auditor Location | Boston, Massachusetts | ||
Auditor Firm ID | 185 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 2,115,869 | $ 1,502,648 |
Short-term investments | 435,923 | |
Accounts receivable | 152,990 | 101,340 |
Inventory | 186,212 | 231,961 |
Other current assets | 149,028 | 213,324 |
Total current assets | 2,604,099 | 2,485,196 |
Property and equipment, net | 191,156 | 190,430 |
Intangible assets, net | 14,239 | 13,628 |
Right of use assets | 45,531 | 91,761 |
Other non-current assets | 292,949 | 203,703 |
Total assets | 3,147,974 | 2,984,718 |
Current liabilities: | ||
Accounts payable | 76,741 | 111,090 |
Accrued expenses | 271,697 | 193,553 |
Deferred revenue, current portion | 89,244 | 89,244 |
Other current liabilities | 15,051 | 22,139 |
Total current liabilities | 452,733 | 416,026 |
Long-term debt | 1,096,876 | 992,493 |
Lease liabilities, net of current portion | 41,512 | 80,367 |
Deferred revenue, net of current portion | 574,244 | |
Contingent consideration | 43,600 | 50,800 |
Other non-current liabilities | 11,000 | 19,785 |
Total liabilities | 2,219,965 | 2,222,959 |
Commitments and contingencies (Note 21) | ||
Stockholders’ equity: | ||
Preferred stock, $.0001 par value, 3,333,333 shares authorized; none issued and outstanding | ||
Common stock, $.0001 par value, 198,000,000 shares authorized; 87,126,974 and 79,374,247 issued and outstanding at December 31, 2021 and 2020, respectively | 9 | 8 |
Additional paid-in capital | 4,134,768 | 3,609,877 |
Accumulated other comprehensive (loss) income, net of tax | 20 | 3 |
Accumulated deficit | (3,206,748) | (2,848,129) |
Total stockholders’ equity | 928,009 | 761,759 |
Total liabilities and stockholders’ equity | $ 3,147,974 | $ 2,984,718 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 3,333,333 | 3,333,333 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 198,000,000 | 198,000,000 |
Common stock, issued | 87,126,974 | 79,374,247 |
Common stock, outstanding | 87,126,974 | 79,374,247 |
Consolidated Statements of Oper
Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |||
Revenues: | |||||
Products, net | $ 612,401 | $ 455,865 | $ 380,833 | ||
Type of Revenue [Extensible List] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | ||
Collaboration and other | $ 89,486 | $ 84,234 | $ 0 | ||
Total revenues | 701,887 | 540,099 | 380,833 | ||
Cost and expenses: | |||||
Cost of sales (excluding amortization of in-licensed rights) | 97,049 | 63,382 | 56,586 | ||
Research and development | 771,182 | 722,343 | 560,909 | ||
Selling, general and administrative | 282,660 | 317,875 | 284,812 | ||
Settlement and license charges | 10,000 | 0 | 10,000 | ||
Amortization of in-licensed rights | 706 | 662 | 849 | ||
Acquired in-process research and development | 0 | 0 | 173,240 | ||
Total cost and expenses | 1,161,597 | 1,104,262 | 1,086,396 | ||
Operating loss | (459,710) | (564,163) | (705,563) | ||
Other income (loss): | |||||
Gain from sale of Priority Review Voucher | 102,000 | 108,069 | |||
Gain (loss) on contingent consideration, net | 7,200 | [1] | (45,000) | [1] | |
Other expense, net | (68,438) | (51,971) | (8,317) | ||
Total other income (loss) | 40,762 | 11,098 | (8,317) | ||
Loss before income tax (benefit) expense | (418,948) | (553,065) | (713,880) | ||
Income tax (benefit) expense | (168) | 1,063 | 1,195 | ||
Net loss | (418,780) | (554,128) | (715,075) | ||
Other comprehensive loss: | |||||
Unrealized (losses) gains on investments, net of tax | (23) | (47) | 149 | ||
Total other comprehensive (loss) income | (23) | (47) | 149 | ||
Comprehensive loss | $ (418,803) | $ (554,175) | $ (714,926) | ||
Net loss per share — basic and diluted | $ (5.15) | $ (7.11) | $ (9.71) | ||
Weighted average number of shares of common stock used in computing basic and diluted net loss per share | 81,262 | 77,956 | 73,615 | ||
[1] | The gain (loss) on contingent consideration, net is related to the fair value adjustment of the regulatory-related contingent payments that are accounted for as derivatives. Please see Note 5. Fair Value Measurements for further details. |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | ASU 2020-06 | Common Stock [Member] | Additional Paid-In Capital [Member] | Additional Paid-In Capital [Member]ASU 2020-06 | Accumulated Other Comprehensive (Loss) Gain [Member] | Accumulated Deficit [Member] | Accumulated Deficit [Member]ASU 2020-06 |
BALANCE at Dec. 31, 2018 | $ 1,032,276 | $ 7 | $ 2,611,294 | $ (99) | $ (1,578,926) | |||
BALANCE (in shares) at Dec. 31, 2018 | 71,072,000 | |||||||
Exercise of options for common stock | 31,522 | 31,522 | ||||||
Exercise of options for common stock, shares | 1,125,000 | |||||||
Grant of restricted stock awards and vest of restricted stock units, net of cancellations, shares | 68,000 | |||||||
Shares withheld for taxes | (9,135) | (9,135) | ||||||
Shares withheld for taxes, shares | (78,000) | |||||||
Issuance of common stock for cash, net of offering costs | 365,354 | $ 1 | 365,353 | |||||
Issuance of common stock for cash, net of offering costs, shares | 2,604,000 | |||||||
Issuance of common stock for collaboration agreement | 29,415 | 29,415 | ||||||
Issuance of common stock for collaboration agreement, shares | 302,000 | |||||||
Issuance of common stock under employee stock purchase plan | 5,079 | 5,079 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 92,000 | |||||||
Stock-based compensation | 78,602 | 78,602 | ||||||
Unrealized gain (loss) from available-for-sale securities | 149 | 149 | ||||||
Net loss | (715,075) | (715,075) | ||||||
BALANCE at Dec. 31, 2019 | 818,187 | $ 8 | 3,112,130 | 50 | (2,294,001) | |||
BALANCE (in shares) at Dec. 31, 2019 | 75,185,000 | |||||||
Exercise of options for common stock | 76,492 | 76,492 | ||||||
Exercise of options for common stock, shares | 1,443,000 | |||||||
Vest of restricted stock units/awards, net of forfeitures | 159,000 | |||||||
Shares withheld for taxes | (6,333) | (6,333) | ||||||
Shares withheld for taxes, shares | (37,000) | |||||||
Issuance of common stock for cash, net of offering costs | 312,053 | 312,053 | ||||||
Issuance of common stock for cash, net of offering costs, shares | 2,522,000 | |||||||
Issuance of common stock under employee stock purchase plan | 7,465 | 7,465 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 102,000 | |||||||
Stock-based compensation | 108,070 | 108,070 | ||||||
Unrealized gain (loss) from available-for-sale securities | (47) | (47) | ||||||
Net loss | (554,128) | (554,128) | ||||||
BALANCE at Dec. 31, 2020 | 761,759 | $ (96,792) | $ 8 | 3,609,877 | $ (156,953) | 3 | (2,848,129) | $ 60,161 |
BALANCE (in shares) at Dec. 31, 2020 | 79,374,000 | |||||||
Exercise of options for common stock | $ 12,963 | 12,963 | ||||||
Exercise of options for common stock, shares | 283,622 | 283,000 | ||||||
Vest of restricted stock units/awards, net of forfeitures | 277,000 | |||||||
Shares withheld for taxes | $ (1,432) | (1,432) | ||||||
Shares withheld for taxes, shares | (18,000) | |||||||
Issuance of common stock for cash, net of offering costs | 548,532 | $ 1 | 548,531 | |||||
Issuance of common stock for cash, net of offering costs, shares | 7,099,000 | |||||||
Issuance of common stock under employee stock purchase plan | 7,839 | 7,839 | ||||||
Issuance of common stock under employee stock purchase plan, shares | 112,000 | |||||||
Stock-based compensation | 113,943 | 113,943 | ||||||
Unrealized gain (loss) from available-for-sale securities | (23) | (23) | ||||||
Net loss | (418,780) | (418,780) | ||||||
BALANCE at Dec. 31, 2021 | $ 928,009 | $ 9 | $ 4,134,768 | $ 20 | $ (3,206,748) | |||
BALANCE (in shares) at Dec. 31, 2021 | 87,127,000 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |||
Cash flows from operating activities: | |||||
Net loss | $ (418,780) | $ (554,128) | $ (715,075) | ||
Adjustments to reconcile net loss to cash flows in operating activities: | |||||
(Gain) loss on contingent consideration, net | (7,200) | [1] | 45,000 | [1] | |
Gain from sale of Priority Review Voucher, net of commission | (102,000) | (108,069) | |||
Depreciation and amortization | 38,017 | 26,911 | 24,500 | ||
Reduction in the carry amounts of the right of use assets | 11,325 | 12,828 | 6,047 | ||
Non-cash interest expense | 7,581 | 25,454 | 21,444 | ||
Stock-based compensation | 113,943 | 108,070 | 78,602 | ||
Impairment of equity investment | 4,488 | ||||
Acquired in-process research and development | 173,240 | ||||
Non-cash up-front payment to StrideBio | 29,415 | ||||
Other | 7,620 | (2,656) | (7,755) | ||
Changes in operating assets and liabilities, net: | |||||
Net increase in accounts receivable | (51,650) | (10,461) | (41,835) | ||
Net increase in inventory | (83,772) | (60,582) | (45,934) | ||
Net decrease (increase) in other assets | 103,203 | (166,328) | (102,091) | ||
Net (decrease) increase in deferred revenue | (89,244) | 749,429 | |||
Net increase in accounts payable, accrued expenses, lease liabilities and other liabilities | 23,297 | 41,998 | 122,979 | ||
Net cash (used in) provided by operating activities | (443,172) | 107,466 | (456,463) | ||
Cash flows from investing activities: | |||||
Purchase of property and equipment | (38,490) | (82,202) | (59,631) | ||
Proceeds from sale of Priority Review Voucher | 102,000 | 108,069 | |||
Purchase of available-for-sale securities | (29,988) | (1,333,568) | (1,193,632) | ||
Maturity and sales of available-for-sale securities | 466,000 | 1,189,480 | 1,715,626 | ||
Acquisition of Myonexus Therapeutics, Inc., net of cash acquired | (172,556) | ||||
Other | (4,109) | (3,500) | (3,082) | ||
Net cash provided by (used in) investing activities | 495,413 | (121,721) | 286,725 | ||
Cash flows from financing activities: | |||||
Proceeds from sales of common stock, net of offering costs | 548,532 | 365,354 | |||
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program | 20,802 | 83,957 | 36,601 | ||
Taxes paid related to net share settlement of equity awards | (7,765) | (4,798) | (4,337) | ||
Proceeds from issuance of common stock to Roche, net of offering costs | 312,053 | ||||
Proceeds from term loans | 291,150 | 245,625 | |||
Debt issuance costs | (39) | (689) | |||
Net cash provided by financing activities | 561,569 | 682,323 | 642,554 | ||
Increase in cash and cash equivalents | 613,810 | 668,068 | 472,816 | ||
Cash, cash equivalents and restricted cash: | |||||
Beginning of year | 1,511,713 | 843,645 | 370,829 | ||
End of year | 2,125,523 | 1,511,713 | 843,645 | ||
Reconciliation of cash, cash equivalents and restricted cash: | |||||
Cash and cash equivalents | 2,115,869 | 1,502,648 | 835,080 | ||
Restricted cash in other assets | $ 9,654 | $ 9,065 | $ 8,565 | ||
Restricted Cash, Noncurrent, Asset, Statement of Financial Position [Extensible List] | Other non-current assets | Other non-current assets | Other non-current assets | ||
Total cash, cash equivalents and restricted cash | $ 2,125,523 | $ 1,511,713 | $ 843,645 | ||
Supplemental disclosure of cash flow information: | |||||
Cash paid during the period for interest | 55,949 | 34,418 | 8,550 | ||
Cash paid during the period for income taxes | 583 | 2,510 | 933 | ||
Lease liabilities arising from obtaining right of use assets | 13,225 | 59,327 | |||
Lease liabilities terminated | 40,133 | ||||
Intangible assets and property and equipment included in accounts payable and accrued expenses | 4,162 | 5,151 | 1,309 | ||
Shares withheld for tax included in accrued expenses | 0 | 6,333 | 4,798 | ||
Accrued debt issuance costs | $ 0 | $ 11,000 | $ 5,000 | ||
[1] | The gain (loss) on contingent consideration, net is related to the fair value adjustment of the regulatory-related contingent payments that are accounted for as derivatives. Please see Note 5. Fair Value Measurements for further details. |
ORGANIZATION AND NATURE OF BUSI
ORGANIZATION AND NATURE OF BUSINESS | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
ORGANIZATION AND NATURE OF BUSINESS | 1. ORGANIZATION AND NATURE OF BUSINESS Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, highly-differentiated and innovative technologies, and through collaborations with its strategic partners, the Company is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne muscular dystrophy (“Duchenne”), Limb-girdle muscular dystrophies (“LGMDs”) and other neuromuscular and central nervous system (“CNS”) disorders. The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) on September 19, 2016, December 12, 2019 and February 25, 2021, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein. As of December 31, 2021, the Company had approximately $ 2,125.8 million of cash, cash equivalents and investments, consisting of $ 2,115.9 million of cash and cash equivalents and $ 9.9 million of restricted cash and investments. The Company believes that its balance of cash, cash equivalents and investments as of December 31, 2021 is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and additional government contracts and establish collaborations with or license its technology to other companies. |
SUMMARY OF SIGNIFICANT ACCOUNTI
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS | 2. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS Basis of Presentation The accompanying consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the U.S. (“U.S. GAAP”), reflect the accounts of Sarepta Therapeutics, Inc. and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. The Company’s CEO, as the chief operating decision-maker, manages and allocates resources to the operations of the Company on a total company basis. The Company’s research and development organization is responsible for the research and discovery of new product candidates and supports development and registration efforts for potential future products. The Company’s supply chain organization manages the development of the manufacturing processes, clinical trial supply and commercial product supply. The Company’s commercial organization is responsible for commercialization of EXONDYS 51, VYONDYS 53 and AMONDYS 45 in the U.S. and internationally. The Company is supported by other back-office general and administration functions. Consistent with this decision-making process, the Company’s CEO uses consolidated, single-segment financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Estimates and Uncertainties The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. Fair Value Measurements The Company has certain financial assets and liabilities that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements: • Level 1—quoted prices for identical instruments in active markets; • Level 2—quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and • Level 3—valuations derived from valuation techniques in which one or more significant value drivers are unobservable. The fair value of the majority of the Company’s financial assets is categorized as Level 1 within the fair value hierarchy. These assets include money market funds, and publicly traded debt and equity securities. For additional information related to fair value measurements, please read Note 5, Fair Value Measurements to the consolidated financial statements. Cash Equivalents Only investments that are highly liquid and readily convertible to cash and have original maturities of three months or less are considered cash equivalents. Investments Available-For-Sale Debt Securities Available-for-sale debt securities are recorded at fair value and unrealized gains and losses are included in accumulated other comprehensive income in stockholder’s equity. Interest income and realized gains and losses are reported in other expense, net, on a specific identification basis. Equity Investments The Company’s equity investments include its investments in a publicly traded biotechnology company and several privately held biotechnology companies and are included in other non-current assets in the Company’s consolidated balance sheets. The equity investment in the publicly traded biotechnology company has a readily determinable fair value and is carried at fair value. The equity investments in the privately held biotechnology companies do not have readily determinable fair values and are measured at cost less any impairment, plus or minus changes resulting from observable price changes for the identical or a similar investment of the same issuer. Any change in the valuation of equity investments is recorded as a gain or loss on the Company’s consolidated statements of operations and comprehensive loss. Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from Medicaid rebates, governmental chargebacks including Public Health Services (“PHS”) chargebacks, prompt pay discounts, co-pay assistance and distribution fees. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of the Company) for PHS chargebacks, prompt pay discounts and certain distribution fees, or a current liability (if a payment is required of the Company) for Medicaid rebates, co-pay assistance and certain distribution fees. The accounts receivable from product sales represents receivables due from the Company’s specialty distributor and specialty pharmacies in the U.S. as well as certain distributors in the European Union (“EU”) , Brazil, Israel and the Middle East. The Company has had no historical write-offs of its accounts receivable and its payment terms range from 60 to 91 days for sales within the U.S. and 45 and 150 days for the majority of product sales outside the U.S. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in the customers’ credit profiles or any specific issues. The Company provides reserves against trade receivables for expected credit losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2021 , the credit profiles for the Company’s customers are deemed to be in good standing and an allowance for credit losses is not considered necessary. Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers and cash. Three individual customers accounted for 48 %, 39 % and 10 % o f net product revenues for the year ended December 31, 2021 , 47 %, 39 % and 11 % for the year ended December 31, 2020 , and 43 %, 41 % and 13 % for the year ended December 31, 2019. Three individual customers accounted for 41 %, 41 % and 10 % of accounts receivable from product sales for the year ended December 31, 2021 and 45 %, 41 % and 9 % for the year ended December 31, 2020. As of December 31, 2021, the Company believes that such customers are of high credit quality. As of December 31, 2021, the Company’s cash was concentrated at three financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. Inventories Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. EXONDYS 51, VYONDYS 53 and AMONDYS 45 inventory used in clinical development programs is charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. The Company periodically analyzes its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. Additionally, though the Company’s products are subject to strict quality control and monitoring which the Company performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications. Expense incurred related to excess inventory, obsolete inventory, or inventories that do not meet the Company's quality specifications are recorded as a component of cost of sales in the Company's consolidated statements of operations and comprehensive loss. For products which are under development and have not yet been approved by regulatory authorities, purchased drug product is charged to research and development expense upon delivery. Delivery occurs when the inventory passes quality inspection and ownership transfers to the Company. Nonrefundable advance payments for research and development activities, including production of purchased drug product, are deferred and capitalized until the goods are delivered. If the Company does not expect the goods to be delivered or services to be rendered, the advanced payment capitalized will be charged to expense. Property and Equipment Property and equipment are initially recorded at cost, including the acquisition cost and all costs necessarily incurred to bring the asset to the location and working condition necessary for their intended use. The cost of normal, recurring or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. The cost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in future economic benefits. Interest costs incurred during the construction period of major capital projects are periodically reviewed, and if determined to be material, capitalized until the asset is ready for its intended use, at which point the interest costs are amortized as depreciation expense over the life of the underlying asset. The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab and manufacturing equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in progress Not depreciated until put into service Intangible assets The Company’s intangible assets consist of in-licensed rights, patent costs and software licenses, which are stated in the Company’s consolidated balance sheets, net of accumulated amortization and impairments, if applicable. The in-licensed rights primarily relate to agreements with BioMarin Pharmaceutical, Inc. (“BioMarin”) and the University of Western Australia (“UWA”). The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents because the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights. Patent costs consist primarily of external legal costs, filing fees incurred to file patent applications and renewal fees on proprietary technology developed or licensed by the Company. Patent costs associated with applying for a patent, being issued a patent and annual renewal fees are capitalized. Costs to defend a patent and costs to invalidate a competitor’s patent or patent application are expensed as incurred. Patent costs are amortized on a straight-line basis over the shorter of the estimated economic lives or the initial term of the patents, which is generally 20 years. Impairment of Long-Lived Assets Long-lived assets held and used by the Company, intangible assets with definite lives and right of use (“ROU”) assets are reviewed for impairment whenever events or circumstances indicate that the carrying amount of assets may not be recoverable. The Company evaluates recoverability of assets to be held and used by comparing the carrying amount of an asset to future net undiscounted cash flows to be generated by the asset. If the asset is considered to be impaired, the impairment to be recognized is measured as the amount by which the carrying amount of the assets exceeds the fair value of the assets. Such reviews assess the fair value of the assets based upon estimates of future cash flows that the assets are expected to generate. Convertible Debt As a result of adopting ASU 2020-06, “ Debt – Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging – Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity ”, the Company accounts for the liability and equity components of convertible debt instruments that can be settled in cash as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives under ASC Topic 815, Derivatives and Hedging (“ASC 815”). Simultaneously with the issuance of the 2024 Notes (defined below) in November 2017, the Company bought capped call options from certain counterparties to minimize the impact of potential dilution upon conversion. The premium for the capped call options was recorded as additional paid-in capital. For additional information related to the convertible debt transactions, please read Note 13, Indebtedness to the consolidated financial statements. Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for the goods or services provided. To determine revenue recognition for arrangements within the scope of ASC Topic 606, Revenue from Contracts with Customers (“ASC 606”), the Company performs the following five steps: (1) identify the contract with the customer; (2) identify the performance obligations in the contract; (3) determine the transaction price; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when or as the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect the consideration it is entitled to in exchange for the goods or services it transfers or provides to the customer. At contract inception, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when or as the performance obligation is satisfied. The only performance obligation in the Company’s contracts with customers is to timely deliver drug products to the customer’s designated location. Product revenues The Company distributes its products principally through its customers. The customers subsequently resell the products to patients and health care providers. The Company provides no right of return to the customers except in cases of shipping error or product defect. Product revenues are recognized when the customers take control of the products, which typically occurs upon delivery to the customers. For the years ended December 31, 2021, 2020 and 2019, the majority of the product revenues recognized were generated by the specialty distributor and specialty pharmacies in the U.S. Variable Consideration Product revenues are recorded at the net sales price (transaction price) which includes estimated reserves for variable consideration, such as Medicaid rebates, governmental chargebacks, including PHS chargebacks, prompt payment discounts, co-pay assistance and distribution fees. These reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contracts. Additional details relating to variable consideration follows: • Medicaid rebates relate to the Company’s estimated obligations to states under established reimbursement arrangements. Medicaid rebate reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Governmental chargebacks, including PHS chargebacks, relate to the Company’s estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices that the Company charges to wholesalers. The wholesaler charges the Company for the difference between what the wholesaler pays for the products and the ultimate selling price to the qualified healthcare providers. Chargeback reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider from the wholesaler, and the Company generally issues credits for such amounts within a few weeks of receiving notification of resale from the wholesaler. • Prompt payment discounts relate to the Company’s estimated obligations for credits to be granted to specialty pharmacies for remitting payment on their purchases within established incentive periods. Reserves for prompt payment discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. • Co-pay assistance relates to financial assistance provided to qualified patients, whereby the Company may assist them with prescription drug co-payments required by the patient’s insurance provider. Reserves for co-pay assistance are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Distribution fees relate to fees paid to customers in the distribution channel that provide the Company with inventory management, data and distribution services and are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the Company’s sale of products to the customers, these payments are accounted for as selling, general and administrative expenses. Reserves for distribution fees result in an increase in a liability if payments are required of the Company or a reduction of accounts receivable if no payments are required of the Company. Collaboration revenue The Company’s collaboration revenue is primarily generated from its collaboration arrangement with F. Hoffman-La Roche Ltd. (“Roche”). For more information, please read Note 3, Collaboration and License Agreements . At the inception of a collaboration arrangement, the Company first assesses whether the contractual arrangement is within the scope of ASC Topic 808, Collaborative Arrangements (“ASC 808”) to determine whether the arrangement involves a joint operating activity and involves two (or more) parties that are both active participants in the activity and exposed to significant risks and rewards dependent on the commercial success of such activity. Then the Company determines whether the collaboration arrangement in its entirety represents a contract with a customer as defined by ASC 606. If only a portion of the collaboration arrangement is potentially with a customer, the Company applies the distinct good or service unit-of-account guidance in ASC 606 to determine whether there is a unit of account that should be accounted for under ASC 606. For the units of account in the collaboration arrangement that do not represent a vendor-customer relationship, the Company will (i) consider applying other GAAP, including by analogy, or (ii) if there is no appropriate analogy, consistently apply a reasonable and rational accounting policy election. In general, by analogy to ASC 606, the Company identifies the performance obligations within the collaboration arrangement and identifies and allocates the transaction price the Company expects to receive on a relative standalone selling price basis to each performance obligation. Variable consideration, consisting of development and regulatory milestones, will be included in the transaction price only if the Company expects to receive such consideration and if it is probable that the inclusion of the variable consideration will not result in a significant reversal in the cumulative amount of revenue recognized under the arrangement. Sales-based royalty and milestone payments are excluded from the transaction price the Company expects to receive until the underlying sales occur because the license to the Company’s intellectual property is deemed to be the predominant item to which the royalties or milestones relate as it is the primary driver of value in its collaboration arrangement. For the recognition of revenue associated with each performance obligation, if the Company determines ASC 606 is not appropriate to apply by analogy, the Company will apply a reasonable, rational and consistently applied accounting policy election to faithfully depict the transfer of services to the collaboration partner over the estimated performance period. Up-front payments from a collaboration partner are recognized as deferred revenue when received and recognized as revenue over the estimated performance period. Reimbursement payments from a collaboration partner associated with cost sharing provisions in a collaboration arrangement are recognized as the related expense is incurred and classified as an offset to operating expenses. Valuation of Product Options The Company's collaboration arrangements may contain options which provide the collaboration partner with the right to obtain additional licenses. If an arrangement contains product options, by analogy to ASC 606, the Company evaluates the product options to determine whether they represent material rights, which may include options to acquire additional goods or services for free or at a discount. If the customer options are determined to represent material rights, they are recognized as a separate performance obligation at inception of the arrangement. The Company allocates a portion of the transaction price of the collaboration arrangement to material rights based on the relative standalone selling price. Amounts allocated to material rights are not recognized as revenue until related options are exercised or expire. Key assumptions to determine the standalone selling price of product options in a collaboration arrangement include, but are not limited to, forecasted revenues, development timelines, incremental costs related to the arrangement, discount rates and likelihood of technical and regulatory success. Research and Development Research and development expenses consist of costs associated with research activities as well as those with the Company’s product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Research and development expenses are expensed as incurred. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed when incurred. Direct research and development expenses associated with the Company’s programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants and other external services, such as data management and statistical analysis support and materials and supplies used in support of clinical programs. Indirect costs of the Company’s clinical programs include salaries, stock-based compensation and an allocation of its facility and technology costs. When third-party service providers’ billing terms do not coincide with the Company’s period-end, the Company is required to make estimates of its obligations to those third parties, including clinical trial and pharmaceutical development costs, contractual services costs and costs for supply of its drug candidates, incurred in a given accounting period and record accruals at the end of the period. The Company bases its estimates on its knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. Stock-Based Compensatio n The Company’s stock-based compensation programs include stock options, restricted stock awards (“RSAs”), restricted stock units (“RSUs”) and an employee stock purchase program (“ESPP”). The Company accounts for stock-based compensation using the fair value method. The fair value of stock options are estimated on the date of grant using the Black-Scholes-Merton option-pricing model. The fair values of RSAs and RSUs are based on the fair market value of the Company’s common stock on the date of the grant. The fair value of stock awards, with consideration given to estimated forfeitures, is recognized as stock-based compensation expense on a straight-line basis over the vesting period of the grants. For stock awards with performance-vesting conditions, the Company does not recognize compensation expense until it is probable that the performance-vesting condition will be achieved. Additionally, the Company granted its CEO options with service and market conditions. A market condition relates to the achievement of a specified price of the Company’s common stock, a specified amount of intrinsic value indexed to the Company’s common stock or a specified price of the Company’s common stock in terms of other similar equity shares. The grant date fair value for the options with service and market conditions is determined by a lattice model with Monte Carlo simulations and is recognized as stock-based compensation expense on a straight-line basis over the service period. Under the Company’s ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85 % of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The fair value of stock purchase rights is estimated using the Black-Scholes-Merton option-pricing model. The fair value of the look-back provision with the 15 % discount is recognized on a graded-vesting basis as stock-based compensation expense over the purchase period. Income Taxes The Company follows the asset and liability method of accounting for income taxes, which requires the recognition of deferred tax assets and liabilities for expected future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates in effect for the year in which the differences are expected to reverse. A valuation allowance is recorded to reduce the net deferred tax asset to zero when it is more likely than not that the net deferred tax asset will not be realized. The Company recognizes the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. The amount of the benefit that may be recognized in the financial statements is the largest amount that has a greater than 50% likelihood of being realized. The Company recognizes interest and penalties related to uncertain tax positions within income tax expense. It is the intention of the Company to reinvest the earnings of its non-U.S. subsidiaries in those operations and not to repatriate the earnings to the U.S. Accordingly, the Company does not provide for deferred taxes on the excess of the financial reporting over the tax basis in its investments in foreign subsidiaries as they are considered permanent in duration. Leases At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than 12 months are recognized on the consolidated balance sheets as right-of-use (“ROU”) assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize leases with terms of 12 months or less on the consolidated balance sheets. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its leases no less than on a quarterly basis. In addition, the Company’s lease agreements generally do not contain any residual value guarantees or restrictive covenants. Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term at lease commencement. The initial measurement of the lease liability is determined based on the future lease payments, which may include lease payments that depend on an index or a rate (such as the consumer price index or other market index). The Company initially measures payments based on an index or rate by using the applicable rate at lease commencement and subsequent changes in such rates are recognized as variable lease costs. Variable payments that do not depend on a rate or index are not included in the lease liability and are recognized as they are incurred. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense with unrecognized variable lease payments recognized as incurred. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. Components of a lease are bifurcated between lease components and non-lease components. The fixed and in-substance fixed contract consideration identified is then allocated based on the relative standalone price to the lease and non-lease components. However, ASC Topic 842, Leases, provides entities with a practical expedient that allows an accounting policy election to not separate lease and non-lease components by class of underlying asset. In using this expedient, entities would account for each lease component and the related non-lease component together as a single component. For new and amended real estate leases beginning after January 1, 2019, the Company elected to account for the lease and non-lease components together for existing classes of underlying assets and allocates the contract consideration to the lease component only. In contrast, the Company does not apply the practical expedient for leases embedded in manufacturing and supply agreements with certain of its contract manufacturing organizations and has instead allocated contract consideration between the lease and non-lease components based on their relative standalone price. Embedded Derivatives The Company evaluates certain of its financial and business development transactions to determine if embedded components of these contracts meet the definition of derivative under ASC 815. In general, embedded derivatives are required to be bifurcated from the host instrument if (i) the embedded feature is not clearly and closely related to the host contract and (ii) the embedded feature, if considered a freestanding instrument, meets the definition of a derivative. The embedded derivative is reported on the consolidated balance sheets at its fair value. Any change in fair value, as determined at each measurement period, is recorded as a component of the consolidated statements of operations and comprehensive lo |
LICENSE AND COLLABORATION AGREE
LICENSE AND COLLABORATION AGREEMENTS | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
LICENSE AND COLLABORATION AGREEMENTS | 3. LICENSE AND COLLABORATION AGREEMENTS F. Hoffman-La Roche Ltd. On December 21, 2019, the Company entered into a license, collaboration and option agreement with Roche and a stock purchase agreement (the “Roche Agreement”) with Roche, providing Roche with exclusive commercial rights to SRP-9001, the Company’s investigational gene therapy for Duchenne, outside the U.S. The Company retains all rights to SRP-9001 in the U.S. and will perform all development activities within the joint global development plan necessary to obtain and maintain regulatory approvals for SRP-9001 in the U.S. and the EU, unless otherwise agreed to by the parties. Further: (i) research and development expenses incurred under the joint global development plan will be equally shared between the Company and Roche, (ii) Roche is solely responsible for all costs incurred in connection with any development activities (other than those within the joint global development plan) that are necessary to obtain or maintain regulatory approvals outside the U.S, and (iii) the Company will continue to be responsible for the manufacturing of clinical and commercial supplies of SRP-9001. The Company has also granted Roche options to acquire ex-U.S. rights to certain future Duchenne-specific programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations, and cost sharing provisions. The agreement became effective on February 4, 2020. The Roche Agreement is governed by a joint steering committee (“JSC”) formed by representatives from Roche and the Company. The JSC, among other activities, manages the overall strategic alignment between the parties, approves any material update to the joint global development plan and budget and oversees the operations of the subcommittees. The Company received an aggregate of approximately $ 1.2 billion in cash consideration from Roche, consisting of an up-front payment and an equity investment in the Company. The Company may receive up to $ 1.7 billion in development, regulatory and sales milestones related to SRP-9001. Upon commercialization, the Company is also eligible to receive tiered royalty payments based on net sales. Of the $1.2 billion cash received from Roche, (i) $ 312.1 million, net of issuance costs, was allocated to the approximately 2.5 million shares of the Company’s common stock issued to Roche based on the closing price when the shares were issued, (ii) $ 485.0 million was allocated to the Options, and (iii) $ 348.7 million was allocated to a single, combined performance obligation (“Combined Performance Obligation”) comprised of: (i) the license of IP relating to SRP-9001 transferred to Roche, (ii) the related research and development services provided under the joint global development plan, (iii) the services provided to manufacture clinical supplies of SRP-9001, and (iv) the Company’s participation in the JSC, because the Company determined that the license of IP and related activities were not capable of being distinct from one another. The value assigned to the Options is reflected as deferred revenue and will not be recognized until an option is either: (i) exercised by Roche, or (ii) expires. If exercised, the value will be aggregated with the option exercise price and recognized over the applicable performance period. If expired, the value will be recognized immediately. The Company recognizes revenue related to the Combined Performance Obligation on a straight-line basis over the expected performance period of the joint global development plan, which is expected to extend through the fourth quarter of 2023. Revenue relating to future development, regulatory and sales milestones will be recognized when the milestone is probable of achievement (which is typically when the milestone has occurred). Any royalties payable by Roche in the future will be recognized in the period earned. For the years ended December 31, 2021 and 2020, the Company recognized $ 89.5 million and $ 84.2 million of collaboration and other revenues, respectively, the majority of which relates to the Combined Performance Obligation. As of December 31, 2021, the Company has total deferred revenue of $ 663.5 million associated with the Roche Agreement, of which $ 89.2 million is classified as current. Through 2021, no Options were exercised or expired. As such, deferred revenue related to the separate material rights for the Options remained unchanged at $ 485.0 million as of December 31, 2021 and 2020. The costs associated with co-development activities performed under the Roche Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the years ended December 31, 2021 and 2020, costs reimbursable by Roche and reflected as a reduction to operating expenses were $ 90.5 million and $ 66.5 million, respectively. As of December 31, 2021, there was $ 18.6 million of collaboration receivable included in other current assets. Genethon The Company entered into a sponsored research agreement in May 2017 and subsequently entered into a license and collaboration agreement with Genethon in November 2019 (the “Genethon Collaboration Agreement”) for Genethon’s micro-dystrophin gene therapy program for the treatment of Duchenne. The Genethon Collaboration Agreement grants the Company with exclusive rights in the majority of the world (primarily excluding the EU) to Genethon’s micro-dystrophin gene therapy products (“Genethon Products”) and other micro-dystrophin gene therapy products (“Other Licensed Products”). The Company may be liable for up to $ 157.5 million and $ 78.8 million in development, regulatory and sales milestones for the Genethon Products and Other Licensed Products, respectively. Furthermore, upon commercialization, the Company will be required to make tiered royalty payments based on net sales of the Genethon Products and the Other Licensed Products. Under the Genethon Collaboration Agreement, a joint steering committee was established to plan, monitor and coordinate development activities for Genethon Products and Other Licensed Products. The Company and Genethon are responsible for 75 % and 25 %, respectively, of development costs related to both the Genethon Products and the Other Licensed Products. Upon signing the Genethon Collaboration Agreement, the Company made an up-front payment of $ 28.0 million, which was recorded as research and development expense in the Company’s consolidated statements of operations and comprehensive loss for the year ended December 31, 2019. Additionally, for the years ended December 31, 2021, 2020 and 2019, the Company record ed $ 11.7 million, $ 10.1 million and $ 9.0 million, respectively, of research and development expense related to reimbursable development costs incurred by Genethon for Genethon Products. For the year ended December 31, 2021 , the Company recorded $ 4.0 million of research and development expense related to milestone achievements. No additional development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. StrideBio, Inc. In November 2019, the Company entered into a collaboration and license agreement and a stock purchase agreement (collectively, the “StrideBio Agreements”) with StrideBio, Inc. (“StrideBio”). The StrideBio Agreements grant the Company exclusive worldwide licenses to develop, collaborate and commercialize StrideBio’s adeno-associated viral capsids for gene therapy with respect to multiple development targets, to which the Company will have the exclusive right to perform development activities (“Sarepta Development Targets”) and targets that the two parties will jointly develop through completion of Phase 1/2 clinical trials (“Joint Development Targets”). For Sarepta Development Targets and Joint Development Targets, respectively, the Company may be liable for up to $ 450.0 million and $ 835.0 million in development, regulatory and sales milestone payments per target. Additionally, upon commercialization, the Company may be required to make tiered royalty payments based on net sales of each target. Upon signing the StrideBio Agreements, the Company made an up-front payment of $ 46.9 million, consisting of a cash payment of $ 17.5 million and 301,980 shares of the Company’s common stock delivered to StrideBio with a fair value of $ 29.4 million. The up-front payment was recorded as research and development expense in the Company’s consolidated statements of operations and comprehensive loss for the year ended December 31, 2019. As of December 31, 2021, no develop ment or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. In March 2021, the Company participated in StrideBio’s Series B round of financing and purchased approximately 0.2 million shares of preferred stock. As of December 31, 2021, the equity investment in StrideBio’s Series B preferred stock was approximately $ 1.8 million. Please read Note 5, Fair Value Measurements for further information. Myonexus Therapeutics In April 2019, the Company completed its acquisition of Myonexus Therapeutics, Inc. (“Myonexus”), a clinical-stage gene therapy biotechnology company that was developing gene therapies for LGMD for $ 178.3 million. The Company may also be required to make up to $ 200.0 million in additional payments to selling shareholders of Myonexus based on the achievement of certain sales-and regulatory- related milestones. The acquisition was accounted for as an asset acquisition as substantially all of the fair value of the gross assets acquired is concentrated in a group of similar identifiable assets (the five LGMD gene therapy programs). The Company determined that one regulatory-related milestone (not solely based on drug approval by the FDA) met the definition of a derivative and recorded a contingent consideration liability. As of December 31, 2021 and 2020, the contingent consideration liability was $ 42.0 million and $ 49.5 million, respectively. The changes in fair value are recorded as other expense in the Company’s consolidated statements of operations and comprehensive loss. Please read Note 5, Fair Value Measurements for further information on the change in fair value of the contingent consideration liability. Lysogene S.A. In October 2018, the Company entered into a license and collaboration agreement to develop and commercialize LYS-SAF302, a gene therapy to treat Mucopolysaccharidosis type IIIA (“MPS IIIA”) as well as an equity investment agreement with Lysogene S.A. (“Lysogene”). Under the license and collaboration agreement, in addition to the payment of up-front fees, the Company may be liable for a total of $ 102.8 million in development, regulatory and sales milestones. Furthermore, the Company may be required to make tiered royalty payments based on net sales of the LYS-SAF302 product subsequent to its commercialization. Beginning January 1, 2020, the Company began to reimburse Lysogene for expenses incurred in connection with development activities of the MPS IIIA product candidate. As of December 31, 2021, the Company owns 1,140,728 shares of common stock issued by Lysogene and recorded $ 2.5 million of equity investment in Lysogene as an other non-current asset in the Company’s consolidated balance sheets. The Company sent a termination notice to Lysogene on January 11, 2022 to notify them of the Company's intent to terminate the license and collaboration agreement. The termination will become effective July 11, 2022. The Company does not have to pay any early termination penalties to Lysogene but is liable for certain research and development reimbursements incurred in the six months following termination, which are not expected to be material. As of December 31, 2021, there was no accounting impact as a result of the termination of the license and collaboration agreement because the notice of termination did not occur until subsequent to year-end. Lacerta Therapeutics In August 2018, the Company entered into a license, development and option agreement (the “Lacerta License Agreement”) and a Series A Preferred Stock Purchase Agreement (the “Stock Purchase Agreement”) with Lacerta Therapeutics, Inc. (“Lacerta”). Pursuant to the Lacerta License Agreement, the Company licensed exclusive worldwide rights to develop, manufacture and commercialize a pre-clinical Pompe product candidate (the “Pompe License”). Lacerta also granted the Company exclusive options to enter into exclusive license agreements to develop, manufacture and commercialize other gene therapy product candidates for Sanfilipo syndrome and L-Amino Acid Decarboxylase Deficiency for additional consideration of $ 42.0 million (collectively, the “Options”) when (and if) the Options are exercised. Additionally, the Company may be liable for up to approximately $ 44.0 million in development, regulatory and sales milestones associated with the Pompe License and may be required to make tiered royalty payments based on net sales of the Pompe product subsequent to its commercialization. Under the Stock Purchase Agreement, the Company purchased approximately 4.5 million shares of Series A preferred stock issued by Lacerta. Under the agreements, the Company made an up-front payment of $ 38.0 million to Lacerta, of which $ 30.0 million was allocated to the Series A preferred stock investment and recorded as an other non-current asset in the accompanying consolidated balance sheets. Changes in the carrying value of the investment are reported as a component of earnings whenever there are triggering events that warrant impairment or observable price changes in orderly transactions for identical or similar investments of Lacerta in the future. For the years ended December 31, 2021, 2020 and 2019, the Company did no t record any changes in carrying value of the investment as Lacerta did not issue identical or similar shares during the corresponding periods. As of December 31, 2021, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional expense has been recognized. Nationwide Children’s Hospital In December 2016, the Company entered into an exclusive option agreement with Nationwide Children’s Hospital (“Nationwide”) from which the Company obtained an exclusive right to acquire a worldwide license of the micro-dystrophin gene therapy technology for Duchenne and Becker muscular dystrophy. In October 2018, the Company exercised the option and entered into a license agreement with Nationwide, which granted the Company exclusive worldwide rights to develop, manufacture and commercialize a micro-dystrophin gene therapy product candidate. In July 2021, the Company entered into an agreement with Nationwide to settle a dispute relating to a sublicense payment owed by the Company resulting from the up-front payment received from Roche under the Roche Agreement. The total sublicense payment payable to Nationwide under the agreement is $ 38.0 million, which was paid in July 2021. Approximately $ 9.3 million of this amount was previously expensed during the year ended December 31, 2020 with the rema ining $ 28.7 million expensed during the year ended December 31, 2021. The expense relating to this payment is recognized to research and development expense. As a result of this payment, the Company has no further financial obligations to Nationwide resulting from the up-front payment received under the Roche Agreement. As of December 31, 2021, no development or regulatory milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized. BioMarin Pharmaceutical, Inc. In July 2017, the Company and UWA entered into a settlement agreement with BioMarin. On the same day, the Company entered into a license agreement, which was subsequently amended in April 2019, with BioMarin and Academisch Ziekenhuis Leiden (“AZL”) (collectively with the Company, UWA and BioMarin, the “Settlement Parties”). Under these agreements and amendment, BioMarin agreed to provide the Company with an exclusive license to certain intellectual property with an option to convert the exclusive license into a co-exclusive license and the Settlement Parties agreed to stop most existing efforts to continue with ongoing litigation and opposition and other administrative proceedings concerning BioMarin’s intellectual property. BioMarin is also eligible to receive tiered royalty payments, ranging from 4 % to 8 %, based on the net sales for the three products and product candidates. In November 2021, the Company entered into a second settlement agreement and second amendment to the license agreement (the “Second Amendment”), which waived certain future milestone payments and altered royalty payment terms of the agreement. Under the Second Amendment, the Company may be liable for up to approximately $ 50.0 million in regulatory milestones for eteplirsen, casimersen and golodirsen. In addition, on and after July 1, 2022, the tiered royalty payments will range from 4 % to 5 %. The royalty terms under the license agreement will expire in March 2024 in the U.S., December 2024 in the EU and no later than December 2024 in other countries. As a result of execution of the license agreement with BioMarin, the Company recorded an in-licensed right intangible asset of $ 6.6 million in its consolidated balance sheets as of December 31, 2017, representing the fair value of the U.S. license to BioMarin’s intellectual property. The intangible asset is being amortized on a straight-line basis over the remaining life of the patent and has a carrying value of $ 3.2 million as of December 31, 2021. The FDA approval of AMONDYS 45 and VYONDYS 53 in February 2021 and December 2019, respectively, resulted in settlement charges to BioMarin of $ 10.0 million during each period and each were expensed as incurred. For the years ended December 31, 2021, 2020 and 2019, the Company recognized royalty expense of $ 31.4 million, $ 23.2 million and $ 19.4 million, respectively. As of December 31, 2021, no other regulatory milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized. University of Western Australia In April 2013, the Company and UWA entered into an amendment to an existing exclusive license agreement relating to the treatment of Duchenne by inducing the skipping of certain exons. The agreement was further amended in June 2016. Under the amended agreement, the Company may be obligated to make payments to UWA totaling up to $ 26.0 million upon the achievement of certain development, regulatory and sales milestones. Additionally, the Company is required to pay a low-single-digit percentage royalty on net sales of products covered by issued patents licensed under the agreements with UWA. Corresponding to the FDA approval of EXONDYS 51 in 2016, VYONDYS 53 in December 2019, and AMONDYS 45 in February 2021, the Company recorded milestone payments of $ 1.0 million, $ 0.5 million and $ 0.5 million as in-licensed right intangible assets in its consolidated balance sheets, respectively. Each in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patents and have a combined carrying value of $ 1.2 million as of December 31, 2021. For the years ended December 31, 2021, 2020 and 2019, the Company recorded $ 7.7 million, $ 5.7 million and $ 3.5 million in royalty expense, respectively, which is included in cost of sales, related to agreements with UWA. As of December 31, 2021 , no other development, regulatory or sales milestones were deemed probable of being achieved and, accordingly, no additional in-licensed rights or expenses have been recognized. Research and Option Agreements The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs, and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $ 11.0 million in resea rch milestone payments. Under these agreements, there are $ 187.0 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and tiered royalty payments based on the sales of the developed products upon commercialization. For the year ended December 31, 2021, the Company recogn ized $ 3.0 million of research milestone expense s, with no similar activity for the years ended December 31, 2020 and 2019. A s of December 31, 2021, the Company has not exercised any options nor have any additional research milestone payments become probable of occurring. Milestone Obligations Including the agreements discussed above, the Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of December 31, 2021, the Company may be obligated to make up to $ 4.0 billion of future development, regulatory, commercial, and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of December 31, 2021, which are discussed above. For the years ended December 31, 2021, 2020 and 2019, the Company recognized approximately $ 50.3 million, $ 47.3 million, $ 113.2 million relating to certain up-front, milestone, settlement and other payments as research and development expense, respectively, under these agreements. The Company is also obligated to pay royalties on net sales of certain of its products related to these collaboration and license agreements. The royalty rates range from the low-single-digit to high teens percentages for both inside and outside the U.S. |
GAIN FROM SALE OF PRIORITY REVI
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 12 Months Ended |
Dec. 31, 2021 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
GAIN FROM SALE OF PRIORITY REVIEW VOUCHER | 4. GAIN FROM SALE OF PRIORITY REVIEW VOUCHER I n February 2021, the Company entered into an agreement to sell the rare pediatric disease PRV (the “ AMONDYS 45 PRV ” ) it received from the FDA in connection with the approval of AMONDYS 45. Following the termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, in April 2021, the Company completed its sale of the AMONDYS 45 PRV and received proceeds of $ 102.0 million, with no commission costs, which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale. In February 2020, the Company entered into an agreement to sell the rare pediatric disease PRV (the “VYONDYS 53 PRV ” ) it received from the FDA in connection with the approval of VYONDYS 53. Following the early termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, in March 2020, the Company completed its sale of the VYONDYS 53 PRV and received proceeds of $ 108.1 million, net of commission, which was recorded as a gain from sale of the PRV as it did not have a carrying value at the time of the sale. |
FAIR VALUE MEASUREMENTS
FAIR VALUE MEASUREMENTS | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
FAIR VALUE MEASUREMENTS | 5. FAIR VALUE MEASUREMENTS There were no transfers between Levels 1, 2 and 3 during the year ended December 31, 2021 . The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of December 31, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,562,358 $ 1,562,358 $ — $ — Strategic equity investments 34,892 2,480 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,597,500 $ 1,565,088 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2020 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 629,440 $ 629,440 $ — $ — Government and government agency 1,037,981 1,037,981 — — Strategic equity investments 38,799 3,699 — 35,100 Certificates of deposit 250 250 — — Total assets $ 1,706,470 $ 1,671,370 $ — $ 35,100 Liabilities Contingent consideration $ 50,800 $ — $ — $ 50,800 Total liabilities $ 50,800 $ — $ — $ 50,800 The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds, certificates of deposit and the Company’s strategic investment in Lysogene, a publicly traded company in France, as more fully described in Note 3, License and Collaboration Agreements . The Company did not hold any government and government agency bonds as of December 31, 2021. Certain of the government and government agency bonds are publicly traded fixed income securities and were presented as cash equivalents on the consolidated balance sheets as of December 31, 2020. The Company’s assets with fair value categorized as Level 3 within the fair value hierarchy consists of a strategic investment in Series A preferred stock of Lacerta as more fully described in Note 3, License and Collaboration Agreements and strategic investments in another two private companies. At the end of each reporting period, the fair value of the Company's strategic investments will be adjusted if the issuers were to issue similar or identical equity securities or when there is a triggering event for impairment. During the year ended December 31, 2021, the Company recorded an impairment loss of $ 4.5 million related to its investment in one of the private companies. The following table represents a roll-forward of the fair value of Level 3 financial assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Fair value, beginning of year $ 35,100 $ 30,000 Additions 1,800 5,100 Changes in estimated fair value ( 4,488 ) — Fair value, end of year $ 32,412 $ 35,100 The Company’s contingent consideration liability with fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus selling shareholders as well as to two academic institutions under separate license agreements that meet the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreements . The contingent consideration liability was estimated using an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. This fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. Significant changes which increase or decrease the probabilities of achieving the milestone or shorten or lengthen the time required to achieve the milestone would result in a corresponding increase or decrease in the fair value of the liability. At the end of each reporting period, the fair value is adjusted to reflect the most current assumptions through earnings. The following table represents a roll-forward of the fair value of Level 3 financial liabilities for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Fair value, beginning of year $ 50,800 $ 5,200 Additions — 600 Changes in estimated fair value, net ( 7,200 ) 45,000 Fair value, end of year $ 43,600 $ 50,800 A net decrease of $ 7.2 million and net increase of $ 45.0 million was recorded during the years ended December 31, 2021 and December 31, 2020, respectively, to account for the change in fair value of existing contingent consideration liabilities. These changes, which are recorded through earnings, were a result of updates made to certain inputs and assumptions impacting the probability-weighted expected cash flows, principally the probability of success of the underlying programs, the estimate of the year that the payments are expected to be made, the expected approval date of the underlying programs and the estimate of the amount of payments to be ultimately made. An increase of $ 0.6 million was recorded during the year ended December 31, 2020 to account for new contingent consideration liabilities associated with new license agreements with certain academic institutions that meet the definition of a derivative. As of December 31, 2021, the contingent consideration was recorded as a non-current liability on the Company's consolidated balance sheets. The carrying amounts reported in the consolidated balance sheets for cash and cash equivalents, accounts receivable and accounts payable approximate fair value because of the immediate or short-term maturity of these financial instruments. For fair value information related to the Company’s debt facilities, please read Note 13, Indebtedness . |
CASH, CASH EQUIVALENTS AND MARK
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 12 Months Ended |
Dec. 31, 2021 | |
Cash and Cash Equivalents [Abstract] | |
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES | 6. CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the consolidated balance sheets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Money market funds $ 1,562,358 $ 629,440 Government and government agency bonds — 602,058 Total $ 1,562,358 $ 1,231,498 It is the Company’s policy to mitigate credit risk in its financial assets by maintaining a well-diversified portfolio that limits the amount of exposure as to maturity and investment type. The Company did no t hold any available-for-sale securities at December 31, 2021. The weighted average maturity of the Company’s available-for-sale securities as of December 31, 2020 was approximately two months . The following tables summarize the Company’s cash, cash equivalents and investments for each of the periods indicated: As of December 31, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As reported: Cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As of December 31, 2020 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 900,590 $ — $ — $ 900,590 Government and government agency bonds 1,037,959 22 — 1,037,981 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 As reported: Cash and cash equivalents $ 1,502,639 $ 9 $ — $ 1,502,648 Short-term investments 435,910 13 — 435,923 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 |
PRODUCT REVENUES, NET, ACCOUNTS
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES | 12 Months Ended |
Dec. 31, 2021 | |
Receivables, Net, Current [Abstract] | |
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES | 7. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES The following table summarizes the Company's product revenues, net disaggregated by product for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) EXONDYS 51 $ 454,361 $ 422,007 $ 380,718 VYONDYS 53 89,511 33,858 115 AMONDYS 45 68,529 — — Products, net $ 612,401 $ 455,865 $ 380,833 The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of December 31, 2021 2020 (in thousands) Product sales receivable, net of discounts and allowances $ 152,990 $ 100,870 Government contract receivables — 470 Total accounts receivable, net $ 152,990 $ 101,340 The relevant government audit was completed and, as a result, the outstanding balance for government contract receivables was received as of December 31, 2021. The following table summarizes an analysis of the change in reserves for discounts and allowances for the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 Provision 9,700 52,180 6,384 10,175 78,439 Payments/credits ( 8,007 ) ( 55,147 ) ( 5,941 ) ( 9,877 ) ( 78,972 ) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 13,308 78,637 9,400 16,107 117,452 Payments/credits ( 14,790 ) ( 59,902 ) ( 8,551 ) ( 14,713 ) ( 97,956 ) Balance, as of December 31, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 The following table summarizes the total reserves above included in the Company’s consolidated balance sheets for the periods indicated: As of December 31, 2021 2020 (in thousands) Reduction to accounts receivable $ 8,321 $ 8,352 Component of accrued expenses 62,145 42,618 Total reserves $ 70,466 $ 50,970 |
INVENTORY
INVENTORY | 12 Months Ended |
Dec. 31, 2021 | |
Inventory Disclosure [Abstract] | |
INVENTORY | 8. INVENTORY The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Raw materials $ 58,822 $ 71,717 Work in progress 230,194 139,704 Finished goods 26,717 20,540 Total inventory $ 315,733 $ 231,961 No material inventory reserves existed as of December 31, 2021 or 2020. Non-current inventory, which consists of raw materials and work in progress, is included in other non-current assets in the Company's consolidated balance sheets. Non-current inventory is anticipated to be consumed beyond our normal operating cycle. As of December 31, 2020, we had no non-current inventory. The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Balance sheet classification Inventory $ 186,212 $ 231,961 Other non-current assets 129,521 — Total inventory $ 315,733 $ 231,961 |
OTHER ASSETS
OTHER ASSETS | 12 Months Ended |
Dec. 31, 2021 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
OTHER ASSETS | 9. OTHER ASSETS The following table summarizes the Company’s other current assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Manufacturing-related deposits and prepaids $ 93,656 $ 134,430 Collaboration receivable 18,647 34,184 Prepaid clinical and pre-clinical expenses 12,667 16,224 Prepaid maintenance services 8,452 6,411 Prepaid insurance 5,282 4,158 Prepaid research expenses 3,082 5,854 Prepaid income tax 1,100 4,939 Leasehold improvement receivable — 3,059 Other 6,142 4,065 Total other current assets $ 149,028 $ 213,324 The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Non-current inventory $ 129,521 $ — Manufacturing-related deposits and prepaids 112,765 148,525 Strategic investments 34,892 38,799 Restricted cash and investments 9,904 9,315 Prepaid clinical expenses 2,007 3,395 Other 3,860 3,669 Total other non-current assets $ 292,949 $ 203,703 |
PROPERTY AND EQUIPMENT, NET
PROPERTY AND EQUIPMENT, NET | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
PROPERTY AND EQUIPMENT, NET | 10. PROPERTY AND EQUIPMENT, NET Property and equipment are recorded at historical cost, net of accumulated depreciation. The following table summarizes components of property and equipment, net, for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Leasehold improvements $ 103,370 $ 55,019 Lab and manufacturing equipment 64,613 49,571 Building and improvements 47,605 39,397 Software and computer equipment 42,506 33,948 Furniture and fixtures 9,242 7,010 Land 5,183 5,183 Land improvements 4,921 3,610 Office equipment 1,189 1,178 Construction in progress 25,159 71,541 Property and equipment, gross 303,788 266,457 Less: accumulated depreciation ( 112,632 ) ( 76,027 ) Property and equipment, net $ 191,156 $ 190,430 For the years ended December 31, 2021, 2020 and 2019, depreciation expense totaled $ 36.6 million, $ 25.2 million and $ 22.8 million, respectively. |
INTANGIBLE ASSETS, NET
INTANGIBLE ASSETS, NET | 12 Months Ended |
Dec. 31, 2021 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
INTANGIBLE ASSETS, NET | 11. INTANGIBLE ASSETS, NET The following table summarizes the components of the Company’s intangible assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Patents $ 12,382 $ 11,210 In-licensed rights 8,573 8,073 Software licenses 299 1,554 Intangible assets, gross 21,254 20,837 Less: accumulated amortization ( 7,015 ) ( 7,209 ) Intangible assets, net $ 14,239 $ 13,628 The in-licensed rights relate to agreements with BioMarin and UWA. As a result of the FDA approval of EXONDYS 51, VYONDYS 53 and AMONDYS 45, the Company recorded in-licensed rights of $ 1.0 million, $ 0.5 million and $ 0.5 million, respectively. Following the execution of the settlement and license agreements with BioMarin in July 2017, the Company recorded a $ 6.6 million intangible asset related to EXONDYS 51 in the U.S. The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patent because the life of the related patent reflects the expected time period that the Company will benefit from the in-licensed right. For more information about the in-licensed rights, please read Note 3, License and Collaboration Agreements . For the years ended December 31, 2021, 2020 and 2019, the Company rec orded $ 0.7 million, $ 0.7 million and $ 0.8 million, respectively, of amortization related to the in-licensed rights. Patent amortization expen se was $ 0.6 million, $ 0.6 million and $ 0.4 million for the years ended December 31, 2021, 2020 and 2019, respectively. The Company also expensed the remaining net book value of previously capitalized patents that were later abandoned of $ 0.5 million, $ 0.1 million and $ 0.2 million for the years ended December 31, 2021, 2020 and 2019, respectively, which were included in research and development expenses on the consolidated statements of operations and comprehensive loss. Amortization related to internal use software was less than $ 0.1 million, $ 0.5 million and $ 0.4 million for the years ended December 31, 2021, 2020 and 2019, respectively. The following table summarizes the estimated future amortization for intangible assets: As of (in thousands) 2022 $ 1,395 2023 1,394 2024 1,391 2025 1,330 2026 1,200 Thereafter 7,529 Total $ 14,239 |
ACCRUED EXPENSES
ACCRUED EXPENSES | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
ACCRUED EXPENSES | 12. ACCRUED EXPENSES The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Accrued contract manufacturing costs $ 104,311 $ 36,543 Product revenue related reserves 62,145 42,618 Accrued employee compensation costs 48,299 50,803 Accrued clinical and pre-clinical costs 25,955 22,169 Accrued royalties 11,965 7,793 Accrued professional fees 9,381 10,221 Accrued collaboration cost-sharing 2,887 3,516 Accrued interest expense 1,045 1,045 Accrued milestone and license expense 100 9,380 Other 5,609 9,465 Total accrued expenses $ 271,697 $ 193,553 |
INDEBTEDNESS
INDEBTEDNESS | 12 Months Ended |
Dec. 31, 2021 | |
Debt Disclosure [Abstract] | |
INDEBTEDNESS | 13. INDEBTEDNESS 2024 Convertible Notes On November 14, 2017, the Company issued $ 570.0 million senior notes due on November 15, 2024 . The 2024 Notes were issued at face value and bear interest at the rate of 1.50 % per annum, payable semi-annually in cash on each May 15 and November 15, commencing on May 15, 2018. The 2024 Notes contain customary covenants and events of default, occurrence of which permits the certain holders to accelerate all outstanding obligations, including principal and interest. The Company incurred $ 10.6 million of offering costs, which represents the total debt discount on the 2024 Notes at issuance. The debt discount is amortized under the effective interest method and recorded as additional interest expense over the life of the 2024 Notes. Upon conversion, the Company may pay cash, shares of its common stock or a combination of cash and stock, as determined by the Company in its discretion. The 2024 Notes may be convertible into 7,763,552 shares of the Company’s common stock under certain circumstances prior to maturity at a conversion rate of 13.621 shares per $1,000 principal amount of the 2024 Notes, which represents a conversion price of $ 73.42 per share, subject to adjustment under certain conditions. To minimize the impact of potential dilution upon conversion of the 2024 Notes, the Company separately entered into capped call transactions with certain counterparties. The capped calls have a strike price of $ 73.42 and a cap price of $ 104.88 and are exercisable when and if the 2024 Notes are converted. If, upon conversion of the 2024 Notes, the price of the Company’s common stock is between the strike price and the cap price of the capped calls, the counterparties will deliver shares of the Company’s common stock and/or cash with an aggregate value equal to the difference between the price of the Company’s common stock at the conversion date and the strike price, multiplied by the number of shares of the Company’s common stock related to the capped calls being exercised. The Company paid $ 50.9 million for these capped calls transactions, which was recorded as additional paid-in capital. Upon adoption of ASU 2020-06, the 2024 Notes are accounted for as a single liability measured at its amortized cost. The cumulative effect of the accounting change as of January 1, 2021 increased the carrying amount of the convertible notes by $ 96.8 million, reduced the accumulated deficit by $ 60.2 million and reduced additional paid-in capital by $ 157.0 million. Upon adoption of ASU 2020-06, the effective interest rate on the liability component of the 2024 Notes for the year ended December 31, 2021 was 1.9 %. The effective interest rate on the liability component of the 2024 Notes for each of the years ended December 31, 2020 and 2019 was 6.9 %. Interest expense of the 2024 Notes was reduced by $ 22.4 million for the year ended December 31, 2021 as a result of adoption of this guidance. For the years ended December 31, 2021, 2020 and 2019, the interest expense related to the 2024 Notes was $ 10.7 million, $ 31.4 million and $ 29.9 million, respectively. December 2019 Term Loan On December 13, 2019, the Company entered into a loan agreement (the “Credit Agreement”) which provides a term loan (“December 2019 Term Loan”) of $ 500.0 million with Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP (collectively, the “Lenders”). The December 2019 Term Loan has two tranches: A and B, each of which had an initial loan amount of $ 250.0 million. On September 24, 2020, the Company entered into a first amendment to Credit Agreement (the “Amendment”),which increased the aggregate principal amount of tranche B of the December 2019 Term Loan from $250.0 million to $ 300.0 million and revised certain fees. Tranche A and B of the December 2019 Term Loan were drawn down on December 20, 2019 and November 2, 2020 and will mature on December 20, 2023 and December 31, 2024 , respectively, when the principal amount of the loan will become due. Borrowings under the Credit Agreement bear interest at a rate per annum equal to 8.5 %, which shall be payable quarterly in arrears. The Company may voluntarily prepay, in whole or in part, the outstanding balance under the December 2019 Term Loan at any time after the tranche A closed but may be obligated to make additional financial considerations to the Lenders. Upon draw-down of tranche A and B, the Company received net proceeds of $ 244.9 million and $ 291.1 million, net of debt discounts of $ 9.4 million and $ 14.9 million relating to fees payable to the Lenders, respectively. Of the fees payable to the Lenders related to tranche A and B, $ 5.0 million and $ 6.0 million are expected to be paid on December 20, 2023 and December 31, 2024, respectively. The debt discounts are being treated as a reduction to the carrying value of tranche A and B of the December 2019 Term Loan and amortized as interest expense over the term of the loan based on an effective interest method. Debt issuance costs associated with the draw-down of tranche A and B were $ 0.7 million and less than $ 0.1 million, respectively. As of December 31, 2021, the Company recorded approximately $ 1,096.9 million as long-term debt on the consolidated balance sheets. For the years ended December 31, 2021, 2020 and 2019, the Company recorded $ 63.5 million, $ 59.9 million and $ 30.7 million of interest expense, respectively. The following table summarizes the Company’s debt facilities for the periods indicated: As of December 31, 2021 2020 (in thousands) Principal amount of the 2024 Notes $ 569,993 $ 569,993 Unamortized discount - equity component — ( 98,721 ) Unamortized discount - debt issuance costs ( 6,320 ) ( 6,510 ) Net carrying value of 2024 Notes 563,673 464,762 Principal amount of the 2019 Term Loan 550,000 550,000 Unamortized discounts ( 16,797 ) ( 22,269 ) Net carrying value of 2019 Term Loan 533,203 527,731 Total carrying value of debt facilities $ 1,096,876 $ 992,493 Fair value of 2024 Notes $ 846,138 $ 1,394,249 Fair value of 2019 Term Loan 576,085 550,000 Total fair value of debt facilities $ 1,422,223 $ 1,944,249 The fair value of the 2024 Notes is based on open market trades and is classified as Level 1 in the fair value hierarchy. The fair value of the December 2019 Term Loan is classified as Level 2 in the fair value hierarchy and is determined using a discounted cash flow analysis with market interest rates adjusted for credit risk as a significant input. The following table summarizes the total gross payments due under the Company’s debt arrangements: As of (in thousands) 2022 $ — 2023 250,000 2024 869,993 2025 — 2026 — Thereafter — Total payments $ 1,119,993 The aggregate annual maturities of long-term debt and interest during the years ending December 31, 2022, 2023 and 2024 are $ 55.9 million, $ 305.3 million and $ 903.4 million, respectively. |
EQUITY
EQUITY | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
EQUITY | 14. EQUITY In October 2021, the Company issued approximately 7.1 million shares of common stock through an underwritten public offering. The offering price was $ 81.00 per share. The Company received net proceeds of approximately $ 548.5 million from the offering, net of commission and offering expenses of approximately $ 26.5 million. In February 2020, the Company issued approximately 2.5 million shares of common stock with a fair value of $ 312.1 million, net of direct transaction fees of $ 4.3 million as part of the Roche transaction (see Note 3, License and Collaboration Agreements ). In November 2019, the Company issued approximately 0.3 million shares of common stock with a fair value of $ 29.4 million as part of the up-front consideration to StrideBio (see Note 3, License and Collaboration Agreements ). In March 2019, the Company sold approximately 2.6 million shares of common stock through an underwritten public offering. The offering price was $ 140.41 per share. The Company received net proceeds of approximately $ 365.4 million from the offering, net of commission and offering expenses of approximately $ 0.3 million. |
STOCK-BASED COMPENSATION
STOCK-BASED COMPENSATION | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
STOCK-BASED COMPENSATION | 15. STOCK-BASED COMPENSATION In June 2013, the Company’s stockholders approved the 2013 Employee Stock Purchase Plan (the “2013 ESPP”) which authorized 0.3 million shares of common stock available to be issued. In June 2016 and 2019, the Company’s stockholders approved an additional 0.3 million and 0.5 million shares, respectively, of common stock available for issuance under the 2013 ESPP. As of December 31, 2021, 0.4 million shares of common stock remain available for future grant under the 2013 ESPP. In September 2014, the Company initiated the 2014 Employment Commencement Incentive Plan (the “2014 Plan”). The 2014 Plan, which authorized 0.6 million shares of common stock to be issued and allows for the grant of stock options, SARs, RSAs, RSUs, performance shares and performance units. As of December 31, 2021, 7.0 million shares ha ve been added to the Company's 2014 Plan. As of December 31, 2021, 1.5 million share s of common stock remain available for future grant under the 2014 Plan. In June 2018, the Company’s stockholders approved the 2018 Equity Incentive Plan (the “2018 Plan”). The 2018 Plan, which authorized 2.9 million shares of common stock to be issued, allows for the grant of stock options, SARs, RSAs, RSUs, performance shares and performan ce units. In June 2020, an additional 3.8 million shares of common stock were approved by the Company’s stockholders and added to the 2018 Plan. Together with the roll-over shares from the Company’s 2011 Equity Incentive Plan, 4.2 million shares of common stock rema in available for future grant under the 2018 Plan as of December 31, 2021. Stock Options In general, stock options have a ten-year term and vest over a four-year period, with one-fourth of the underlying shares vesting on the first anniversary of the grant and 1/48th of the underlying shares vesting monthly thereafter, such that the underlying shares will be fully vested on the fourth anniversary of the grant, subject to the terms of the applicable plan under which they were granted. The fair values of stock options granted during the periods presented are measured on the date of grant using the Black-Scholes-Merton option-pricing model, with the following assumptions: For the Year Ended December 31, 2021 2020 2019 Risk-free interest rate (1) 0.4 - 1.3 % 0.1 - 1.3 % 1.4 - 2.5 % Expected dividend yield (2) — — — Expected term (3) 4.99 years 5.00 years 5.04 years Expected volatility (4) 60.1 - 70.8 % 57.3 - 68.2 % 52.5 - 68.9 % (1) The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. (2) The expected dividend yield is zero as the Company has not paid any dividends to date and does not expect to pay dividends in the future. (3) The expected term is estimated using historical exercise behavior. (4) The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. The amounts estimated according to the Black-Scholes-Merton option-pricing model may not be indicative of the actual values realized upon the exercise of these options by the holders. The following tables summarize the Company’s stock option activity for each of the periods indicated: For the Year Ended December 31, 2021 Weighted Average Exercise Shares Price Grants outstanding at beginning of 7,844,947 $ 70.61 Granted 1,685,860 86.41 Exercised ( 283,622 ) 45.71 Cancelled and forfeited ( 1,050,264 ) 112.18 Grants outstanding at end of the period 8,196,921 $ 69.39 Grants exercisable at end of the period 2,532,438 $ 84.71 Grants vested and expected to vest at 7,867,722 $ 68.17 The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2021, 2020 and 2019 was $48.16, $ 61.38 and $ 70.93 , respectively. Weighted Aggregate Average Intrinsic Remaining Value Contractual (in thousands) Life (Years) Options outstanding at December 31, 2021 $ 248,479 6.4 Options exercisable at December 31, 2021 $ 59,069 5.6 Options vested and expected to vest at December 31, 2021 $ 247,434 6.3 The following table summarizes the Company’s stock options vested and exercised for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Aggregate grant date fair value of stock options $ 79,068 $ 80,355 $ 50,878 Aggregate intrinsic value of stock options $ 10,622 $ 144,750 $ 109,707 As of December 31, 2021, there were 3,300,000 options with service and market conditions included in the grants outstanding for the Company’s CEO, which have a five-year cliff vesting schedule and a grant date fair value of $ 13.48 determined by a lattice model with Monte Carlo simulations. These options have an exercise price of $ 34.65 and were granted in June 2017. Restricted Stock Awards The Company has granted RSAs to members of its board of directors and certain employees. The following table summarizes the Company’s RSA activity for each of the periods indicated: For the Year Ended December 31, 2021 Weighted Average Grant Date Shares Fair Value Grants outstanding at beginning of the 48,875 $ 50.11 Granted — — Vested ( 48,875 ) 50.11 Forfeited — — Grants outstanding at end of the period — $ — Restricted Stock Units The Company grants RSUs to members of its board of directors and employees. The following table summarizes the Company’s RSU activity for the periods indicated: For the Year Ended December 31, 2021 Weighted Average Grant Date Shares Fair Value Grants outstanding at beginning of the 947,079 $ 121.46 Granted 987,464 85.32 Vested ( 276,980 ) 119.54 Forfeited ( 337,357 ) 106.37 Grants outstanding at end of the period 1,320,206 $ 98.69 2013 Employee Stock Purchase Plan Under the Company’s 2013 ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85 % of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The 24 -month offering period will end on August 31, 2023 . The following table summarizes the Company’s ESPP activity for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 Number of shares purchased 111,171 102,031 92,086 Proceeds received (in millions) $ 7.8 $ 7.5 $ 5.1 Stock-based Compensation Expense The following table summarizes stock-based compensation expense by function included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2021 2020 2019 (in thousands) Research and development $ 50,526 $ 41,671 $ 27,681 Selling, general and administrative 63,417 66,399 50,921 Total stock-based compensation $ 113,943 $ 108,070 $ 78,602 The following table summarizes stock-based compensation expense by grant type included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2021 2020 2019 (in thousands) Stock options $ 68,995 $ 68,832 $ 53,427 Restricted stock awards/units 40,055 33,457 20,103 Employee stock purchase plan 4,893 5,781 5,072 Total stock-based compensation $ 113,943 $ 108,070 $ 78,602 As of December 31, 2021 , there was $ 175.7 million of total unrecognized stock-based compensation expense related to the Company’s stock-based compensation plans. The expense is expected to be recognized over a weighted-average period of approximately 3 years. Of this amount, $ 92.4 million relates to options with service conditions only, $ 4.3 million relates to awards with service and market conditions, and the remaining $ 79.0 million related to restricted stock units with service conditions only. |
401 (K) PLAN
401 (K) PLAN | 12 Months Ended |
Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |
401 (K) PLAN | 16. 401 (K) PLAN The Company sponsors a 401(k) Plan (“the Plan”) in the U.S. and other retirement plans in the rest of the world, all of which are defined contribution plans. The Plan is available to all employees who are age 21 or older. Participants may make voluntary contributions and the Company makes matching contributions according to the Plan’s matching formula. Matching contributions fully vest after one year of service for all employees. The expense related to the Plan primarily consists of the Company’s matching contributions. Expense related to the Plan totaled $ 5.3 million , $ 5.3 million and $ 3.4 million for the years ended December 31, 2021, 2020 and 2019 , respectively. |
OTHER INCOME (LOSS)
OTHER INCOME (LOSS) | 12 Months Ended |
Dec. 31, 2021 | |
Other Income and Expenses [Abstract] | |
OTHER INCOME (LOSS) | 17. OTHER INCOME (LOSS) The following table summarizes other income (loss) for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Interest expense $ ( 63,525 ) $ ( 59,947 ) $ ( 30,669 ) Interest income 354 2,970 7,238 Amortization of investment discount 157 4,489 15,350 Gain from sale of Priority Review Voucher 102,000 108,069 — Gain (loss) on contingent consideration, net* 7,200 ( 45,000 ) — Impairment of equity investment ( 4,488 ) — — Other (expense) income ( 936 ) 517 ( 236 ) Total other income (loss) $ 40,762 $ 11,098 $ ( 8,317 ) |
INCOME TAXES
INCOME TAXES | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
INCOME TAXES | 18. INCOME TAXES The following table summarizes the loss before the provision (benefit) for income taxes by jurisdiction for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Domestic $ ( 47,633 ) $ ( 204,956 ) $ ( 489,747 ) Foreign ( 371,315 ) ( 348,109 ) ( 224,133 ) Total $ ( 418,948 ) $ ( 553,065 ) $ ( 713,880 ) The following table summarizes provision (benefit) for income taxes in the accompanying consolidated financial statements for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Current provision: Federal $ — $ 4 $ — State ( 40 ) 624 521 Foreign 181 680 1,050 Total current provision 141 1,308 1,571 Deferred benefit: Federal — — ( 15 ) State — — ( 5 ) Foreign ( 309 ) ( 245 ) ( 356 ) Total deferred benefit ( 309 ) ( 245 ) ( 376 ) Total income tax (benefit) expense $ ( 168 ) $ 1,063 $ 1,195 The following table summarizes the reconciliation between the Company’s effective tax rate and the statutory income tax rate for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 Federal income tax rate 21.0 % 21.0 % 21.0 % State taxes 0.4 0.6 4.5 Research and development and other tax 10.0 10.1 5.1 Valuation allowance ( 9.8 ) ( 21.3 ) ( 16.8 ) Permanent differences ( 0.3 ) ( 1.6 ) 2.3 Stock-based compensation ( 3.0 ) 3.5 ( 0.6 ) Basis difference in subsidiary — — ( 8.4 ) Foreign rate differential ( 18.4 ) ( 12.9 ) ( 7.4 ) Other 0.1 0.4 0.1 Effective tax rate ( 0.0 ) % ( 0.2 ) % ( 0.2 ) % Permanent differences affecting the Company’s effective tax rate primarily include excess stock-based compensation tax deductions, net of non-deductible stock-based compensation and limitation on deductibility of officer compensations. In February 2019, the Company exercised its option to acquire Myonexus. Accumulated costs of $ 253.7 million, associated with the Myonexus acquisition, were expensed for U.S. GAAP purposes. Of the $ 253.7 million in accumulated costs, $ 85.0 million relates to up-front and milestone payments as a result of the execution of the Warrant Agreement in May 2018 as well as certain development milestones being achieved or becoming probable of being achieved and $ 168.7 million relates to the exercise of the exclusive option to acquire Myonexus in February 2019. For U.S. income tax purposes, these costs are considered to be an investment in the subsidiary and are not currently deductible for tax purposes. The permanent difference related to this acquisition is separately stated in the rate reconciliation above. The following table summarizes the analysis of the deferred tax assets and liabilities for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 330,392 $ 333,703 Difference in depreciation and amortization 31,563 31,259 Research and development tax credits 201,512 159,917 Stock-based compensation 38,132 31,212 Lease liabilities 10,890 13,120 Capitalized inventory 24,172 35,959 Debt discount 5,875 — Other 38,315 28,381 Total deferred tax assets 680,851 633,551 Deferred tax liabilities: Right of use asset ( 7,405 ) ( 8,772 ) Debt discount — ( 18,044 ) Total deferred tax liabilities ( 7,405 ) ( 26,816 ) Valuation allowance ( 672,319 ) ( 605,848 ) Net deferred tax assets $ 1,127 $ 887 The Company has evaluated the positive and negative evidence bearing upon the realizability of its U.S. net deferred tax assets, which are comprised principally of federal and state net operating loss carryforwards, research and development tax credit carryforwards, stock-based compensation expense, capitalized inventory, and intangibles. Under the applicable accounting standards, management has considered the Company’s history of losses and concluded that it is more likely than not that the Company will not recognize the benefits of net federal and state deferred tax assets. Accordingly, a full valuation allowance of the U.S. net deferred tax asset is maintained at December 31, 2021 and 2020. The net change in the valuation allowance for deferred tax assets was an increase of $ 66.5 million and $ 117.0 million for the years ended December 31, 2021 and 2020, respectively. This increase for the year ended December 31, 2021 was primarily due to the generation of federal and state income tax credits and a decrease in the debt discount deferred tax liability that was recorded through additional paid-in capital as a result of the Company's early adoption of ASU 2020-06. The Company generated foreign deferred tax assets mainly consisting of net operating loss carryforwards, stock-based compensation and unrealized gain/losses. Based upon the income projections in the majority of the foreign jurisdictions, the Company believes it will realize the benefit of its future deductible differences in these jurisdictions. As such, the Company has not recorded a valuation allowance against these foreign jurisdictions. Brazil, the Netherlands, Czech Republic and one of the entities in the United Kingdom have generated deferred tax assets, which consist of net operating loss carryforwards and stock-based compensation expense. The Company has concluded that it is more likely than not that we will not recognize the future benefits of the deferred tax assets, and accordingly, a full valuation allowance has been recorded against these foreign deferred tax assets. As of December 31, 2 021, the Company had federal and state net operating loss carryforwards of $ 1,280.4 million and $ 841.2 million, respectively, available to reduce future taxable income. The federal and state net operating loss carry forwards of $ 577.2 million and $ 797.0 million will expire at various dates between 2022 and 2041 . The federal and state net operating loss carryforwards of $ 703.2 million and $ 44.2 million, respectively, can be carried forward indefinitely. Utilization of these net operating losses could be limited under Section 382 of the Internal Revenue Code and similar state laws based on historical or future ownership changes and the value of the Company’s stock. Additionally, the Company has $ 139.2 million and $ 77.0 million of federal and state research and development credits, respectively, available to offset future taxable income. These federal and state research and development credits begin to expire between 2022 and 2041 and between 2022 and 2036, respectively. The Company also has foreign net operating loss carryforwards of $ 13.6 million, mainly derived from the net operating loss generated by its subsidiary in Brazil, which may be carried forward indefinitely. The Company, or one of its subsidiaries, files income tax returns in the U.S., and various state and foreign jurisdictions. The federal, state and foreign income tax returns are generally subject to tax examinations for the tax years ended December 31, 2018 through December 31, 2021. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service, state or foreign tax authorities to the extent utilized in a future period. The follow table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Balance at beginning of the period $ 48,475 $ 41,753 $ 37,544 Increase related to current year tax positions 5,503 6,722 4,275 Increase related to prior year tax positions — — 109 Decrease related to prior year tax positions ( 163 ) — ( 175 ) Balance at end of the period $ 53,815 $ 48,475 $ 41,753 The balance of total unrecognized tax benefits at December 31, 2021, if recognized, would not affect the effective tax rate on income from continuing operations, due to a full valuation allowance against the Company’s U.S. deferred tax assets. The Company does not expect that the amount of unrecognized tax benefits to change significantly in the next twelve months. The Company’s policy is to recognize interest and/or penalties related to income tax matters in income tax expense. It had no accrual for interest or penalties on its consolidated balance sheets at December 31, 2021 or 2020. No interest and/or penalties were recognized in 2021 or 2020. The Company’s intent is to only make distributions from non-U.S. subsidiaries in the future when they can be made at no net tax cost. Otherwise, the Company considers all of its foreign earnings to be permanently reinvested outside of the U.S. and has no plans to repatriate these foreign earnings to the U.S. The Company has no material unremitted earnings from its non-U.S. subsidiaries. The Tax Cuts and Jobs Act created a new provision that certain income earned by foreign subsidiaries, known as global intangible low-tax income, must be included in the gross income of their U.S. shareholder. The Company has adopted a policy to account for this provision as a period cost. |
LEASES
LEASES | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
LEASES | 19. LEASES The Company has real estate operating leases in Cambridge, Andover and Burlington, Massachusetts, Dublin and Columbus, Ohio, and Durham, NC that provide for scheduled annual rent increases throughout each lease’s term. The Company has also identified leases embedded in certain of its manufacturing and supply agreements as the Company determined that it controls the use of the facilities and related equipment therein. For more information related to manufacturing and supply agreements with Thermo Fisher Scientific, Inc. (“Thermo”) and Catalent, Inc. (“Catalent”), please refer to Note 21, Commitments and Contingencies . As of December 31, 2021 , ROU assets for operating leases were $ 45.5 million and operating lease liabilities were $ 56.6 million. The following table contains a summary of the lease costs recognized and other information pertaining to the Company’s operating leases for the periods indicated: For the Year Ended December 31, 2021 2020 (in thousands) Lease cost Operating lease cost $ 28,737 $ 18,978 Variable lease cost 18,742 17,065 Total lease cost $ 47,479 $ 36,043 Other information Operating lease payments $ 24,449 $ 19,344 Operating lease liabilities arising from obtaining ROU assets 13,225 59,327 Weighted average remaining lease term 3.5 years 4.6 years Weighted average discount rate 7.6 % 7.6 % The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2021: For the Year Ended (in thousands) 2022 $ 18,702 2023 18,326 2024 17,582 2025 9,052 2026 571 Thereafter — Total minimum lease payments 64,233 Less: imputed interest ( 7,672 ) Total operating lease liabilities $ 56,561 Included in the consolidated balance sheet: Current portion of lease liabilities within other current liabilities $ 15,049 Lease liabilities 41,512 Total operating lease liabilities $ 56,561 |
NET LOSS PER SHARE
NET LOSS PER SHARE | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
NET LOSS PER SHARE | 20. NET LOSS PER SHARE Basic net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock outstanding. Diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Year Ended December 31, 2021 2020 2019 (in thousands, except per share amounts) Net loss $ ( 418,780 ) $ ( 554,128 ) $ ( 715,075 ) Weighted-average common shares outstanding - basic 81,262 77,956 73,615 Effect of dilutive securities* — — — Weighted-average common shares outstanding - diluted 81,262 77,956 73,615 Net loss per share — basic and diluted $ ( 5.15 ) $ ( 7.11 ) $ ( 9.71 ) * For the years ended December 31, 2021, 2020 and 2019, stock options, RSAs, RSUs and ESPP to purchase approximately 9.7 million, 9.0 million and 9.1 million shares of common stock, respectively, were excluded from the net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of the 2024 Notes on diluted net earnings per share using the if-converted method as this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted net earnings per share calculation if the effect is more dilutive. During the year ended December 31, 2021, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net earnings per share as of December 31, 2021. |
COMMITMENTS AND CONTINGENCIES
COMMITMENTS AND CONTINGENCIES | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
COMMITMENTS AND CONTINGENCIES | 21. COMMITMENTS AND CONTINGENCIES Manufacturing Obligations The Company has entered into long-term contractual arrangements from time to time for the provision of goods and services. Thermo Fisher Scientific, Inc. The Company entered into a development, commercial manufacturing, and supply agreement in June 2018 and, subsequently, entered into the first and second amendments in May 2019 and July 2020, respectively, with Thermo, formerly Brammer Bio MA, LLC (collectively, the “Thermo Agreements”). Pursuant to the terms of the Thermo Agreements, the Company had access to substantially all of the facility’s eight clean room suites for the Company’s gene therapy programs, subject to certain minimum and maximum volume limitations. The Company determined that the Thermo Agreements contained a lease because the Company had the right to direct the use of the facility and related equipment therein. The lease on four of the eight dedicated clean room suites at Thermo commenced during 2020 and the remaining four commenced during 2021, which is when the dedicated clean room suites became available for use by the Company. In October 2021, the Company executed a third amendment (the “Amendment”) that modified the terms of the Thermo Agreements. The modification significantly decreased the Company’s right of use of the facility’s capacity and significantly reduced the fixed and in-substance fixed payments due over the remaining term of the agreement. The modification was accounted for as a lease termination, resulting in: (i) the derecognition of right of use assets of $ 23.4 million, (ii) the derecognition of lease liabilities of $ 20.1 million, and (iii) the recognition of a loss of $ 3.3 million, which is included in research and development expense. In addition, as a result of the capacity changes associated with the Amendment, $ 21.1 million of accelerated amortization of nonrefundable advance payments made to Thermo that were previously recorded as other assets in the accompanying consolidated balance sheets were charged to research and development expense. Under the Amendment, the Thermo Agreements will expire on December 31, 2028 , or earlier if certain conditions are met. The Company has the ability to extend the term with an 18-months’ notice and an agreement between the two parties. The Company also has the ability to terminate the Thermo Agreements prior to expiration, subject to the payment of additional financial consideration. Further, the Company has committed to guaranteed purchases under the Amendment on a take-or-pay basis regardless of whether services or goods are ordered. As of December 31, 2021, the Company believes it is probable that the guaranteed purchase requirements will be met in the normal course of business throughout the term of the agreement. Catalent, Inc. The Company entered into a manufacturing collaboration agreement and, subsequently, entered into a manufacturing and supply agreement with Catalent, formerly Paragon Biosciences, Inc. in October 2018 and February 2019, respectively (collectively, the “Catalent Agreements”). Pursuant to the terms of the Catalent Agreements, Catalent agreed to provide the Company with two dedicated clean room suites and an option to reserve two additional clean room suites for its gene therapy programs, subject to certain minimum and maximum volume limitations. In September 2019, the Company exercised the option to gain access to the two additional clean room suites. The Catalent Agreements will expire on December 31, 2024 . The Company has the ability to terminate the Catalent Agreements prior to expiration, subject to the payment of additional financial consideration. The Company determined that the Catalent Agreements contained a lease because the Company had the right to direct the use of the facility and related equipment therein. The lease on all four dedicated clean room suites at Catalent commenced during 2020, which is when the dedicated clean room suites became available for use by the Company. In March 2021, the Company modified the terms of the Catalent Agreements. The modification decreased the Company’s right of use of certain dedicated clean room suites and reduced the fixed and in-substance fixed payments due over the remaining term of the agreement. The modification was accounted for as a partial lease termination, resulting in: (i) the derecognition of right of use assets of $ 22.8 million, (ii) the derecognition of lease liabilities of $ 20.0 million, and (iii) the recognition of a loss of $ 2.8 million, which is included in research and development expense. Aldevron, LLC The Company entered into a clinical and commercial supply agreement in December 2018, as subsequently amended in June 2020, with Aldevron LLC (“Aldevron”) for the supply of plasmid DNA to fulfill its needs for gene therapy clinical trials and commercial supply (collectively, the “Aldevron Agreements”). Pursuant to the terms of the Aldevron Agreements, Aldevron agreed to reserve a certain amount of manufacturing capacity on a quarterly basis. In return, the Company is required to make advance payments to Aldevron related to the manufacturing capacity. The term of the Aldevron Agreements will expire on December 31, 2026 . The Company has the option to extend the term of the Aldevron Agreements by one year if the Company delivers a written notice of its intention to extend to Aldevron no later than June 1, 2025. Both parties have the right to early terminate without additional penalty. The Company has determined that the Aldevron Agreements do not contain an embedded lease because it does not convey the right to control the use of Aldevron’s facility or related equipment therein. The following table presents non-cancelable contractual obligations arising from long-term contractual arrangements, including obligations related to leases embedded in certain supply agreements: As of (in thousands) 2022 $ 647,124 2023 178,345 2024 138,725 2025 100,838 2026 54,720 Thereafter 109,440 Total manufacturing commitments $ 1,229,192 Additionally, should the Company obtain regulatory approval for any drug product candidate produced as a part of the Company’s manufacturing obligations above, additional minimum batch requirements with the respective manufacturing parties would be required. Other Funding Commitments The Company has several on-going clinical trials in various clinical trial stages. Its most significant clinical trial expenditures are to contract research organizations (“CROs”). The CRO contracts are generally cancellable at the Company’s option. As of December 31, 2021, the Company has approximately $ 378.5 million in cancellable future commitments based on existing CRO contracts. For the years ended December 31, 2021, 2020 and 2019, the Company recognized approximately $ 47.9 million, $ 40.4 million and $ 31.6 million, respectively, for expenditures incurred by CROs. Litigation In the normal course of business, the Company may from time to time be named as a party to various legal claims, actions and complaints, including matters involving securities, employment, intellectual property, arising from the use of therapeutics utilizing its technology, or others. We record a loss contingency reserve for a legal proceeding when we consider the potential loss probable and we can reasonably estimate the amount of the loss or determine a probable range of loss. We provide disclosure when we consider a loss reasonably possible or when we determine that a loss in excess of a reserve is reasonably possible. We provide an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless we believe that such an estimate cannot be made. The Company has not recorded any material accruals for loss contingencies and in management's opinion no material range of loss is estimable for the matters described below as of December 31, 2021. On September 15, 2020, REGENXBIO INC. (“RegenX”) and the Trustees of the University of Pennsylvania filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC (together, “Sarepta”), in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ‘617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on Sarepta’s alleged direct or indirect manufacture and use of the patented cultured host cell technology allegedly used to make adeno-associated virus (“AAV”) gene therapy products, including SRP-9001. Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes upon the ‘617 Patent asserted by RegenX. Plaintiffs seek injunctive relief, a judgment of infringement and willful infringement, an unspecified amount of damages that is no less than a reasonable royalty (treble damages), attorneys’ fees and costs, and such other relief as the court deems just and proper. On January 4, 2022, the Court denied Sarepta’s motion to dismiss the case pursuant to Federal Rule of Civil Procedure 12(b)(6) based on the Safe Harbor provision of non-infringement contained in 35 U.S.C. § 271(e)(1). Sarepta answered the Complaint on January 18, 2022, and a case schedule has been set with a trial commencing on January 29, 2024. On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserts a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the Patent Trial and Appeal Board at the USPTO (PTAB Case Nos. IPR2021-01134, IPR2021-01135, IPR2021-01136, IPR2021-01137, IPR2021-01138, IPR2021-01139, IPR2021-01140) in which Sarepta is seeking to invalidate certain NS patents concerning exon 53 skipping technology (U.S. Patent Nos. 9,708,361, 10,385,092, 10,407,461, 10,487,106, 10,647,741, 10,662,217, and 10,683,322, respectively, and collectively the “NS Patents”). In addition, NS asserts claims for patent infringement and willful infringement of each of the NS Patents arising from Sarepta’s alleged activities concerning, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further seeks a determination of non-infringement by NS arising from NS’s alleged activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from UWA (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827, collectively the “UWA Patents”). NS is seeking legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to Sarepta’s alleged infringement, and such other relief as the court deems just and proper. NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions. The district court denied NS’ motion for preliminary injunction on September 24, 2021, and the U.S. Court of Appeals for the Federal Circuit reversed and remanded the district court on February 8, 2022. The deadline for the Company to seek rehearing of the Federal Circuit decision is March 10, 2022. In January 2022, the PTAB granted institution of all claims of all NS Patents in response to Sarepta’s IPR Petitions and determined that Sarepta has demonstrated a reasonable likelihood of success in proving that the NS Patents are unpatentable. On December 27, 2021, the district court partially granted and denied the motion to dismiss by Sarepta and ordered NS to file a Second Amended Complaint (“SAC”), which it did on January 14, 2022. In the SAC, NS maintains all claims of the original complaint of July 13, 2021, except a determination of non-infringement of the UWA Patents. On January 28, 2022, Sarepta filed its answer to the SAC, with defenses and counterclaims against NS and NS Pharma Inc. that include infringement of the UWA Patents arising from their alleged activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and breach of contract. Sarepta is also seeking a determination of invalidity of the NS Patents. Sarepta is seeking an award of relief in its defenses to NS’ allegations, a judgment of breach of contract, a determination of invalidity of the NS Patents, a judgment of infringement and willful infringement of the UWA Patents, legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to NS’ alleged infringement, and such other relief as the court deems just and proper. Case scheduling in district court, including a trial date, will follow. |
SUMMARY OF SIGNIFICANT ACCOUN_2
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the U.S. (“U.S. GAAP”), reflect the accounts of Sarepta Therapeutics, Inc. and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates in one segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. The Company’s CEO, as the chief operating decision-maker, manages and allocates resources to the operations of the Company on a total company basis. The Company’s research and development organization is responsible for the research and discovery of new product candidates and supports development and registration efforts for potential future products. The Company’s supply chain organization manages the development of the manufacturing processes, clinical trial supply and commercial product supply. The Company’s commercial organization is responsible for commercialization of EXONDYS 51, VYONDYS 53 and AMONDYS 45 in the U.S. and internationally. The Company is supported by other back-office general and administration functions. Consistent with this decision-making process, the Company’s CEO uses consolidated, single-segment financial information for purposes of evaluating performance, forecasting future period financial results, allocating resources and setting incentive targets. In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. |
Estimates and Uncertainties | Estimates and Uncertainties The preparation of the consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates. |
Fair Value Measurements | Fair Value Measurements The Company has certain financial assets and liabilities that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements: • Level 1—quoted prices for identical instruments in active markets; • Level 2—quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and • Level 3—valuations derived from valuation techniques in which one or more significant value drivers are unobservable. The fair value of the majority of the Company’s financial assets is categorized as Level 1 within the fair value hierarchy. These assets include money market funds, and publicly traded debt and equity securities. For additional information related to fair value measurements, please read Note 5, Fair Value Measurements to the consolidated financial statements. |
Cash and Cash Equivalents | Cash Equivalents Only investments that are highly liquid and readily convertible to cash and have original maturities of three months or less are considered cash equivalents. |
Investments | Investments Available-For-Sale Debt Securities Available-for-sale debt securities are recorded at fair value and unrealized gains and losses are included in accumulated other comprehensive income in stockholder’s equity. Interest income and realized gains and losses are reported in other expense, net, on a specific identification basis. Equity Investments The Company’s equity investments include its investments in a publicly traded biotechnology company and several privately held biotechnology companies and are included in other non-current assets in the Company’s consolidated balance sheets. The equity investment in the publicly traded biotechnology company has a readily determinable fair value and is carried at fair value. The equity investments in the privately held biotechnology companies do not have readily determinable fair values and are measured at cost less any impairment, plus or minus changes resulting from observable price changes for the identical or a similar investment of the same issuer. Any change in the valuation of equity investments is recorded as a gain or loss on the Company’s consolidated statements of operations and comprehensive loss. |
Accounts Receivable | Accounts Receivable The Company’s accounts receivable primarily arise from product sales. They are generally stated at the invoiced amount and do not bear interest. Revenues from product sales are recorded at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from Medicaid rebates, governmental chargebacks including Public Health Services (“PHS”) chargebacks, prompt pay discounts, co-pay assistance and distribution fees. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of the Company) for PHS chargebacks, prompt pay discounts and certain distribution fees, or a current liability (if a payment is required of the Company) for Medicaid rebates, co-pay assistance and certain distribution fees. The accounts receivable from product sales represents receivables due from the Company’s specialty distributor and specialty pharmacies in the U.S. as well as certain distributors in the European Union (“EU”) , Brazil, Israel and the Middle East. The Company has had no historical write-offs of its accounts receivable and its payment terms range from 60 to 91 days for sales within the U.S. and 45 and 150 days for the majority of product sales outside the U.S. The Company monitors the financial performance and creditworthiness of its customers so that it can properly assess and respond to changes in the customers’ credit profiles or any specific issues. The Company provides reserves against trade receivables for expected credit losses that may result from a customer’s inability to pay. Amounts determined to be uncollectible are written-off against the established reserve. As of December 31, 2021 , the credit profiles for the Company’s customers are deemed to be in good standing and an allowance for credit losses is not considered necessary. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist of accounts receivable from customers and cash. Three individual customers accounted for 48 %, 39 % and 10 % o f net product revenues for the year ended December 31, 2021 , 47 %, 39 % and 11 % for the year ended December 31, 2020 , and 43 %, 41 % and 13 % for the year ended December 31, 2019. Three individual customers accounted for 41 %, 41 % and 10 % of accounts receivable from product sales for the year ended December 31, 2021 and 45 %, 41 % and 9 % for the year ended December 31, 2020. As of December 31, 2021, the Company believes that such customers are of high credit quality. As of December 31, 2021, the Company’s cash was concentrated at three financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. |
Inventories | Inventories Inventories are stated at the lower of cost and net realizable value with cost determined on a first-in, first-out basis. The Company capitalizes inventory costs associated with products following regulatory approval when future commercialization is considered probable and the future economic benefit is expected to be realized. EXONDYS 51, VYONDYS 53 and AMONDYS 45 inventory used in clinical development programs is charged to research and development expense when the product enters the research and development process and no longer can be used for commercial purposes. The Company periodically analyzes its inventories for excess amounts or obsolescence and writes down obsolete or otherwise unmarketable inventory to its estimated net realizable value. Additionally, though the Company’s products are subject to strict quality control and monitoring which the Company performs throughout the manufacturing processes, certain batches or units of product may not meet quality specifications. Expense incurred related to excess inventory, obsolete inventory, or inventories that do not meet the Company's quality specifications are recorded as a component of cost of sales in the Company's consolidated statements of operations and comprehensive loss. For products which are under development and have not yet been approved by regulatory authorities, purchased drug product is charged to research and development expense upon delivery. Delivery occurs when the inventory passes quality inspection and ownership transfers to the Company. Nonrefundable advance payments for research and development activities, including production of purchased drug product, are deferred and capitalized until the goods are delivered. If the Company does not expect the goods to be delivered or services to be rendered, the advanced payment capitalized will be charged to expense. |
Property and Equipment | Property and Equipment Property and equipment are initially recorded at cost, including the acquisition cost and all costs necessarily incurred to bring the asset to the location and working condition necessary for their intended use. The cost of normal, recurring or periodic repairs and maintenance activities related to property and equipment are expensed as incurred. The cost for planned major maintenance activities, including the related acquisition or construction of assets, is capitalized if the repair will result in future economic benefits. Interest costs incurred during the construction period of major capital projects are periodically reviewed, and if determined to be material, capitalized until the asset is ready for its intended use, at which point the interest costs are amortized as depreciation expense over the life of the underlying asset. The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab and manufacturing equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in progress Not depreciated until put into service |
Intangible assets | Intangible assets The Company’s intangible assets consist of in-licensed rights, patent costs and software licenses, which are stated in the Company’s consolidated balance sheets, net of accumulated amortization and impairments, if applicable. The in-licensed rights primarily relate to agreements with BioMarin Pharmaceutical, Inc. (“BioMarin”) and the University of Western Australia (“UWA”). The in-licensed rights are being amortized on a straight-line basis over the remaining life of the related patents because the life of the related patents reflects the expected time period that the Company will benefit from the in-licensed rights. Patent costs consist primarily of external legal costs, filing fees incurred to file patent applications and renewal fees on proprietary technology developed or licensed by the Company. Patent costs associated with applying for a patent, being issued a patent and annual renewal fees are capitalized. Costs to defend a patent and costs to invalidate a competitor’s patent or patent application are expensed as incurred. Patent costs are amortized on a straight-line basis over the shorter of the estimated economic lives or the initial term of the patents, which is generally 20 years. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets Long-lived assets held and used by the Company, intangible assets with definite lives and right of use (“ROU”) assets are reviewed for impairment whenever events or circumstances indicate that the carrying amount of assets may not be recoverable. The Company evaluates recoverability of assets to be held and used by comparing the carrying amount of an asset to future net undiscounted cash flows to be generated by the asset. If the asset is considered to be impaired, the impairment to be recognized is measured as the amount by which the carrying amount of the assets exceeds the fair value of the assets. Such reviews assess the fair value of the assets based upon estimates of future cash flows that the assets are expected to generate. |
Convertible Debt | Convertible Debt As a result of adopting ASU 2020-06, “ Debt – Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging – Contracts in Entity’s Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity’s Own Equity ”, the Company accounts for the liability and equity components of convertible debt instruments that can be settled in cash as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives under ASC Topic 815, Derivatives and Hedging (“ASC 815”). Simultaneously with the issuance of the 2024 Notes (defined below) in November 2017, the Company bought capped call options from certain counterparties to minimize the impact of potential dilution upon conversion. The premium for the capped call options was recorded as additional paid-in capital. For additional information related to the convertible debt transactions, please read Note 13, Indebtedness to the consolidated financial statements. |
Revenue Recognition | Revenue Recognition The Company recognizes revenue when a customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for the goods or services provided. To determine revenue recognition for arrangements within the scope of ASC Topic 606, Revenue from Contracts with Customers (“ASC 606”), the Company performs the following five steps: (1) identify the contract with the customer; (2) identify the performance obligations in the contract; (3) determine the transaction price; (4) allocate the transaction price to the performance obligations in the contract; and (5) recognize revenue when or as the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the entity will collect the consideration it is entitled to in exchange for the goods or services it transfers or provides to the customer. At contract inception, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when or as the performance obligation is satisfied. The only performance obligation in the Company’s contracts with customers is to timely deliver drug products to the customer’s designated location. Product revenues The Company distributes its products principally through its customers. The customers subsequently resell the products to patients and health care providers. The Company provides no right of return to the customers except in cases of shipping error or product defect. Product revenues are recognized when the customers take control of the products, which typically occurs upon delivery to the customers. For the years ended December 31, 2021, 2020 and 2019, the majority of the product revenues recognized were generated by the specialty distributor and specialty pharmacies in the U.S. Variable Consideration Product revenues are recorded at the net sales price (transaction price) which includes estimated reserves for variable consideration, such as Medicaid rebates, governmental chargebacks, including PHS chargebacks, prompt payment discounts, co-pay assistance and distribution fees. These reserves reflect the Company’s best estimates of the amount of consideration to which it is entitled based on the terms of the contracts. Additional details relating to variable consideration follows: • Medicaid rebates relate to the Company’s estimated obligations to states under established reimbursement arrangements. Medicaid rebate reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Governmental chargebacks, including PHS chargebacks, relate to the Company’s estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices that the Company charges to wholesalers. The wholesaler charges the Company for the difference between what the wholesaler pays for the products and the ultimate selling price to the qualified healthcare providers. Chargeback reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. Chargeback amounts are generally determined at the time of resale to the qualified healthcare provider from the wholesaler, and the Company generally issues credits for such amounts within a few weeks of receiving notification of resale from the wholesaler. • Prompt payment discounts relate to the Company’s estimated obligations for credits to be granted to specialty pharmacies for remitting payment on their purchases within established incentive periods. Reserves for prompt payment discounts are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and accounts receivable. • Co-pay assistance relates to financial assistance provided to qualified patients, whereby the Company may assist them with prescription drug co-payments required by the patient’s insurance provider. Reserves for co-pay assistance are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue and the establishment of a liability which is included in accrued expenses. • Distribution fees relate to fees paid to customers in the distribution channel that provide the Company with inventory management, data and distribution services and are generally accounted for as a reduction of revenue. To the extent that the services received are distinct from the Company’s sale of products to the customers, these payments are accounted for as selling, general and administrative expenses. Reserves for distribution fees result in an increase in a liability if payments are required of the Company or a reduction of accounts receivable if no payments are required of the Company. Collaboration revenue The Company’s collaboration revenue is primarily generated from its collaboration arrangement with F. Hoffman-La Roche Ltd. (“Roche”). For more information, please read Note 3, Collaboration and License Agreements . At the inception of a collaboration arrangement, the Company first assesses whether the contractual arrangement is within the scope of ASC Topic 808, Collaborative Arrangements (“ASC 808”) to determine whether the arrangement involves a joint operating activity and involves two (or more) parties that are both active participants in the activity and exposed to significant risks and rewards dependent on the commercial success of such activity. Then the Company determines whether the collaboration arrangement in its entirety represents a contract with a customer as defined by ASC 606. If only a portion of the collaboration arrangement is potentially with a customer, the Company applies the distinct good or service unit-of-account guidance in ASC 606 to determine whether there is a unit of account that should be accounted for under ASC 606. For the units of account in the collaboration arrangement that do not represent a vendor-customer relationship, the Company will (i) consider applying other GAAP, including by analogy, or (ii) if there is no appropriate analogy, consistently apply a reasonable and rational accounting policy election. In general, by analogy to ASC 606, the Company identifies the performance obligations within the collaboration arrangement and identifies and allocates the transaction price the Company expects to receive on a relative standalone selling price basis to each performance obligation. Variable consideration, consisting of development and regulatory milestones, will be included in the transaction price only if the Company expects to receive such consideration and if it is probable that the inclusion of the variable consideration will not result in a significant reversal in the cumulative amount of revenue recognized under the arrangement. Sales-based royalty and milestone payments are excluded from the transaction price the Company expects to receive until the underlying sales occur because the license to the Company’s intellectual property is deemed to be the predominant item to which the royalties or milestones relate as it is the primary driver of value in its collaboration arrangement. For the recognition of revenue associated with each performance obligation, if the Company determines ASC 606 is not appropriate to apply by analogy, the Company will apply a reasonable, rational and consistently applied accounting policy election to faithfully depict the transfer of services to the collaboration partner over the estimated performance period. Up-front payments from a collaboration partner are recognized as deferred revenue when received and recognized as revenue over the estimated performance period. Reimbursement payments from a collaboration partner associated with cost sharing provisions in a collaboration arrangement are recognized as the related expense is incurred and classified as an offset to operating expenses. |
Valuation of Product Options | Valuation of Product Options The Company's collaboration arrangements may contain options which provide the collaboration partner with the right to obtain additional licenses. If an arrangement contains product options, by analogy to ASC 606, the Company evaluates the product options to determine whether they represent material rights, which may include options to acquire additional goods or services for free or at a discount. If the customer options are determined to represent material rights, they are recognized as a separate performance obligation at inception of the arrangement. The Company allocates a portion of the transaction price of the collaboration arrangement to material rights based on the relative standalone selling price. Amounts allocated to material rights are not recognized as revenue until related options are exercised or expire. Key assumptions to determine the standalone selling price of product options in a collaboration arrangement include, but are not limited to, forecasted revenues, development timelines, incremental costs related to the arrangement, discount rates and likelihood of technical and regulatory success. |
Research and Development | Research and Development Research and development expenses consist of costs associated with research activities as well as those with the Company’s product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Research and development expenses are expensed as incurred. Up-front fees and milestones paid to third parties in connection with technologies which have not reached technological feasibility and do not have an alternative future use are expensed when incurred. Direct research and development expenses associated with the Company’s programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants and other external services, such as data management and statistical analysis support and materials and supplies used in support of clinical programs. Indirect costs of the Company’s clinical programs include salaries, stock-based compensation and an allocation of its facility and technology costs. When third-party service providers’ billing terms do not coincide with the Company’s period-end, the Company is required to make estimates of its obligations to those third parties, including clinical trial and pharmaceutical development costs, contractual services costs and costs for supply of its drug candidates, incurred in a given accounting period and record accruals at the end of the period. The Company bases its estimates on its knowledge of the research and development programs, services performed for the period, past history for related activities and the expected duration of the third-party service contract, where applicable. |
Stock-Based Compensation | Stock-Based Compensatio n The Company’s stock-based compensation programs include stock options, restricted stock awards (“RSAs”), restricted stock units (“RSUs”) and an employee stock purchase program (“ESPP”). The Company accounts for stock-based compensation using the fair value method. The fair value of stock options are estimated on the date of grant using the Black-Scholes-Merton option-pricing model. The fair values of RSAs and RSUs are based on the fair market value of the Company’s common stock on the date of the grant. The fair value of stock awards, with consideration given to estimated forfeitures, is recognized as stock-based compensation expense on a straight-line basis over the vesting period of the grants. For stock awards with performance-vesting conditions, the Company does not recognize compensation expense until it is probable that the performance-vesting condition will be achieved. Additionally, the Company granted its CEO options with service and market conditions. A market condition relates to the achievement of a specified price of the Company’s common stock, a specified amount of intrinsic value indexed to the Company’s common stock or a specified price of the Company’s common stock in terms of other similar equity shares. The grant date fair value for the options with service and market conditions is determined by a lattice model with Monte Carlo simulations and is recognized as stock-based compensation expense on a straight-line basis over the service period. Under the Company’s ESPP, participating employees purchase common stock through payroll deductions. The purchase price is equal to 85 % of the lower of the closing price of the Company’s common stock on the first business day and the last business day of the relevant purchase period. The fair value of stock purchase rights is estimated using the Black-Scholes-Merton option-pricing model. The fair value of the look-back provision with the 15 % discount is recognized on a graded-vesting basis as stock-based compensation expense over the purchase period. |
Income Taxes | Income Taxes The Company follows the asset and liability method of accounting for income taxes, which requires the recognition of deferred tax assets and liabilities for expected future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases. Deferred tax assets and liabilities are measured using enacted tax rates in effect for the year in which the differences are expected to reverse. A valuation allowance is recorded to reduce the net deferred tax asset to zero when it is more likely than not that the net deferred tax asset will not be realized. The Company recognizes the effect of income tax positions only if those positions are more likely than not of being sustained upon an examination. The amount of the benefit that may be recognized in the financial statements is the largest amount that has a greater than 50% likelihood of being realized. The Company recognizes interest and penalties related to uncertain tax positions within income tax expense. It is the intention of the Company to reinvest the earnings of its non-U.S. subsidiaries in those operations and not to repatriate the earnings to the U.S. Accordingly, the Company does not provide for deferred taxes on the excess of the financial reporting over the tax basis in its investments in foreign subsidiaries as they are considered permanent in duration. |
Leases | Leases At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances present in the arrangement. Leases with a term greater than 12 months are recognized on the consolidated balance sheets as right-of-use (“ROU”) assets and short-term and long-term lease liabilities, as applicable. The Company has elected not to recognize leases with terms of 12 months or less on the consolidated balance sheets. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew. The Company monitors its plans to renew its leases no less than on a quarterly basis. In addition, the Company’s lease agreements generally do not contain any residual value guarantees or restrictive covenants. Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term at lease commencement. The initial measurement of the lease liability is determined based on the future lease payments, which may include lease payments that depend on an index or a rate (such as the consumer price index or other market index). The Company initially measures payments based on an index or rate by using the applicable rate at lease commencement and subsequent changes in such rates are recognized as variable lease costs. Variable payments that do not depend on a rate or index are not included in the lease liability and are recognized as they are incurred. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense with unrecognized variable lease payments recognized as incurred. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment. Components of a lease are bifurcated between lease components and non-lease components. The fixed and in-substance fixed contract consideration identified is then allocated based on the relative standalone price to the lease and non-lease components. However, ASC Topic 842, Leases, provides entities with a practical expedient that allows an accounting policy election to not separate lease and non-lease components by class of underlying asset. In using this expedient, entities would account for each lease component and the related non-lease component together as a single component. For new and amended real estate leases beginning after January 1, 2019, the Company elected to account for the lease and non-lease components together for existing classes of underlying assets and allocates the contract consideration to the lease component only. In contrast, the Company does not apply the practical expedient for leases embedded in manufacturing and supply agreements with certain of its contract manufacturing organizations and has instead allocated contract consideration between the lease and non-lease components based on their relative standalone price. |
Embedded Derivatives | Embedded Derivatives The Company evaluates certain of its financial and business development transactions to determine if embedded components of these contracts meet the definition of derivative under ASC 815. In general, embedded derivatives are required to be bifurcated from the host instrument if (i) the embedded feature is not clearly and closely related to the host contract and (ii) the embedded feature, if considered a freestanding instrument, meets the definition of a derivative. The embedded derivative is reported on the consolidated balance sheets at its fair value. Any change in fair value, as determined at each measurement period, is recorded as a component of the consolidated statements of operations and comprehensive loss. |
Contingent Consideration | Contingent Consideration Certain of the Company’s license and collaboration agreements include future payments that are contingent upon the receipt, or receipt and subsequent sale, of a Priority Review Voucher (“PRV”) . The Company has concluded that these contingent payments represent embedded derivatives. The Company records a liability for such contingent payments at fair value on the date the agreements are effective. The Company estimates the fair value of contingent consideration derivatives through a valuation model that includes an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. Changes in the fair value of the contingent consideration derivatives can result from changes to one or multiple assumptions, including adjustments to the discount rates, the assumed development timeline and the probability of achievement of certain regulatory milestones. The Company revalues its contingent consideration derivatives upon a material change to one or more of the assumptions discussed above. Changes in the fair value of the Company’s contingent consideration derivatives are recognized in the Company’s consolidated statements of operations and comprehensive loss. Such changes are classified as other income (loss) which corresponds to the classification of any gain recognized upon the actual sale of a PRV. |
Commitments and Contingencies | Commitments and Contingencies The Company records liabilities for legal and other contingencies when information available to the Company indicates that it is probable that a liability has been incurred and the amount of loss can be reasonably estimated. Legal costs in connection with legal and other contingencies are expensed as costs are incurred. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements Recently adopted In August 2020, the Financial Accounting Standards Board (“FASB”) issued ASU 2020-06, “ Debt - Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging - Contracts in Entity's Own Equity (Subtopic 815-40): Accounting for Convertible Instruments and Contracts in an Entity's Own Equity .” This ASU simplifies the complexity associated with applying U.S. GAAP for certain financial instruments with characteristics of liabilities and equity. More specifically, the amendments focus on the guidance for convertible instruments and derivative scope exceptions for contracts in an entity’s own equity. Under ASU 2020-06, the embedded conversion features are no longer separated from the host contract for convertible instruments with conversion features that are not required to be accounted for as derivatives under ASC 815, or that do not result in substantial premiums accounted for as paid-in capital. Consequently, a convertible debt instrument, such as the Company’s senior notes due on November 15, 2024 (the “2024 Notes”), will be accounted for as a single liability measured at its amortized cost, as long as no other features require bifurcation and recognition as derivatives. The new guidance also requires the if-converted method to be applied for all convertible instruments and requires additional disclosures. This guidance is required to be adopted by January 1, 2022, and early adoption is permitted, but no earlier than fiscal years beginning after December 15, 2020. The Company elected to early adopt this guidance on January 1, 2021, using the modified retrospective method. Under this transition method, the cumulative effect of the accounting change removed the impact of recognizing the equity component of the Company’s convertible notes (at issuance and the subsequent accounting impact of additional interest expense from debt discount amortization). The cumulative effect of the accounting change as of January 1, 2021 increased the carrying amount of the convertible notes by $ 96.8 million, reduced accumulated deficit by $ 60.2 million and reduced additional paid-in capital by $ 157.0 million. Interest expense of the 2024 Notes will be lower as a result of adoption of this guidance. The if-converted method for such instruments will be used to compute diluted net earnings per share if and when profitability is achieved. As a result of the adoption of this guida nce, interest expense and net loss was reduced by $ 22.4 million, or $ 0.28 per share, for the year ended December 31, 2021. In December 2019, the FASB issued ASU 2019-12, “ Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes”, which is intended to simplify the accounting for income taxes. This ASU removes certain exceptions to the general principles in Topic 740 and clarifies and amends existing guidance to improve consistent application. The new guidance was effective beginning January 1, 2021. The adoption of this guidance did not have a material effect on the Company’s consolidated financial statements. |
SUMMARY OF SIGNIFICANT ACCOUN_3
SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Estimated Useful Lives of Property and Equipment | The Company generally depreciates the cost of its property and equipment using the straight-line method over the estimated useful lives of the respective assets, which are summarized as follows: Asset Category Useful lives Lab and manufacturing equipment 5 years Office equipment 5 years Software and computer equipment 3 - 5 years Furniture and fixtures 7 years Leasehold improvements Lesser of the useful life or the term of Land improvements 25 years Land Not depreciated Building and improvements 30 years Construction in progress Not depreciated until put into service |
FAIR VALUE MEASUREMENTS (Tables
FAIR VALUE MEASUREMENTS (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Assets and Liabilities Measured and Carried at Fair Value | The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value: Fair Value Measurement as of December 31, 2021 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 1,562,358 $ 1,562,358 $ — $ — Strategic equity investments 34,892 2,480 — 32,412 Certificates of deposit 250 250 — — Total assets $ 1,597,500 $ 1,565,088 $ — $ 32,412 Liabilities Contingent consideration $ 43,600 $ — $ — $ 43,600 Total liabilities $ 43,600 $ — $ — $ 43,600 Fair Value Measurement as of December 31, 2020 Total Level 1 Level 2 Level 3 (in thousands) Assets Money market funds $ 629,440 $ 629,440 $ — $ — Government and government agency 1,037,981 1,037,981 — — Strategic equity investments 38,799 3,699 — 35,100 Certificates of deposit 250 250 — — Total assets $ 1,706,470 $ 1,671,370 $ — $ 35,100 Liabilities Contingent consideration $ 50,800 $ — $ — $ 50,800 Total liabilities $ 50,800 $ — $ — $ 50,800 |
Summary of Fair Value of Level 3 Financial Assets | The following table represents a roll-forward of the fair value of Level 3 financial assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Fair value, beginning of year $ 35,100 $ 30,000 Additions 1,800 5,100 Changes in estimated fair value ( 4,488 ) — Fair value, end of year $ 32,412 $ 35,100 |
Summary of Fair Value of Level 3 Financial Liabilities | The following table represents a roll-forward of the fair value of Level 3 financial liabilities for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Fair value, beginning of year $ 50,800 $ 5,200 Additions — 600 Changes in estimated fair value, net ( 7,200 ) 45,000 Fair value, end of year $ 43,600 $ 50,800 |
CASH, CASH EQUIVALENTS AND MA_2
CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Cash and Cash Equivalents [Abstract] | |
Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents | The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the consolidated balance sheets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Money market funds $ 1,562,358 $ 629,440 Government and government agency bonds — 602,058 Total $ 1,562,358 $ 1,231,498 |
Summary of Company Cash, Cash Equivalents and Investments | The following tables summarize the Company’s cash, cash equivalents and investments for each of the periods indicated: As of December 31, 2021 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As reported: Cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 Total cash and cash equivalents $ 2,115,869 $ — $ — $ 2,115,869 As of December 31, 2020 Amortized Gross Gross Fair (in thousands) Cash and money market funds $ 900,590 $ — $ — $ 900,590 Government and government agency bonds 1,037,959 22 — 1,037,981 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 As reported: Cash and cash equivalents $ 1,502,639 $ 9 $ — $ 1,502,648 Short-term investments 435,910 13 — 435,923 Total cash, cash equivalents and investments $ 1,938,549 $ 22 $ — $ 1,938,571 |
PRODUCT REVENUES, NET, ACCOUN_2
PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE AND RESERVES FOR PRODUCT REVENUES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Receivables, Net, Current [Abstract] | |
Schedule of product revenues | The following table summarizes the Company's product revenues, net disaggregated by product for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) EXONDYS 51 $ 454,361 $ 422,007 $ 380,718 VYONDYS 53 89,511 33,858 115 AMONDYS 45 68,529 — — Products, net $ 612,401 $ 455,865 $ 380,833 |
Summary of Components of Accounts Receivable | The following table summarizes the components of the Company’s accounts receivable for the periods indicated: As of December 31, 2021 2020 (in thousands) Product sales receivable, net of discounts and allowances $ 152,990 $ 100,870 Government contract receivables — 470 Total accounts receivable, net $ 152,990 $ 101,340 |
Summary of Change in Reserves for Discounts and Allowances | The following table summarizes an analysis of the change in reserves for discounts and allowances for the periods indicated: Chargebacks Rebates Prompt Pay Other Accruals Total (in thousands) Balance, as of December 31, 2019 $ 588 $ 44,738 $ 1,506 $ 4,671 $ 51,503 Provision 9,700 52,180 6,384 10,175 78,439 Payments/credits ( 8,007 ) ( 55,147 ) ( 5,941 ) ( 9,877 ) ( 78,972 ) Balance, as of December 31, 2020 $ 2,281 $ 41,771 $ 1,949 $ 4,969 $ 50,970 Provision 13,308 78,637 9,400 16,107 117,452 Payments/credits ( 14,790 ) ( 59,902 ) ( 8,551 ) ( 14,713 ) ( 97,956 ) Balance, as of December 31, 2021 $ 799 $ 60,506 $ 2,798 $ 6,363 $ 70,466 |
Summary of Total Reserves Included in Consolidated Balance Sheets | The following table summarizes the total reserves above included in the Company’s consolidated balance sheets for the periods indicated: As of December 31, 2021 2020 (in thousands) Reduction to accounts receivable $ 8,321 $ 8,352 Component of accrued expenses 62,145 42,618 Total reserves $ 70,466 $ 50,970 |
INVENTORY (Tables)
INVENTORY (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Inventory Disclosure [Abstract] | |
Summary of Components of Inventory | The following table summarizes the components of the Company’s inventory for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Raw materials $ 58,822 $ 71,717 Work in progress 230,194 139,704 Finished goods 26,717 20,540 Total inventory $ 315,733 $ 231,961 |
SummaryOfInventoryBalanceSheetClassificationTableTextBlock | The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Balance sheet classification Inventory $ 186,212 $ 231,961 Other non-current assets 129,521 — Total inventory $ 315,733 $ 231,961 |
OTHER ASSETS (Tables)
OTHER ASSETS (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Deferred Costs, Capitalized, Prepaid, and Other Assets Disclosure [Abstract] | |
Summary of Other Current Assets | The following table summarizes the Company’s other current assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Manufacturing-related deposits and prepaids $ 93,656 $ 134,430 Collaboration receivable 18,647 34,184 Prepaid clinical and pre-clinical expenses 12,667 16,224 Prepaid maintenance services 8,452 6,411 Prepaid insurance 5,282 4,158 Prepaid research expenses 3,082 5,854 Prepaid income tax 1,100 4,939 Leasehold improvement receivable — 3,059 Other 6,142 4,065 Total other current assets $ 149,028 $ 213,324 |
Summary of Other Non-current Assets | The following table summarizes the Company’s other non-current assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Non-current inventory $ 129,521 $ — Manufacturing-related deposits and prepaids 112,765 148,525 Strategic investments 34,892 38,799 Restricted cash and investments 9,904 9,315 Prepaid clinical expenses 2,007 3,395 Other 3,860 3,669 Total other non-current assets $ 292,949 $ 203,703 |
PROPERTY AND EQUIPMENT, NET (Ta
PROPERTY AND EQUIPMENT, NET (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
Summarizes Components of Property and Equipment, Net | Property and equipment are recorded at historical cost, net of accumulated depreciation. The following table summarizes components of property and equipment, net, for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Leasehold improvements $ 103,370 $ 55,019 Lab and manufacturing equipment 64,613 49,571 Building and improvements 47,605 39,397 Software and computer equipment 42,506 33,948 Furniture and fixtures 9,242 7,010 Land 5,183 5,183 Land improvements 4,921 3,610 Office equipment 1,189 1,178 Construction in progress 25,159 71,541 Property and equipment, gross 303,788 266,457 Less: accumulated depreciation ( 112,632 ) ( 76,027 ) Property and equipment, net $ 191,156 $ 190,430 |
INTANGIBLE ASSETS, NET (Tables)
INTANGIBLE ASSETS, NET (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Summary of Components of Intangible Assets | The following table summarizes the components of the Company’s intangible assets for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Patents $ 12,382 $ 11,210 In-licensed rights 8,573 8,073 Software licenses 299 1,554 Intangible assets, gross 21,254 20,837 Less: accumulated amortization ( 7,015 ) ( 7,209 ) Intangible assets, net $ 14,239 $ 13,628 |
Summary of Estimated Future Amortization for Intangible Assets | The following table summarizes the estimated future amortization for intangible assets: As of (in thousands) 2022 $ 1,395 2023 1,394 2024 1,391 2025 1,330 2026 1,200 Thereafter 7,529 Total $ 14,239 |
ACCRUED EXPENSES (Tables)
ACCRUED EXPENSES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Summary of Accrued Expenses | The following table summarizes the Company’s accrued expenses for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Accrued contract manufacturing costs $ 104,311 $ 36,543 Product revenue related reserves 62,145 42,618 Accrued employee compensation costs 48,299 50,803 Accrued clinical and pre-clinical costs 25,955 22,169 Accrued royalties 11,965 7,793 Accrued professional fees 9,381 10,221 Accrued collaboration cost-sharing 2,887 3,516 Accrued interest expense 1,045 1,045 Accrued milestone and license expense 100 9,380 Other 5,609 9,465 Total accrued expenses $ 271,697 $ 193,553 |
INDEBTEDNESS (Tables)
INDEBTEDNESS (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Debt Disclosure [Abstract] | |
Summary of Debt Facilities | The following table summarizes the Company’s debt facilities for the periods indicated: As of December 31, 2021 2020 (in thousands) Principal amount of the 2024 Notes $ 569,993 $ 569,993 Unamortized discount - equity component — ( 98,721 ) Unamortized discount - debt issuance costs ( 6,320 ) ( 6,510 ) Net carrying value of 2024 Notes 563,673 464,762 Principal amount of the 2019 Term Loan 550,000 550,000 Unamortized discounts ( 16,797 ) ( 22,269 ) Net carrying value of 2019 Term Loan 533,203 527,731 Total carrying value of debt facilities $ 1,096,876 $ 992,493 Fair value of 2024 Notes $ 846,138 $ 1,394,249 Fair value of 2019 Term Loan 576,085 550,000 Total fair value of debt facilities $ 1,422,223 $ 1,944,249 |
Summarizes Total Gross Payments Due under Company's Debt Arrangements | The following table summarizes the total gross payments due under the Company’s debt arrangements: As of (in thousands) 2022 $ — 2023 250,000 2024 869,993 2025 — 2026 — Thereafter — Total payments $ 1,119,993 The aggregate annual maturities of long-term debt and interest during the years ending December 31, 2022, 2023 and 2024 are $ 55.9 million, $ 305.3 million and $ 903.4 million, respectively. |
STOCK-BASED COMPENSATION (Table
STOCK-BASED COMPENSATION (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Summary of Stock Option Activity | The following tables summarize the Company’s stock option activity for each of the periods indicated: For the Year Ended December 31, 2021 Weighted Average Exercise Shares Price Grants outstanding at beginning of 7,844,947 $ 70.61 Granted 1,685,860 86.41 Exercised ( 283,622 ) 45.71 Cancelled and forfeited ( 1,050,264 ) 112.18 Grants outstanding at end of the period 8,196,921 $ 69.39 Grants exercisable at end of the period 2,532,438 $ 84.71 Grants vested and expected to vest at 7,867,722 $ 68.17 The weighted-average grant date fair value per share of stock options granted during the years ended December 31, 2021, 2020 and 2019 was $48.16, $ 61.38 and $ 70.93 , respectively. Weighted Aggregate Average Intrinsic Remaining Value Contractual (in thousands) Life (Years) Options outstanding at December 31, 2021 $ 248,479 6.4 Options exercisable at December 31, 2021 $ 59,069 5.6 Options vested and expected to vest at December 31, 2021 $ 247,434 6.3 |
Summary of Company's Stock Options Vested and Exercised | The following table summarizes the Company’s stock options vested and exercised for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Aggregate grant date fair value of stock options $ 79,068 $ 80,355 $ 50,878 Aggregate intrinsic value of stock options $ 10,622 $ 144,750 $ 109,707 |
Summary of Employee Stock Purchase Plan Activity and Expense | The following table summarizes the Company’s ESPP activity for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 Number of shares purchased 111,171 102,031 92,086 Proceeds received (in millions) $ 7.8 $ 7.5 $ 5.1 |
Summary of Stock-Based Compensation Expense by Function Included within Consolidated Statements of Operations and Comprehensive Loss | The following table summarizes stock-based compensation expense by function included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2021 2020 2019 (in thousands) Research and development $ 50,526 $ 41,671 $ 27,681 Selling, general and administrative 63,417 66,399 50,921 Total stock-based compensation $ 113,943 $ 108,070 $ 78,602 |
Summary of Stock-Based Compensation Expense by Grant Type Included within Consolidated Statements of Operations and Comprehensive Loss | The following table summarizes stock-based compensation expense by grant type included within the consolidated statements of operations and comprehensive loss: For the Year Ended December 31, 2021 2020 2019 (in thousands) Stock options $ 68,995 $ 68,832 $ 53,427 Restricted stock awards/units 40,055 33,457 20,103 Employee stock purchase plan 4,893 5,781 5,072 Total stock-based compensation $ 113,943 $ 108,070 $ 78,602 |
Restricted Stock Awards (RSAs) [Member] | |
Summary of Restricted Stock Award and Restricted Stock Units Activity | The following table summarizes the Company’s RSA activity for each of the periods indicated: For the Year Ended December 31, 2021 Weighted Average Grant Date Shares Fair Value Grants outstanding at beginning of the 48,875 $ 50.11 Granted — — Vested ( 48,875 ) 50.11 Forfeited — — Grants outstanding at end of the period — $ — |
Restricted Stock Units (RSUs) [Member] | |
Summary of Restricted Stock Award and Restricted Stock Units Activity | The following table summarizes the Company’s RSU activity for the periods indicated: For the Year Ended December 31, 2021 Weighted Average Grant Date Shares Fair Value Grants outstanding at beginning of the 947,079 $ 121.46 Granted 987,464 85.32 Vested ( 276,980 ) 119.54 Forfeited ( 337,357 ) 106.37 Grants outstanding at end of the period 1,320,206 $ 98.69 |
Stock Options [Member] | |
Assumptions for Measuring Fair Values of Stocks | The fair values of stock options granted during the periods presented are measured on the date of grant using the Black-Scholes-Merton option-pricing model, with the following assumptions: For the Year Ended December 31, 2021 2020 2019 Risk-free interest rate (1) 0.4 - 1.3 % 0.1 - 1.3 % 1.4 - 2.5 % Expected dividend yield (2) — — — Expected term (3) 4.99 years 5.00 years 5.04 years Expected volatility (4) 60.1 - 70.8 % 57.3 - 68.2 % 52.5 - 68.9 % (1) The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. (2) The expected dividend yield is zero as the Company has not paid any dividends to date and does not expect to pay dividends in the future. (3) The expected term is estimated using historical exercise behavior. (4) The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. |
OTHER INCOME (LOSS) (Tables)
OTHER INCOME (LOSS) (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Other Income and Expenses [Abstract] | |
Summary of Other Income (Loss) | The following table summarizes other income (loss) for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Interest expense $ ( 63,525 ) $ ( 59,947 ) $ ( 30,669 ) Interest income 354 2,970 7,238 Amortization of investment discount 157 4,489 15,350 Gain from sale of Priority Review Voucher 102,000 108,069 — Gain (loss) on contingent consideration, net* 7,200 ( 45,000 ) — Impairment of equity investment ( 4,488 ) — — Other (expense) income ( 936 ) 517 ( 236 ) Total other income (loss) $ 40,762 $ 11,098 $ ( 8,317 ) |
INCOME TAXES (Tables)
INCOME TAXES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Summary of Loss before Provision (Benefit) for Income Taxes by Jurisdiction | The following table summarizes the loss before the provision (benefit) for income taxes by jurisdiction for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Domestic $ ( 47,633 ) $ ( 204,956 ) $ ( 489,747 ) Foreign ( 371,315 ) ( 348,109 ) ( 224,133 ) Total $ ( 418,948 ) $ ( 553,065 ) $ ( 713,880 ) |
Summary of Provision (Benefit) for Income Taxes | The following table summarizes provision (benefit) for income taxes in the accompanying consolidated financial statements for the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Current provision: Federal $ — $ 4 $ — State ( 40 ) 624 521 Foreign 181 680 1,050 Total current provision 141 1,308 1,571 Deferred benefit: Federal — — ( 15 ) State — — ( 5 ) Foreign ( 309 ) ( 245 ) ( 356 ) Total deferred benefit ( 309 ) ( 245 ) ( 376 ) Total income tax (benefit) expense $ ( 168 ) $ 1,063 $ 1,195 |
Reconciliation Between Effective Tax Rate and Statutory Income Tax Rate | The following table summarizes the reconciliation between the Company’s effective tax rate and the statutory income tax rate for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 Federal income tax rate 21.0 % 21.0 % 21.0 % State taxes 0.4 0.6 4.5 Research and development and other tax 10.0 10.1 5.1 Valuation allowance ( 9.8 ) ( 21.3 ) ( 16.8 ) Permanent differences ( 0.3 ) ( 1.6 ) 2.3 Stock-based compensation ( 3.0 ) 3.5 ( 0.6 ) Basis difference in subsidiary — — ( 8.4 ) Foreign rate differential ( 18.4 ) ( 12.9 ) ( 7.4 ) Other 0.1 0.4 0.1 Effective tax rate ( 0.0 ) % ( 0.2 ) % ( 0.2 ) % |
Analysis of Deferred Tax Assets and Liabilities | The following table summarizes the analysis of the deferred tax assets and liabilities for each of the periods indicated: As of December 31, 2021 2020 (in thousands) Deferred tax assets: Net operating loss carryforwards $ 330,392 $ 333,703 Difference in depreciation and amortization 31,563 31,259 Research and development tax credits 201,512 159,917 Stock-based compensation 38,132 31,212 Lease liabilities 10,890 13,120 Capitalized inventory 24,172 35,959 Debt discount 5,875 — Other 38,315 28,381 Total deferred tax assets 680,851 633,551 Deferred tax liabilities: Right of use asset ( 7,405 ) ( 8,772 ) Debt discount — ( 18,044 ) Total deferred tax liabilities ( 7,405 ) ( 26,816 ) Valuation allowance ( 672,319 ) ( 605,848 ) Net deferred tax assets $ 1,127 $ 887 |
Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits | The follow table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits for each of the periods indicated: For the Year Ended December 31, 2021 2020 2019 (in thousands) Balance at beginning of the period $ 48,475 $ 41,753 $ 37,544 Increase related to current year tax positions 5,503 6,722 4,275 Increase related to prior year tax positions — — 109 Decrease related to prior year tax positions ( 163 ) — ( 175 ) Balance at end of the period $ 53,815 $ 48,475 $ 41,753 |
LEASES (Tables)
LEASES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
Summary of Lease Costs Recognized Under Topic 842 and Other Information Pertaining to Operating Leases | The following table contains a summary of the lease costs recognized and other information pertaining to the Company’s operating leases for the periods indicated: For the Year Ended December 31, 2021 2020 (in thousands) Lease cost Operating lease cost $ 28,737 $ 18,978 Variable lease cost 18,742 17,065 Total lease cost $ 47,479 $ 36,043 Other information Operating lease payments $ 24,449 $ 19,344 Operating lease liabilities arising from obtaining ROU assets 13,225 59,327 Weighted average remaining lease term 3.5 years 4.6 years Weighted average discount rate 7.6 % 7.6 % |
Summary of Maturities of Lease Liabilities and Reconciliation of Lease Liabilities Recognized Under Topic 842 | The following table summarizes maturities of lease liabilities and the reconciliation of lease liabilities as of December 31, 2021: For the Year Ended (in thousands) 2022 $ 18,702 2023 18,326 2024 17,582 2025 9,052 2026 571 Thereafter — Total minimum lease payments 64,233 Less: imputed interest ( 7,672 ) Total operating lease liabilities $ 56,561 Included in the consolidated balance sheet: Current portion of lease liabilities within other current liabilities $ 15,049 Lease liabilities 41,512 Total operating lease liabilities $ 56,561 |
NET LOSS PER SHARE (Tables)
NET LOSS PER SHARE (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Basic and Diluted Net Loss Per Share | Given that the Company recorded a net loss for each of the periods presented, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation. For the Year Ended December 31, 2021 2020 2019 (in thousands, except per share amounts) Net loss $ ( 418,780 ) $ ( 554,128 ) $ ( 715,075 ) Weighted-average common shares outstanding - basic 81,262 77,956 73,615 Effect of dilutive securities* — — — Weighted-average common shares outstanding - diluted 81,262 77,956 73,615 Net loss per share — basic and diluted $ ( 5.15 ) $ ( 7.11 ) $ ( 9.71 ) * For the years ended December 31, 2021, 2020 and 2019, stock options, RSAs, RSUs and ESPP to purchase approximately 9.7 million, 9.0 million and 9.1 million shares of common stock, respectively, were excluded from the net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of the 2024 Notes on diluted net earnings per share using the if-converted method as this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted net earnings per share calculation if the effect is more dilutive. During the year ended December 31, 2021, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net earnings per share as of December 31, 2021. |
COMMITMENTS AND CONTINGENCIES (
COMMITMENTS AND CONTINGENCIES (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Summary of Aggregate Non-Cancelable Contractual Obligations Arising from Manufacturing Obligations | The following table presents non-cancelable contractual obligations arising from long-term contractual arrangements, including obligations related to leases embedded in certain supply agreements: As of (in thousands) 2022 $ 647,124 2023 178,345 2024 138,725 2025 100,838 2026 54,720 Thereafter 109,440 Total manufacturing commitments $ 1,229,192 |
Organization and Nature of Bu_2
Organization and Nature of Business - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||
Cash, cash equivalents and investments | $ 2,125,800 | ||
Cash and cash equivalents | 2,115,869 | $ 1,502,648 | $ 835,080 |
Short-term investments | 435,923 | ||
Restricted cash and investments | $ 9,904 | $ 9,315 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Additional Information (Detail) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021USD ($)$ / shares | Dec. 31, 2020USD ($)Segment | Dec. 31, 2019USD ($) | |
Summary Of Significant Accounting Policies [Line Items] | |||
Number of operating segments | Segment | 1 | ||
Percentage of discount recognized on graded-vesting based on stock-based compensation expense over the purchase period | 15.00% | ||
Right of use assets | $ 45,531 | $ 91,761 | |
Operating lease liabilities | 56,561 | ||
Net loss | (418,780) | $ (554,128) | $ (715,075) |
ASU 2020-06 [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Increase in convertible notes | 96,800 | ||
Decrease in accumulated deficit | (60,200) | ||
Decrease in additional paid-in-capital | (157,000) | ||
Net loss | $ 22,400 | ||
Effect on diluted net earnings (loss) per share | $ / shares | $ 0.28 | ||
Employee Stock Purchase Plan [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Percentage of closing price of common stock | 85.00% | ||
Patents [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Initial term of patents | 20 years | ||
Outside of U.S. [Member] | Product Revenues [Member] | Customer One [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 48.00% | 47.00% | 43.00% |
Outside of U.S. [Member] | Product Revenues [Member] | Customer Two [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 39.00% | 39.00% | 41.00% |
Outside of U.S. [Member] | Product Revenues [Member] | Customer Three [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 10.00% | 11.00% | 13.00% |
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer One [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 41.00% | 45.00% | |
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Two [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 41.00% | 41.00% | |
Outside of U.S. [Member] | Accounts Receivable [Member] | Customer Three [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Concentration of credit risk percentage | 10.00% | 9.00% | |
Minimum [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Accounts receivable payment term | 60 days | ||
Minimum [Member] | Outside of U.S. [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Accounts receivable payment term | 45 days | ||
Maximum [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Accounts receivable payment term | 91 days | ||
Maximum [Member] | Outside of U.S. [Member] | |||
Summary Of Significant Accounting Policies [Line Items] | |||
Accounts receivable payment term | 150 days |
Summary of Estimated Useful Liv
Summary of Estimated Useful Lives of Plant and Equipment (Detail) | 12 Months Ended |
Dec. 31, 2021 | |
Lab and manufacturing equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Office Equipment [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Software and Computer Equipment [Member] | Minimum [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 3 years |
Software and Computer Equipment [Member] | Maximum [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 5 years |
Leasehold Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Lesser of the useful life or the term of the respective lease |
Furniture and Fixtures [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 7 years |
Land [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Not depreciated |
Land Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 25 years |
Construction in Progress [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | Not depreciated until put into service |
Building and Improvements [Member] | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, Estimated useful life | 30 years |
License and Collaboration Agr_2
License and Collaboration Agreements - Additional Information (Detail) | Dec. 21, 2019USD ($)shares | Nov. 30, 2021USD ($) | Oct. 31, 2021USD ($) | Jul. 31, 2021USD ($) | Mar. 31, 2021shares | Nov. 30, 2019USD ($)shares | Apr. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Oct. 31, 2018USD ($)shares | Jul. 31, 2017 | Jun. 30, 2016USD ($) | Dec. 31, 2021USD ($)Milestoneshares | Dec. 31, 2020USD ($)shares | Dec. 31, 2019USD ($) | Feb. 28, 2021USD ($) | Feb. 29, 2020USD ($) | Aug. 08, 2018USD ($)shares | Dec. 31, 2017USD ($) | Dec. 31, 2016USD ($) |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Issuance costs | $ 26,500,000 | $ 300,000 | |||||||||||||||||
Common stock fair value | $ 9,000 | $ 8,000 | $ 312,100,000 | ||||||||||||||||
Product revenues, net | $ 612,401,000 | $ 455,865,000 | $ 380,833,000 | ||||||||||||||||
Common stock, issued | shares | 87,126,974 | 79,374,247 | |||||||||||||||||
Contingent consideration | $ 43,600,000 | $ 50,800,000 | |||||||||||||||||
Intangible asset, net | 14,239,000 | 13,628,000 | |||||||||||||||||
Amortization of in-licensed rights | 706,000 | 662,000 | 849,000 | ||||||||||||||||
Maximum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Percentage of royalty payments | 8.00% | ||||||||||||||||||
Amortization of in-licensed rights | 800,000 | ||||||||||||||||||
Minimum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Percentage of royalty payments | 4.00% | ||||||||||||||||||
Genethon [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Research and development expense | $ 4,000,000 | ||||||||||||||||||
Up-front cash payment under development | $ 157,500,000 | ||||||||||||||||||
Up-front cash payment under sales milestone | $ 78,800,000 | ||||||||||||||||||
Up-front cash payment under development percentage | 75.00% | ||||||||||||||||||
Number of development milestone | Milestone | 0 | ||||||||||||||||||
Other Licensed Products [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Up-front cash payment under development percentage | 25.00% | ||||||||||||||||||
StrideBio, Inc. [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Common stock fair value | $ 29,400,000 | ||||||||||||||||||
Research and development expense | $ 0 | ||||||||||||||||||
Up-front cash payment under development | 450,000,000 | ||||||||||||||||||
Up-front cash payment under sales milestone | 835,000,000 | ||||||||||||||||||
Up-front cash expense | 46,900,000 | ||||||||||||||||||
Up-front cash payment under agreements | $ 17,500,000 | ||||||||||||||||||
Common stock, issued | shares | 301,980 | ||||||||||||||||||
Number of development milestone | Milestone | 0 | ||||||||||||||||||
StrideBio, Inc. [Member] | Series B Preferred Stock [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Preferred stock purchase, shares | shares | 200,000 | ||||||||||||||||||
Preferred stock purchase, value | $ 1,800,000 | ||||||||||||||||||
Myonexus Therapeutics, Inc. [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Final exercise price | $ 178,300,000 | ||||||||||||||||||
Additional development milestone payments to be paid | $ 200,000,000 | ||||||||||||||||||
Contingent consideration | 42,000,000 | 49,500,000 | |||||||||||||||||
Lacerta Therapeutics [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Research and development expense | $ 0 | ||||||||||||||||||
Number of development milestone | Milestone | 0 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory and sales milestone payments | $ 0 | ||||||||||||||||||
Intangible asset, net | $ 6,600,000 | ||||||||||||||||||
Sales milestone payment recorded as an in-license right | 0 | ||||||||||||||||||
Royalty expense | 31,400,000 | 23,200,000 | 19,400,000 | ||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | VYONDYS 53 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory milestone payable | 10,000,000 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | AMONDYS 45 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory milestone payable | $ 10,000,000 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Intellectual Property | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Amortization of in-licensed rights | 3,200,000 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | US [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Royalty payment expiry period | 2024-03 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | EU [member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Royalty payment expiry period | 2024-12 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory and sales milestone payments | $ 50,000,000 | ||||||||||||||||||
Percentage of royalty payments | 5.00% | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Maximum [Member] | Other Countries [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Royalty payment expiry period | 2024-12 | ||||||||||||||||||
BioMarin Pharmaceutical, Inc. [Member] | Minimum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Percentage of royalty payments | 4.00% | ||||||||||||||||||
University of Western Australia [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory and sales milestone payments | 0 | ||||||||||||||||||
Sales milestone payment recorded as an in-license right | 0 | ||||||||||||||||||
Royalty expense | 7,700,000 | 5,700,000 | 3,500,000 | ||||||||||||||||
Carrying value of intangible assets | 1,200,000 | ||||||||||||||||||
University of Western Australia [Member] | EXONDYS 51 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Sales milestone payment recorded as an in-license right | $ 1,000,000 | ||||||||||||||||||
University of Western Australia [Member] | VYONDYS 53 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Sales milestone payment recorded as an in-license right | 500,000 | ||||||||||||||||||
University of Western Australia [Member] | AMONDYS 45 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Sales milestone payment recorded as an in-license right | $ 500,000 | ||||||||||||||||||
University of Western Australia [Member] | Maximum [Member] | EXONDYS 51 [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Payments to UWA on successful achievement of certain development and regulatory milestones | $ 26,000,000 | ||||||||||||||||||
Collaborative Arrangement | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Payment for option exercise | 187,000,000 | ||||||||||||||||||
Development Milestone and Settlement Upfront Fee Recognized as Research and Development Expense | 50,300,000 | 47,300,000 | 113,200,000 | ||||||||||||||||
Contingent research milestone payments | 3,000,000 | ||||||||||||||||||
Collaborative Arrangement | Maximum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Contingent research milestone payments | 11,000,000 | ||||||||||||||||||
Development, regulatory, commercial milestone and up-front royalty payments | $ 4,000,000,000 | ||||||||||||||||||
Collaborative Arrangement | Roche Holding A.G. [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Proceeds From License And Collaborative Agreement | $ 1,200,000,000 | ||||||||||||||||||
Issuance costs | $ 312,100,000 | ||||||||||||||||||
Stock Issued During Period Shares Collaboration and License Agreement | shares | 2,500,000 | ||||||||||||||||||
Payment for option exercise | $ 485,000,000 | ||||||||||||||||||
Single combined performance obligation | 348,700,000 | ||||||||||||||||||
Stock Options Exercised Or Expired | shares | 0 | ||||||||||||||||||
Product revenues, net | $ 89,500,000 | 84,200,000 | |||||||||||||||||
Deferred Revenue | 663,500,000 | ||||||||||||||||||
Deferred Revenue, Current | 89,200,000 | ||||||||||||||||||
Deferred Revenue Separate Material Options Right | 485,000,000 | 485,000,000 | |||||||||||||||||
Research and development expense | 90,500,000 | 66,500,000 | |||||||||||||||||
Collaboration receivable | 18,600,000 | ||||||||||||||||||
Collaborative Arrangement | Roche Holding A.G. [Member] | Maximum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Regulatory and Sales Milestones Payment Received | $ 1,700,000,000 | ||||||||||||||||||
Collaborative Arrangement | Genethon [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Research and development expense | 11,700,000 | 10,100,000 | 9,000,000 | ||||||||||||||||
Up-front cash payment under development | $ 28,000,000 | ||||||||||||||||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Development, regulatory and sales milestones payments to be paid | $ 102,800,000 | ||||||||||||||||||
Common stock purchased,shares | shares | 1,140,728 | ||||||||||||||||||
Equity Investment Agreement [Member] | Lysogene S.A. [Member] | Other Noncurrent Assets [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Upfront License Fee | 2,500,000 | ||||||||||||||||||
Equity Investment Agreement [Member] | Lacerta Therapeutics [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Investment change in carrying value | 0 | 0 | $ 0 | ||||||||||||||||
License Development And Option Agreement | Lacerta Therapeutics [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Deferred Revenue | $ 42,000,000 | ||||||||||||||||||
Pompe License Agreement | Lacerta Therapeutics [Member] | Maximum [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Deferred Revenue | $ 44,000,000 | ||||||||||||||||||
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Series A Preferred Stock | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Up-front payment | shares | 4,500,000 | ||||||||||||||||||
Series A Preferred Stock Purchase Agreement | Lacerta Therapeutics [Member] | Other Noncurrent Assets [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Deferred Revenue | $ 30,000,000 | ||||||||||||||||||
License And Purchase Agreement | Lacerta Therapeutics [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Deferred Revenue | $ 38,000,000 | ||||||||||||||||||
Nationwide License Agreement [Member] | Nationwide Children's Hospital [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Development, regulatory and sales milestones payments to be paid | 0 | ||||||||||||||||||
Sublicense payment | $ 38,000,000 | ||||||||||||||||||
Sales milestone payment recorded as an in-license right | 0 | ||||||||||||||||||
Nationwide License Agreement [Member] | Nationwide Children's Hospital [Member] | Research and Development Expense [Member] | |||||||||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||||||||||||||||
Sublicense payment | $ 28,700,000 | $ 9,300,000 |
Gain from Sale of Priority Re_2
Gain from Sale of Priority Review Voucher - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
F D A | ||
Gain From Sale Of Intangible Asset [Line Items] | ||
Proceeds from sale of rare pediatric disease priority review voucher, Net of commission | $ 102 | $ 108.1 |
Assets and Liabilities Measured
Assets and Liabilities Measured and Carried at Fair Value (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | $ 1,597,500 | $ 1,706,470 | |
Contingent consideration | 43,600 | 50,800 | |
Total liabilities | 43,600 | 50,800 | |
Money Market Funds [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 1,562,358 | 629,440 | |
Government and Government Agency Bonds [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 1,037,981 | ||
Strategic Equity Investments [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 34,892 | 38,799 | |
Certificates of Deposit [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 250 | 250 | |
Level 1 [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 1,565,088 | 1,671,370 | |
Level 1 [Member] | Money Market Funds [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 1,562,358 | 629,440 | |
Level 1 [Member] | Government and Government Agency Bonds [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 1,037,981 | ||
Level 1 [Member] | Strategic Equity Investments [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 2,480 | 3,699 | |
Level 1 [Member] | Certificates of Deposit [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 250 | 250 | |
Level 3 [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | 32,412 | 35,100 | $ 30,000 |
Contingent consideration | 43,600 | 50,800 | |
Total liabilities | 43,600 | 50,800 | $ 5,200 |
Level 3 [Member] | Strategic Equity Investments [Member] | |||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |||
Total assets | $ 32,412 | $ 35,100 |
Summary of Fair Value of Level
Summary of Fair Value of Level 3 Financial Assets (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value at the beginning of the period | $ 1,706,470 | |
Fair value at the End of the period | 1,597,500 | $ 1,706,470 |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value at the beginning of the period | 35,100 | 30,000 |
Additions | 1,800 | 5,100 |
Changes in estimated fair value | (4,488) | |
Fair value at the End of the period | $ 32,412 | $ 35,100 |
Summary of Fair Value of Leve_2
Summary of Fair Value of Level 3 Financial Liabilities (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value at the beginning of the period | $ 50,800 | |
Additions | $ 600 | |
Changes in estimated fair value | (7,200) | 45,000 |
Fair value at the end of the period | 43,600 | 50,800 |
Level 3 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Fair value at the beginning of the period | 50,800 | 5,200 |
Additions | 600 | |
Changes in estimated fair value | (7,200) | 45,000 |
Fair value at the end of the period | $ 43,600 | $ 50,800 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Fair Value Disclosures [Abstract] | |||
Impairment of investment in private companies | $ 4,488 | ||
Business Combination, Contingent Consideration Arrangements, Change in Amount of Contingent Consideration, Liability | 600 | ||
Business Combination, Contingent Consideration Arrangements, Change in Amount of Contingent Consideration, Liability | (7,200) | 45,000 | |
Contingent consideration | $ 43,600 | $ 50,800 |
Summary of Company Financial As
Summary of Company Financial Assets with Maturities of Less Than 90 Days Included in Cash Equivalents (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 1,562,358 | $ 1,231,498 |
Money Market Funds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | 1,562,358 | 629,440 |
Government and Government Agency Bonds [Member] | ||
Cash and Cash Equivalents [Line Items] | ||
Cash equivalents | $ 0 | $ 602,058 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2021 | |
Cash and Cash Equivalents [Abstract] | ||
Weighted average maturity period of available-for-sale securities | 2 months | |
Available for sale debt securities current, Fair market value | $ 435,923,000 | $ 0 |
Summary of Company Cash, Cash E
Summary of Company Cash, Cash Equivalents and Investments (Detail) - USD ($) | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | $ 2,115,869,000 | $ 1,938,549,000 | |
Cash and cash equivalents, Gross unrealized gains | 9,000 | ||
Cash and cash equivalents | 2,115,869,000 | 1,502,648,000 | $ 835,080,000 |
Cash, cash equivalents and investments, Amortized cost | 2,115,869,000 | 1,502,639,000 | |
Cash, cash equivalents and investments, Gross unrealized gains | 22,000 | ||
Cash, cash equivalents and investments, Fair market value | 2,115,869,000 | 1,938,571,000 | |
Available for sale debt securities current, Amortized cost | 435,910,000 | ||
Available for sale debt securities current, Gross unrealized gains | 13,000 | ||
Available for sale debt securities current, Fair market value | 0 | 435,923,000 | |
Cash and Money Market Funds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Cash and cash equivalents, Amortized cost | 2,115,869,000 | 900,590,000 | $ 1,938,549,000 |
Cash and cash equivalents | 2,115,869,000 | 900,590,000 | |
Cash, cash equivalents and investments, Fair market value | 1,938,571,000 | ||
Government and Government Agency Bonds [Member] | |||
Cash and Cash Equivalents [Line Items] | |||
Available for sale debt securities, Amortized cost | 1,037,959,000 | ||
Available for sale debt securities, Gross unrealized gains | 22,000 | ||
Available for sale debt securities, Fair market value | $ 1,037,981,000 | ||
Cash, cash equivalents and investments, Fair market value | $ 2,115,869,000 |
Summary of product revenues, ne
Summary of product revenues, net by product (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Product Information [Line Items] | |||
Product revenues, net | $ 612,401 | $ 455,865 | $ 380,833 |
EXONDYS 51 [Member] | |||
Product Information [Line Items] | |||
Product revenues, net | 454,361 | 422,007 | 380,718 |
VYONDYS 53 [Member] | |||
Product Information [Line Items] | |||
Product revenues, net | 89,511 | 33,858 | 115 |
AMONDYS 45 [Member] | |||
Product Information [Line Items] | |||
Product revenues, net | $ 68,529 |
Summary of Components of Accoun
Summary of Components of Accounts Receivable (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Receivables, Net, Current [Abstract] | ||
Product sales receivable, net of discounts and allowances | $ 152,990 | $ 100,870 |
Government contract receivables | 470 | |
Total accounts receivable, net | $ 152,990 | $ 101,340 |
Summary of Change in Reserves f
Summary of Change in Reserves for Discounts and Allowances (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | $ 50,970 | $ 51,503 |
Provision | 117,452 | 78,439 |
Payments/credits | 97,956 | 78,972 |
Ending balance | 70,466 | 50,970 |
Chargebacks [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 2,281 | 588 |
Provision | 13,308 | 9,700 |
Payments/credits | 14,790 | 8,007 |
Ending balance | 799 | 2,281 |
Rebates [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 41,771 | 44,738 |
Provision | 78,637 | 52,180 |
Payments/credits | 59,902 | 55,147 |
Ending balance | 60,506 | 41,771 |
Prompt Pay [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 1,949 | 1,506 |
Provision | 9,400 | 6,384 |
Payments/credits | 8,551 | 5,941 |
Ending balance | 2,798 | 1,949 |
Other Accruals [Member] | ||
Accounts Notes And Loans Receivable [Line Items] | ||
Beginning balance | 4,969 | 4,671 |
Provision | 16,107 | 10,175 |
Payments/credits | 14,713 | 9,877 |
Ending balance | $ 6,363 | $ 4,969 |
Summary of Total Reserves Inclu
Summary of Total Reserves Included in Consolidated Balance Sheets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Receivables, Net, Current [Abstract] | |||
Reduction to accounts receivable | $ 8,321 | $ 8,352 | |
Component of accrued expenses | 62,145 | 42,618 | |
Total reserves | $ 70,466 | $ 50,970 | $ 51,503 |
Inventory - Summary of Componen
Inventory - Summary of Components of Inventory (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 58,822 | $ 71,717 |
Work in progress | 230,194 | 139,704 |
Finished goods | 26,717 | 20,540 |
Total inventory | $ 315,733 | $ 231,961 |
Inventory - Summarizes The Bala
Inventory - Summarizes The Balance Sheet Classification (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Inventory Disclosure [Abstract] | ||
Inventory | $ 186,212 | $ 231,961 |
Other non-current assets | 129,521 | |
Total inventory | $ 315,733 | $ 231,961 |
Summary of Other Current Assets
Summary of Other Current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Prepaid Expense and Other Assets, Current [Abstract] | ||
Manufacturing-related deposits and prepaids | $ 93,656 | $ 134,430 |
Collaboration receivable | 18,647 | 34,184 |
Prepaid clinical and pre-clinical expenses | 12,667 | 16,224 |
Prepaid maintenance services | 8,452 | 6,411 |
Prepaid research expenses | 3,082 | 5,854 |
Prepaid income tax | 1,100 | 4,939 |
Prepaid insurance | 5,282 | 4,158 |
Leasehold improvement receivable | 0 | 3,059 |
Other | 6,142 | 4,065 |
Total other current assets | $ 149,028 | $ 213,324 |
Summary of Other Non-current As
Summary of Other Non-current Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Prepaid Expense and Other Assets, Noncurrent [Abstract] | ||
Non-current inventory | $ 129,521 | $ 0 |
Manufacturing-related deposits and prepaids | 112,765 | 148,525 |
Strategic investments | 34,892 | 38,799 |
Restricted cash and investments | 9,904 | 9,315 |
Prepaid clinical expenses | 2,007 | 3,395 |
Other | 3,860 | 3,669 |
Total other non-current assets | $ 292,949 | $ 203,703 |
Summarizes Components of Proper
Summarizes Components of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 303,788 | $ 266,457 |
Less: accumulated depreciation | (112,632) | (76,027) |
Property and equipment, net | 191,156 | 190,430 |
Leasehold Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 103,370 | 55,019 |
Lab and manufacturing equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 64,613 | 49,571 |
Building and Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 47,605 | 39,397 |
Software and Computer Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 42,506 | 33,948 |
Furniture and Fixtures [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 9,242 | 7,010 |
Land [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 5,183 | 5,183 |
Land Improvements [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 4,921 | 3,610 |
Office Equipment [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 1,189 | 1,178 |
Construction in Progress [Member] | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 25,159 | $ 71,541 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | |||
Depreciation expense | $ 36.6 | $ 25.2 | $ 22.8 |
Intangible Assets, Net - Summar
Intangible Assets, Net - Summary of Components of Intangible Assets (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 21,254 | $ 20,837 |
Less: accumulated amortization | (7,015) | (7,209) |
Intangible assets, net | 14,239 | 13,628 |
Patents [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | 12,382 | 11,210 |
In-Licensed Rights [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | 8,573 | 8,073 |
Software Licenses [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Intangible assets, gross | $ 299 | $ 1,554 |
Intangible Assets, Net - Additi
Intangible Assets, Net - Additional Information (Detail) - USD ($) | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Jul. 31, 2017 | |
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of in-licensed rights | $ 706,000 | $ 662,000 | $ 849,000 | |
Patents [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of in-licensed rights | 600,000 | 600,000 | 400,000 | |
Patents [Member] | Research and Development Expense [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Book value of patent abandoned | 500,000 | 100,000 | 200,000 | |
Software Development [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of in-licensed rights | $ 500,000 | 400,000 | ||
Maximum [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of in-licensed rights | $ 800,000 | |||
Minimum [Member] | Software Development [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Amortization of in-licensed rights | 100,000 | |||
EXONDYS 51 [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
In-lincensed Rights | 1,000,000 | |||
EXONDYS 51 [Member] | Maximum [Member] | Bio Marin [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
Sales milestone payment recorded as an in-license right | $ 6,600,000 | |||
VYONDYS 53 [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
In-lincensed Rights | 500,000 | |||
VYONDYS Fifty Three [Member] | ||||
Finite Lived Intangible Assets [Line Items] | ||||
In-lincensed Rights | $ 500,000 |
Intangible Assets, Net - Summ_2
Intangible Assets, Net - Summary of Estimated Future Amortization for Intangible Assets (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Goodwill and Intangible Assets Disclosure [Abstract] | |
2022 | $ 1,395 |
2023 | 1,394 |
2024 | 1,391 |
2025 | 1,330 |
2026 | 1,200 |
Thereafter | 7,529 |
Total | $ 14,239 |
Summary of Accrued Expenses (De
Summary of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Payables and Accruals [Abstract] | ||
Accrued employee compensation costs | $ 48,299 | $ 50,803 |
Product revenue related reserves | 62,145 | 42,618 |
Accrued contract manufacturing costs | 104,311 | 36,543 |
Accrued clinical and pre-clinical costs | 25,955 | 22,169 |
Accrued professional fees | 9,381 | 10,221 |
Accrued milestone expense | 100 | 9,380 |
Accrued royalties | 11,965 | 7,793 |
Accrued collaboration cost-sharing | 2,887 | 3,516 |
Accrued interest expense | 1,045 | 1,045 |
Other | 5,609 | 9,465 |
Total accrued expenses | $ 271,697 | $ 193,553 |
Indebtedness - Additional Infor
Indebtedness - Additional Information (Detail) | Dec. 31, 2024USD ($) | Dec. 20, 2023USD ($) | Sep. 24, 2020USD ($) | Dec. 13, 2019USD ($)Tranch | Nov. 14, 2017USD ($)shares$ / shares | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Dec. 31, 2023USD ($) | Dec. 31, 2022USD ($) |
Debt Instrument [Line Items] | ||||||||||
Debt instrument, face amount | $ 1,119,993,000 | |||||||||
Long-term debt | 1,096,876,000 | $ 992,493,000 | ||||||||
ASU 2020-06 [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Increase in convertible notes | 96,800,000 | |||||||||
Decrease in accumulated deficit | (60,200,000) | |||||||||
Decrease in additional paid-in-capital | (157,000,000) | |||||||||
Convertible Debt, The Term Loan, The Revolver and The Mortgage Loans [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Long-term debt | 1,096,900,000 | |||||||||
Interest expense related to debt facilities | 63,500,000 | 59,900,000 | $ 30,700,000 | |||||||
Aggregate long-term debt | $ 903,400,000 | $ 305,300,000 | $ 55,900,000 | |||||||
2024 Convertible Notes [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Debt instrument, face amount | $ 570,000,000 | $ 569,993,000 | $ 569,993,000 | |||||||
Debt instrument, maturity date | Nov. 15, 2024 | |||||||||
Debt instrument, interest rate per annum | 1.50% | |||||||||
Debt instrument, payment frequency | The 2024 Notes contain customary covenants and events of default, occurrence of which permits the certain holders to accelerate all outstanding obligations, including principal and interest. | |||||||||
Debt instrument, conversion rate | 0.013621 | |||||||||
Debt instrument, conversion price | $ / shares | $ 73.42 | |||||||||
Debt issuance costs | $ 10,600,000 | |||||||||
Effective interest rate percentage | 6.90% | 6.90% | ||||||||
Capped calls strike price | $ / shares | 73.42 | |||||||||
Capped calls cap price | $ / shares | 104.88 | |||||||||
Amount paid for capped calls transactions | $ 50,900,000 | |||||||||
Debt discount | $ 0 | $ 98,721,000 | ||||||||
Debt issuance costs | 6,320,000 | 6,510,000 | ||||||||
Aggregate long-term debt | $ 563,673,000 | 464,762,000 | ||||||||
2024 Convertible Notes [Member] | ASU 2020-06 [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Effective interest rate percentage | 1.90% | |||||||||
Interest expense related to debt facilities | $ 10,700,000 | $ 31,400,000 | $ 29,900,000 | |||||||
Increase in convertible notes | 96,800,000 | |||||||||
Decrease in accumulated deficit | (60,200,000) | |||||||||
Decrease in additional paid-in-capital | (157,000,000) | |||||||||
Decrease in interest expense | (22,400,000) | |||||||||
2024 Convertible Notes [Member] | Common Stock [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Debt instrument, convertible into shares | shares | 7,763,552 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Debt instrument, maturity date | Dec. 31, 2024 | Dec. 20, 2023 | ||||||||
Debt instrument, interest rate per annum | 8.50% | |||||||||
Current borrowing capacity | $ 500,000,000 | |||||||||
Number of tranches | Tranch | 2 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch A [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Current borrowing capacity | $ 250,000,000 | |||||||||
Proceeds after debt discount and issuance cost | 244,900,000 | |||||||||
Debt discount | 9,400,000 | |||||||||
Debt issuance costs | 700,000 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch A [Member] | Forecast [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Proceeds after debt discount and issuance cost | $ 5,000,000 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch B [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Current borrowing capacity | $ 300,000,000 | $ 250,000,000 | ||||||||
Proceeds after debt discount and issuance cost | 291,100,000 | |||||||||
Debt discount | 14,900,000 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | December 2019 Term Loan [Member] | Tranch B [Member] | Forecast [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Proceeds after debt discount and issuance cost | $ 6,000,000 | |||||||||
Biopharma Credit PLC and Biopharma Credit Investments V (Master) LP [Member] | Maximum [Member] | December 2019 Term Loan [Member] | Tranch B [Member] | ||||||||||
Debt Instrument [Line Items] | ||||||||||
Debt issuance costs | $ 100,000 |
Indebtedness - Summary of Debt
Indebtedness - Summary of Debt Facilities (Detail) - USD ($) | Dec. 31, 2021 | Dec. 31, 2020 | Nov. 14, 2017 |
Debt Instrument [Line Items] | |||
Principal amount | $ 1,119,993,000 | ||
Total carrying value of debt facilities | 1,096,876,000 | $ 992,493,000 | |
Total fair value of debt facilities | 1,422,223,000 | 1,944,249,000 | |
2024 Convertible Notes [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 569,993,000 | 569,993,000 | $ 570,000,000 |
Unamortized discount | 0 | (98,721,000) | |
Unamortized discount - debt issuance costs | (6,320,000) | (6,510,000) | |
Net carrying value | 563,673,000 | 464,762,000 | |
Total fair value of debt facilities | 846,138,000 | 1,394,249,000 | |
2019 Term Loan [Member] | |||
Debt Instrument [Line Items] | |||
Principal amount | 550,000,000 | 550,000,000 | |
Unamortized discount | (16,797,000) | (22,269,000) | |
Net carrying value | 533,203,000 | 527,731,000 | |
Total fair value of debt facilities | $ 576,085,000 | $ 550,000,000 |
Indebtedness - Summarizes Total
Indebtedness - Summarizes Total Gross Payments Due under Company's Debt Arrangements (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Debt Disclosure [Abstract] | |
2022 | $ 0 |
2023 | 250,000 |
2024 | 869,993 |
2025 | 0 |
2026 | 0 |
Thereafter | 0 |
Total payments | $ 1,119,993 |
Equity - Additional Information
Equity - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands, shares in Millions | 1 Months Ended | |||||
Oct. 31, 2021 | Feb. 29, 2020 | Nov. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2021 | Dec. 31, 2020 | |
Class Of Stock [Line Items] | ||||||
Common stock issued | 2.5 | 0.3 | ||||
Common stock fair value | $ 312,100 | $ 9 | $ 8 | |||
Common stock average price per share | $ 81 | $ 140.41 | ||||
Net proceeds from issue of common stock | $ 548,500 | $ 365,400 | ||||
Issuance costs | $ 26,500 | $ 300 | ||||
Roche Holding A.G. [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Direct Transaction Fees | $ 4,300 | |||||
StrideBio, Inc. [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Common stock fair value | $ 29,400 | |||||
Underwritten Public Offering [Member] | ||||||
Class Of Stock [Line Items] | ||||||
Common stock issued | 7.1 | 2.6 |
Stock Based Compensation - Addi
Stock Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |||||||||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | Jun. 30, 2020 | Jun. 30, 2019 | Jun. 30, 2018 | Jun. 30, 2016 | Sep. 30, 2014 | Jun. 30, 2013 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Number of shares approved under the plan | 3,800,000 | |||||||||
Term of award | 10 years | |||||||||
Share based compensation granted under plan vest period | 4 years | |||||||||
Weighted-average grant date fair value per share of stock-based awards, granted to employees | $ 61.38 | $ 70.93 | ||||||||
Fair value of options granted | $ 61.38 | $ 70.93 | ||||||||
Granted | $ 86.41 | |||||||||
Stock-based compensation expense | $ 113,943 | $ 108,070 | $ 78,602 | |||||||
Unrecognized stock based compensation expense | 175,700 | |||||||||
Options with Service conditions [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Unrecognized stock based compensation expense | 92,400 | |||||||||
Awards with Service and Market Conditions [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Unrecognized stock based compensation expense | 4,300 | |||||||||
Stock Units with Service Conditions [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Unrecognized stock based compensation expense | 79,000 | |||||||||
Stock Options [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Stock-based compensation expense | $ 68,995 | $ 68,832 | $ 53,427 | |||||||
Restricted Stock Units (RSUs) [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Grants, Restricted stock units and awards | 987,464 | |||||||||
Restricted stock units and awards with performance conditions remaining to be vested | 1,320,206 | 947,079 | ||||||||
CEO [Member] | Stock Options [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Weighted-average grant date fair value per share of stock-based awards, granted to employees | $ 13.48 | |||||||||
Stock options granted | 3,300,000 | |||||||||
Fair value of options granted | $ 13.48 | |||||||||
Granted | $ 34.65 | |||||||||
2013 Employee Stock Purchase Plan [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Number of shares approved under the plan | 500,000 | 300,000 | 300,000 | |||||||
Common stock remain available for future grant | 400,000 | |||||||||
Term of award | 24 months | |||||||||
Percentage of closing price of common stock | 85.00% | |||||||||
2013 Employee Stock Purchase Plan [Member] | Maximum [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Share-based compensation award, expiration date | Aug. 31, 2023 | |||||||||
2014 Employment Commencement Incentive Plan (2014 Plan) [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Number of shares approved under the plan | 600,000 | |||||||||
Common stock remain available for future grant | 1,500,000 | |||||||||
Common stock increase in available for future grant shares | 7,000,000 | |||||||||
2018 Equity Incentive Plan [Member] | ||||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||||
Number of shares approved under the plan | 2,900,000 |
Assumptions for Measuring Fair
Assumptions for Measuring Fair Values of Stocks (Detail) | 12 Months Ended | |||||||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Expected dividend yield | 0.00% | |||||||
Stock Options [Member] | ||||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||||
Risk-free interest rate, minimum | [1] | 0.40% | 0.10% | 1.40% | ||||
Risk-free interest rate, maximum | 1.30% | [1] | 1.30% | [1] | 2.50% | [1] | 3.00% | |
Expected dividend yield | [2] | 0.00% | ||||||
Expected term | [3] | 4 years 11 months 26 days | 5 years | 5 years 14 days | ||||
Expected volatility, minimum | [4] | 60.10% | 57.30% | 52.50% | ||||
Expected volatility, maximum | 70.80% | [4] | 68.20% | [4] | 68.90% | [4] | 60.80% | |
[1] | The risk-free interest rate is estimated using an average of Treasury bill interest rates over a historical period commensurate with the expected term of the option that correlates to the prevailing interest rates at the time of grant. | |||||||
[2] | The expected dividend yield is zero as the Company has not paid any dividends to date and does not expect to pay dividends in the future. | |||||||
[3] | The expected term is estimated using historical exercise behavior. | |||||||
[4] | The expected volatility is the implied volatility in exchange-traded options of the Company’s common stock. |
Assumptions for Measuring Fai_2
Assumptions for Measuring Fair Values of Stocks (Parenthetical) (Detail) | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Expected dividend yield | 0.00% |
Summary of Stock Option Activit
Summary of Stock Option Activity (Detail) $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2021USD ($)$ / sharesshares | |
Stock Option Shares | |
Grants outstanding at beginning of the period | shares | 7,844,947 |
Granted | shares | 1,685,860 |
Exercised | shares | (283,622) |
Cancelled and forfeited | shares | (1,050,264) |
Grants outstanding at end of the period | shares | 8,196,921 |
Grants exercisable at end of the period | shares | 2,532,438 |
Grants vested and expected to vest at end of the period | shares | 7,867,722 |
Weighted Average Exercise Price | |
Grants outstanding at beginning of the period | $ / shares | $ 70.61 |
Granted | $ / shares | 86.41 |
Exercised | $ / shares | 45.71 |
Cancelled and forfeited | $ / shares | 112.18 |
Grants outstanding at end of the period | $ / shares | 69.39 |
Grants exercisable at end of the period | $ / shares | 84.71 |
Grants vested and expected to vest at end of the period | $ / shares | $ 68.17 |
Aggregate Intrinsic Value | |
Outstanding at end of year | $ | $ 248,479 |
Exercisable at end of year | $ | 59,069 |
Vested and expected to vest | $ | $ 247,434 |
Weighted Average Remaining Contractual Life (Years) | |
Outstanding at end of year | 6 years 4 months 24 days |
Exercisable at end of year | 5 years 7 months 6 days |
Vested and expected to vest | 6 years 3 months 18 days |
Summary of Company's Stock Opti
Summary of Company's Stock Options Vested and Exercised (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share-based Payment Arrangement [Abstract] | |||
Aggregate grant date fair value of stock options vested | $ 79,068 | $ 80,355 | $ 50,878 |
Aggregate intrinsic value of stock options exercised | $ 10,622 | $ 144,750 | $ 109,707 |
Summary of Restricted Stock Awa
Summary of Restricted Stock Award Activity (Detail) - Restricted Stock Awards (RSAs) [Member] | 12 Months Ended |
Dec. 31, 2021$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares, Grants outstanding at beginning of the period | shares | 48,875 |
Shares, Vested | shares | (48,875) |
Shares, Grants outstanding at end of the period | shares | 0 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at beginning of the period | $ / shares | $ 50.11 |
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares | 50.11 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at end of the period | $ / shares | $ 0 |
Summary of Restricted Stock Uni
Summary of Restricted Stock Unit Activity (Detail) - Restricted Stock Units (RSUs) [Member] | 12 Months Ended |
Dec. 31, 2021$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Shares, Grants outstanding at beginning of the period | shares | 947,079 |
Shares, Granted | shares | 987,464 |
Shares, Vested | shares | (276,980) |
Shares, Forfeited | shares | (337,357) |
Shares, Grants outstanding at end of the period | shares | 1,320,206 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at beginning of the period | $ / shares | $ 121.46 |
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | 85.32 |
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares | 119.54 |
Weighted Average Grant Date Fair Value per Share, Forfeited | $ / shares | 106.37 |
Weighted Average Grant Date Fair Value per Share, Grants outstanding at end of the period | $ / shares | $ 98.69 |
Summary of Employee Stock Purch
Summary of Employee Stock Purchase Plan Activity and Expense (Detail) - 2013 Employee Stock Purchase Plan [Member] - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||
Number of shares purchased | 111,171 | 102,031 | 92,086 |
Proceeds received (in millions) | $ 7.8 | $ 7.5 | $ 5.1 |
Summary of Stock-Based Compensa
Summary of Stock-Based Compensation Expense by Function Included within Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 113,943 | $ 108,070 | $ 78,602 |
Research and Development [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 50,526 | 41,671 | 27,681 |
Selling, General and Administrative [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 63,417 | $ 66,399 | $ 50,921 |
Summary of Stock-Based Compen_2
Summary of Stock-Based Compensation Expense by Grant Type Included within Consolidated Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 113,943 | $ 108,070 | $ 78,602 |
Stock Options [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 68,995 | 68,832 | 53,427 |
Restricted Stock Awards/Units (RSAs/RSUs) [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | 40,055 | 33,457 | 20,103 |
Employee Stock Purchase Plan [Member] | |||
Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | |||
Total stock-based compensation | $ 4,893 | $ 5,781 | $ 5,072 |
401 (K) PLAN - Additional Infor
401 (K) PLAN - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2015 | |
Retirement Benefits [Abstract] | ||||
Expense related to 401 (K) Plan | $ 5.3 | $ 5.3 | $ 3.4 | |
Defined contribution plan, employers matching contribution, service period | 1 year | |||
Defined contribution plan, employers matching contribution, vesting percentage after one years of service | 100.00% | |||
Description of 401(k) plan | The Company sponsors a 401(k) Plan (“the Plan”) in the U.S. and other retirement plans in the rest of the world, all of which are defined contribution plans. The Plan is available to all employees who are age 21 or older. Participants may make voluntary contributions and the Company makes matching contributions according to the Plan’s matching formula. Matching contributions fully vest after one year of service for all employees. The expense related to the Plan primarily consists of the Company’s matching contributions. | |||
Age limit for eligibility to participate in the defined contribution plan | 21 years |
Summary of Other Income (Loss)
Summary of Other Income (Loss) (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |||
Nonoperating Income (Expense) [Abstract] | |||||
Interest expense | $ (63,525) | $ (59,947) | $ (30,669) | ||
Interest income | 354 | 2,970 | 7,238 | ||
Amortization of investment discount | 157 | 4,489 | 15,350 | ||
Gain from sale of Priority Review Voucher | 102,000 | 108,069 | |||
Gain (loss) on contingent consideration, net | 7,200 | [1] | (45,000) | [1] | |
Impairment of equity investment | (4,488) | ||||
Other expense | (936) | 517 | (236) | ||
Total other income (loss) | $ 40,762 | $ 11,098 | $ (8,317) | ||
[1] | The gain (loss) on contingent consideration, net is related to the fair value adjustment of the regulatory-related contingent payments that are accounted for as derivatives. Please see Note 5. Fair Value Measurements for further details. |
Summary of Loss before Provisio
Summary of Loss before Provision (Benefit) for Income Taxes by Jurisdiction (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |||
Domestic | $ (47,633) | $ (204,956) | $ (489,747) |
Foreign | (371,315) | (348,109) | (224,133) |
Loss before income tax (benefit) expense | $ (418,948) | $ (553,065) | $ (713,880) |
Summary of Provision (Benefit)
Summary of Provision (Benefit) for Income Taxes (Detail) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Current provision: | ||||
Federal | $ 4 | |||
State | $ (40) | 624 | $ (521) | |
Foreign | 181 | 680 | 1,050 | |
Total current provision | 141 | 1,308 | $ 1,571 | |
Deferred benefit: | ||||
Federal | $ (15) | |||
State | (5) | |||
Foreign | (309) | (245) | (356) | |
Total deferred benefit | (309) | (245) | (376) | |
Total current provision | $ (168) | $ 1,063 | $ 1,195 |
Reconciliation Between Effectiv
Reconciliation Between Effective Tax Rate and Statutory Income Tax Rate (Detail) | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Income Tax Disclosure [Abstract] | |||
Federal income tax rate | 21.00% | 21.00% | 21.00% |
State taxes | 0.40% | 0.60% | 4.50% |
Research and development and other tax credits | 10.00% | 10.10% | 5.10% |
Valuation allowance | (9.80%) | (21.30%) | (16.80%) |
Permanent differences | (0.30%) | (1.60%) | 2.30% |
Stock-based compensation | (3.00%) | 3.50% | (0.60%) |
Basis difference in subsidiary | (8.40%) | ||
Foreign rate differential | (18.40%) | (12.90%) | (7.40%) |
Other | 0.10% | 0.40% | 0.10% |
Effective tax rate | (0.00%) | (0.20%) | 0.20% |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 1 Months Ended | 12 Months Ended | |
Feb. 28, 2019 | Dec. 31, 2021 | Dec. 31, 2020 | |
Income Taxes [Line Items] | |||
Net change in valuation allowance for deferred tax assets | $ 66,500,000 | $ 117,000,000 | |
Net operating loss carried forwards | 330,392,000 | 333,703,000 | |
Accrual for interest or penalties | 0 | 0 | |
Recognized interest and/or penalties | $ 0 | $ 0 | |
Minimum [Member] | |||
Income Taxes [Line Items] | |||
Operating loss carryforwards, ending of expiration year | 2022 | ||
Maximum [Member] | |||
Income Taxes [Line Items] | |||
Operating loss carryforwards, ending of expiration year | 2041 | ||
Federal [Member] | |||
Income Taxes [Line Items] | |||
Net operating loss carryforwards | $ 1,280,400,000 | ||
Net operating loss carryforwards subject to expiration | 577,200,000 | ||
Net operating loss carried forwards | 703,200,000 | ||
State [Member] | |||
Income Taxes [Line Items] | |||
Net operating loss carryforwards | 841,200,000 | ||
Net operating loss carryforwards subject to expiration | 797,000,000 | ||
Net operating loss carried forwards | 44,200,000 | ||
Foreign [Member] | Sarepta International Holdings GmbH [Member] | |||
Income Taxes [Line Items] | |||
Net operating loss carryforwards | $ 13,600,000 | ||
Research and Development Credits [Member] | |||
Income Taxes [Line Items] | |||
Research and development credits carryforward, Description | These federal and state research and development credits begin to expire between 2022 and 2041 and between 2022 and 2036, respectively. | ||
Research and Development Credits [Member] | Federal [Member] | |||
Income Taxes [Line Items] | |||
Research and development credits carryforward, Amount | $ 139,200,000 | ||
Research and Development Credits [Member] | State [Member] | |||
Income Taxes [Line Items] | |||
Research and development credits carryforward, Amount | $ 77,000,000 | ||
Warrant Agreement | Myonexus Therapeutics, Inc. [Member] | |||
Income Taxes [Line Items] | |||
Accumulated costs related to acquisition | $ 253,700,000 | ||
Up-front and milestone payments | 85,000,000 | ||
Exercise option | $ 168,700,000 |
Analysis of Deferred Tax Assets
Analysis of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Deferred tax assets: | ||
Net operating loss carryforwards | $ 330,392 | $ 333,703 |
Difference in depreciation and amortization | 31,563 | 31,259 |
Research and development tax credits | 201,512 | 159,917 |
Stock-based compensation | 38,132 | 31,212 |
Lease liabilities | 10,890 | 13,120 |
Capitalized inventory | 24,172 | 35,959 |
Debt discount | 5,875 | |
Other | 38,315 | 28,381 |
Total deferred tax assets | 680,851 | 633,551 |
Deferred tax liabilities: | ||
Right of use asset | (7,405) | (8,772) |
Debt discount | (18,044) | |
Total deferred tax liabilities | (7,405) | (26,816) |
Valuation allowance | (672,319) | (605,848) |
Net deferred tax assets | $ 1,127 | $ 887 |
Reconciliation of Beginning and
Reconciliation of Beginning and Ending Amount of Unrecognized Tax Benefits (Detail) - USD ($) $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Tax Disclosure [Abstract] | ||||
Balance at beginning of the period | $ 48,475 | $ 41,753 | $ 37,544 | |
Increase related to current year tax positions | 5,503 | 6,722 | $ 4,275 | |
Increase related to prior year tax positions | 109 | |||
Decrease related to prior year tax positions | (163) | (175) | ||
Balance at end of the period | $ 53,815 | $ 48,475 | $ 41,753 | $ 37,544 |
Leases - Additional Information
Leases - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Lessee Lease Description [Line Items] | ||
Right of use assets | $ 45,531 | $ 91,761 |
Operating lease liabilities | $ 56,561 |
Summary of Lease Costs Recogniz
Summary of Lease Costs Recognized Under Topic 842 and Other Information Pertaining to Operating Leases (Detail) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Lease cost | |||
Operating lease cost | $ 28,737 | $ 18,978 | |
Variable lease cost | 18,742 | 17,065 | |
Total lease cost | 47,479 | 36,043 | |
Other information | |||
Operating lease payments | 24,449 | 19,344 | |
Lease liabilities arising from obtaining right of use assets | $ 13,225 | $ 59,327 | |
Weighted average remaining lease term | 3 years 6 months | 4 years 7 months 6 days | |
Weighted average discount rate | 7.60% | 7.60% |
Summary of Maturities of Lease
Summary of Maturities of Lease Liabilities and Reconciliation of Lease Liabilities Recognized Under Topic 842 (Detail) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Maturity of lease liability | ||
2022 | $ 18,702 | |
2023 | 18,326 | |
2024 | 17,582 | |
2025 | 9,052 | |
2026 | 571 | |
Thereafter | 0 | |
Total minimum lease payments | 64,233 | |
Less: imputed interest | (7,672) | |
Total operating lease liabilities | 56,561 | |
Included in the consolidated balance sheet: | ||
Current portion of lease liabilities within other current liabilities | $ 15,049 | |
Operating Lease, Liability, Current, Statement of Financial Position [Extensible List] | Other current liabilities | |
Lease liabilities | $ 41,512 | $ 80,367 |
Total operating lease liabilities | $ 56,561 |
Basic and Diluted Net Loss Per
Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 12 Months Ended | |||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | ||
Earnings Per Share [Abstract] | ||||
Net loss | $ (418,780) | $ (554,128) | $ (715,075) | |
Weighted-average common shares outstanding - basic | 81,262 | 77,956 | 73,615 | |
Effect of dilutive securities* | [1] | 0 | 0 | 0 |
Weighted-average common shares outstanding - diluted | 81,262 | 77,956 | 73,615 | |
Net loss per share — basic and diluted | $ (5.15) | $ (7.11) | $ (9.71) | |
[1] | For the years ended December 31, 2021, 2020 and 2019, stock options, RSAs, RSUs and ESPP to purchase approximately 9.7 million, 9.0 million and 9.1 million shares of common stock, respectively, were excluded from the net loss per share calculation as their effect would have been anti-dilutive. The Company accounts for the effect of the 2024 Notes on diluted net earnings per share using the if-converted method as this obligation may be settled in cash or shares at the Company’s option. The effect of potential share settlement is included in the diluted net earnings per share calculation if the effect is more dilutive. During the year ended December 31, 2021, the inclusion of the potential share settlement of the 2024 Notes was anti-dilutive. Accordingly, the potential conversion of 7,763,552 shares has been excluded from the computation of diluted net earnings per share as of December 31, 2021. |
Basic and Diluted Net Loss Pe_2
Basic and Diluted Net Loss Per Share (Parenthetical) (Detail) - $ / shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Anti-dilutive securities excluded from computation of net loss per share | 9,700,000 | 9,000,000 | 9,100,000 |
2024 Convertible Notes [Member] | |||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |||
Conversion of shares excluded from computation of diluted EPS | 7,763,552 | ||
Effect on diluted net earnings (loss) per share | $ 0 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) $ in Millions | 1 Months Ended | 12 Months Ended | |||||
Oct. 31, 2021USD ($) | Mar. 31, 2021USD ($) | Oct. 31, 2018Suite | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($)Suite | Dec. 31, 2019USD ($) | Jun. 30, 2018mo | |
CRO [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Cancellable future commitments | $ 378.5 | ||||||
CRO [Member] | Other Funding Commitments [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Expenditures incurred by CROs | $ 47.9 | $ 40.4 | $ 31.6 | ||||
Supply Agreement [Member] | Thermo Fisher Scientific, Inc. [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Number of dedicated clean room suites | mo | 4 | ||||||
Agreement expiration date | Dec. 31, 2028 | ||||||
Supply Agreement [Member] | Thermo Fisher Scientific, Inc. [Member] | Research and Development Expense [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Derecognition of right of use asset | $ 23.4 | ||||||
Derecognition of lease liabilities | 20.1 | ||||||
Recognition of loss | 3.3 | ||||||
Non refundable advance payments | $ 21.1 | ||||||
Catalent Agreements [Member] | Research and Development Expense [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Derecognition of right of use asset | $ 22.8 | ||||||
Derecognition of lease liabilities | 20 | ||||||
Recognition of loss | $ 2.8 | ||||||
Catalent Agreements [Member] | Paragon [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Number of dedicated clean room suites | Suite | 2 | 4 | |||||
Number of additional clean room suites available on optional reserve | Suite | 2 | ||||||
Agreement expiration date | Dec. 31, 2024 | ||||||
Clinical and Commercial Supply Agreement [Member] | Aldevron LLC [Member] | |||||||
Commitments And Contingencies [Line Items] | |||||||
Agreement expiration date | Dec. 31, 2026 | ||||||
Agreement expiration description | The Company has the option to extend the term of the Aldevron Agreements by one year if the Company delivers a written notice of its intention to extend to Aldevron no later than June 1, 2025. Both parties have the right to early terminate without additional penalty. The Company has determined that the Aldevron Agreements do not contain an embedded lease because it does not convey the right to control the use of Aldevron’s facility or related equipment therein. |
Summary of Non-Cancelable Contr
Summary of Non-Cancelable Contractual Obligations Arising From Long-term Contractual Arrangements (Detail) $ in Thousands | Dec. 31, 2021USD ($) |
Commitments and Contingencies Disclosure [Abstract] | |
2022 | $ 647,124 |
2023 | 178,345 |
2024 | 138,725 |
2025 | 100,838 |
2026 | 54,720 |
Thereafter | 109,440 |
Total manufacturing commitments | $ 1,229,192 |