Amicus Therapeutics (FOLD) 10-K 19 Feb 25 2024 FY Annual report Financial data

Cover

Cover - USD ($)	12 Months Ended
	Dec. 31, 2024	Feb. 10, 2025	Jun. 30, 2024
Cover [Abstract]
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2024
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity File Number	001-33497
Entity Registrant Name	Amicus Therapeutics, Inc.
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	71-0869350
Entity Address, Address Line One	47 Hulfish Street
Entity Address, City or Town	Princeton
Entity Address, State or Province	NJ
Entity Address, Postal Zip Code	08542
City Area Code	(609)
Local Phone Number	662-2000
Title of 12(b) Security	Common Stock, par value $0.01 per share
Trading Symbol	FOLD
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction	false
Entity Shell Company	false
Entity Public Float			$ 2,940,574,460
Entity Common Stock, Shares Outstanding		300,116,081
Documents Incorporated by Reference	Portions of the Proxy Statement for the registrant's 2025 Annual Meeting of Stockholders which is to be filed subsequent to the date hereof are incorporated by reference into Part III of this Annual Report on Form 10-K.
Entity Central Index Key	0001178879
Amendment Flag	false
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	FY

Audit Information

Audit Information	12 Months Ended
	Dec. 31, 2024
Audit Information [Abstract]
Auditor Firm ID	42
Auditor Name	Ernst & Young LLP
Auditor Location	Iselin, New Jersey

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 213,752	$ 246,994
Investments in marketable securities	36,194	39,206
Accounts receivable	101,099	87,632
Inventories	118,782	59,696
Prepaid expenses and other current assets	34,909	49,533
Total current assets	504,736	483,061
Operating lease right-of-use assets, net	22,278	26,312
Property and equipment, less accumulated depreciation of $28,775 and $25,429 at December 31, 2024 and 2023, respectively	29,383	31,667
Intangible assets, less accumulated amortization of $5,802 and $2,510 at December 31, 2024 and December 31, 2023, respectively	17,198	20,490
Goodwill	197,797	197,797
Other non-current assets	13,641	18,553
Total Assets	785,033	777,880
Current liabilities:
Accounts payable	12,947	15,120
Accrued expenses and other current liabilities	127,300	144,245
Operating lease liabilities	8,455	8,324
Total current liabilities	148,702	167,689
Long-term debt	390,111	387,858
Operating lease liabilities	45,078	48,877
Other non-current liabilities	7,097	13,282
Total liabilities	590,988	617,706
Commitments and contingencies
Stockholders' equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 299,041,653 and 293,594,209 shares issued and outstanding at December 31, 2024 and 2023, respectively	2,944	2,918
Additional paid-in capital	2,926,115	2,836,018
Accumulated other comprehensive gain (loss):
Foreign currency translation adjustment	5,302	5,429
Unrealized loss on available-for-sale securities	(207)	(188)
Warrants	71	71
Accumulated deficit	(2,740,180)	(2,684,074)
Total stockholders' equity	194,045	160,174
Total Liabilities and Stockholders' Equity	$ 785,033	$ 777,880

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Statement of Financial Position [Abstract]
Property and equipment, accumulated depreciation	$ 28,775	$ 25,429
Intangible assets, accumulated amortization	$ 5,802	$ 2,510
Common stock, par value (in dollars per share)	$ 0.01	$ 0.01
Common stock, shares authorized (in shares)	500,000,000	500,000,000
Common stock, shares issued (in shares)	299,041,653	293,594,209
Common stock, shares outstanding (in shares)	299,041,653	293,594,209

Consolidated Statements of Oper

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Income Statement [Abstract]
Net product sales	$ 528,295	$ 399,356	$ 329,233
Cost of goods sold	52,943	37,326	38,599
Gross profit	475,352	362,030	290,634
Operating expenses:
Research and development	109,362	152,381	276,677
Selling, general, and administrative	323,379	275,270	213,041
Changes in fair value of contingent consideration payable	0	2,583	1,078
Loss on impairment of assets	0	1,134	6,616
Restructuring charges	9,188	0	0
Depreciation and amortization	8,547	7,873	5,342
Total operating expenses	450,476	439,241	502,754
Income (loss) from operations	24,876	(77,211)	(212,120)
Other (expense) income:
Interest income	5,407	7,078	3,024
Interest expense	(49,598)	(50,149)	(37,119)
Loss on extinguishment of debt	0	(13,933)	0
Other (expense) income	(9,441)	(15,886)	4,176
Loss before income tax	(28,756)	(150,101)	(242,039)
Income tax (expense) benefit	(27,350)	(1,483)	5,471
Net loss attributable to common stockholders	$ (56,106)	$ (151,584)	$ (236,568)
Net loss attributable to common stockholders per common share - basic (in dollars per share)	$ (0.18)	$ (0.51)	$ (0.82)
Net loss attributable to common stockholders per common share - diluted (in dollars per share)	$ (0.18)	$ (0.51)	$ (0.82)
Weighted-average common shares outstanding - basic (in shares)	304,380,502	295,164,515	289,057,198
Weighted-average common shares outstanding - diluted (in shares)	304,380,502	295,164,515	289,057,198

Consolidated Statements of Comp

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Statement of Comprehensive Income [Abstract]
Net loss	$ (56,106)	$ (151,584)	$ (236,568)
Other comprehensive (loss) gain, net of tax:
Foreign currency translation adjustment (loss) gain	(127)	17,418	(17,240)
Unrealized (loss) gain on available-for-sale securities	(19)	(72)	154
Other comprehensive (loss) gain	(146)	17,346	(17,086)
Comprehensive loss	$ (56,252)	$ (134,238)	$ (253,654)

Consolidated Statements of Chan

Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Total	At-The-Market	Private Placement	Common Stock	Common Stock At-The-Market	Common Stock Private Placement	Additional Paid-In Capital	Additional Paid-In Capital At-The-Market	Additional Paid-In Capital Private Placement	Warrants	Other Comprehensive Gain (Loss)	Accumulated Deficit
Beginning balance (in shares) at Dec. 31, 2021				278,912,800
Beginning balance at Dec. 31, 2021	$ 307,369			$ 2,808			$ 2,595,419			$ 83	$ 4,981	$ (2,295,922)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	660,000			656,377
Stock options exercised, net	$ 4,310			$ 7			4,303
Vesting of restricted stock units, net of taxes (in shares)				1,539,096
Vesting of restricted stock units, net of taxes	(11,490)						(11,490)
Stock-based compensation	76,512						76,512
Unrealized (loss) gain on available-for-sale securities	154										154
Foreign currency translation adjustment	(17,240)										(17,240)
Net loss	(236,568)											(236,568)
Ending balance (in shares) at Dec. 31, 2022				281,108,273
Ending balance at Dec. 31, 2022	$ 123,047			$ 2,815			2,664,744			83	(12,105)	(2,532,490)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	1,361,000			1,357,945
Stock options exercised, net	$ 10,261			$ 14			10,247
Common stock issued (in shares)					5,244,936	2,467,104
Common stock issued		$ 63,108	$ 29,827		$ 52	$ 25		$ 63,056	$ 29,802
Vesting of restricted stock units, net of taxes (in shares)				2,195,851
Vesting of restricted stock units, net of taxes	(17,920)						(17,920)
Stock-based compensation	86,077						86,077
Warrants exercised (in shares)				1,220,100
Warrants exercised	12			$ 12			12			(12)
Unrealized (loss) gain on available-for-sale securities	(72)										(72)
Foreign currency translation adjustment	17,418										17,418
Net loss	$ (151,584)											(151,584)
Ending balance (in shares) at Dec. 31, 2023	293,594,209			293,594,209
Ending balance at Dec. 31, 2023	$ 160,174			$ 2,918			2,836,018			71	5,241	(2,684,074)
Increase (Decrease) in Stockholders' Equity
Stock options exercised, net (in shares)	957,000			962,064
Stock options exercised, net	$ 7,677			$ 9			7,668
Common stock issued (in shares)					1,732,204
Common stock issued		$ 19,566			$ 17			$ 19,549
Vesting of restricted stock units, net of taxes (in shares)				2,753,176
Vesting of restricted stock units, net of taxes	(22,025)						(22,025)
Stock-based compensation	84,905						84,905
Unrealized (loss) gain on available-for-sale securities	(19)										(19)
Foreign currency translation adjustment	(127)										(127)
Net loss	$ (56,106)											(56,106)
Ending balance (in shares) at Dec. 31, 2024	299,041,653			299,041,653
Ending balance at Dec. 31, 2024	$ 194,045			$ 2,944			$ 2,926,115			$ 71	$ 5,095	$ (2,740,180)

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Operating activities
Net loss	$ (56,106)	$ (151,584)	$ (236,568)
Adjustments to reconcile net loss to net cash used in operating activities:
Amortization of debt discount and deferred financing	2,262	2,616	2,634
Depreciation and amortization	8,547	7,873	5,342
Stock-based compensation	84,905	86,077	76,512
Loss on extinguishment of debt	0	13,933	0
Non-cash changes in the fair value of contingent consideration payable	0	2,583	1,078
Foreign currency remeasurement loss	16,234	19,613	6,121
Non-cash deferred taxes	0	(4,939)	9
Asset impairment charges and other asset write-offs	9,210	2,727	18,177
Changes in operating assets and liabilities:
Accounts receivable	(19,484)	(20,108)	(17,330)
Inventories	(73,652)	(44,614)	(5,343)
Prepaid expenses and other current assets	13,961	(8,062)	(6,194)
Accounts payable, accrued expenses, and other current liabilities	(16,520)	49,195	(6,377)
Other non-current assets and liabilities	(3,248)	(3,054)	(4,636)
Payment of contingent consideration	0	(21,347)	0
Net cash used in operating activities	(33,891)	(69,091)	(166,575)
Investing activities
Sale and redemption of marketable securities	117,779	197,227	335,926
Purchases of marketable securities	(114,786)	(91,723)	(243,255)
Capital expenditures	(3,553)	(7,440)	(3,766)
Proceeds from sale of assets	0	0	3,411
Net cash (used in) provided by investing activities	(560)	98,064	92,316
Financing activities
Proceeds from the issuance of shares in connection with at-the-market offering, net of issuance costs	19,566	63,108	0
Proceeds from equity financing, net of issuance costs	0	29,827	0
Withholding taxes paid on vested restricted stock units	(22,025)	(17,920)	(11,490)
Proceeds from stock options exercised, net	7,677	10,261	4,310
Proceeds from warrants exercised, net	0	12	0
Payment of long-term debt	0	(408,043)	0
Proceeds from long-term debt, net of issuance costs	0	387,360	0
Payment of contingent consideration	0	(2,653)	0
Payment of finance leases	(97)	(275)	(283)
Net cash provided by (used in) financing activities	5,121	61,677	(7,463)
Effect of exchange rate changes on cash, cash equivalents, and restricted cash	(4,031)	6,312	(14,619)
Net (decrease) increase in cash, cash equivalents, and restricted cash	(33,361)	96,962	(96,341)
Cash, cash equivalents, and restricted cash at the beginning of the year	250,077	153,115	249,456
Cash, cash equivalents, and restricted cash at the end of the year	216,716	250,077	153,115
Supplemental disclosures of cash flow information
Tenant improvements paid through lease incentive	315	105	0
Cash paid during the period for interest	58,960	36,090	34,358
Capital expenditures unpaid at the end of period	103	868	1,141
Cash paid for income taxes	$ 8,214	$ 8,525	$ 1,609

Description of Business

Description of Business	12 Months Ended
	Dec. 31, 2024
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Description of Business	Description of Business Amicus Therapeutics, Inc. (the "Company") is a global, patient-dedicated biotechnology company focused on discovering, developing, and delivering novel medicines for rare diseases. The Company seeks to deliver the highest quality therapies that have the potential to obsolete current treatments, provide significant benefits to patients, and be first- or best-in-class. The Company's two marketed therapies are Galafold ® , the first oral monotherapy for people living with Fabry disease who have amenable genetic variants, and Pombiliti ® + Opfolda ® , a novel two-component treatment for adults living with late-onset Pompe disease. Galafold ® (also referred to as "migalastat"), is approved in over 40 countries around the world, including the United States ("U.S."), European Union ("E.U."), United Kingdom ("U.K."), and Japan. Additionally, Galafold ® has been granted orphan drug designation in the U.S., E.U., U.K., Japan and several other countries. Pombiliti ® + Opfolda ® (also referred to as "cipaglucosidase alfa-atga/miglustat"), is approved in the U.S., the E.U., the U.K., and Switzerland. Multiple regulatory submissions and reimbursement processes with global health authorities are currently underway. Additionally, Pombiliti® + Opfolda® has been granted orphan drug designation or status in the U.S., U.K., Switzerland and Japan and data exclusivity in the E.U. The Company had an accumulated deficit of $2.7 billion as of December 31, 2024 and anticipates incurring losses into the second half of 2025. The Company has historically funded its operations through stock offerings, product revenues, debt issuances, collaborations, and other financing arrangements. Based on its current operating model, the Company believes the current cash position, which includes expected revenues, is sufficient to fund the Company's operations and ongoing research programs for at least the next 12 months. Potential business development opportunities, pipeline expansion, and investment in manufacturing capabilities could impact the Company's long-term capital requirements.

Summary of Significant Accounti

Summary of Significant Accounting Policies	12 Months Ended
	Dec. 31, 2024
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	Summary of Significant Accounting Policies Basis of Presentation The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. Certain prior year amounts have been reclassified for comparative purposes. The reclassifications did not affect results of operations, net assets or cash flows. Consolidation The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation. Foreign Currency Transactions The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other (expense) income. Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates. Cash, Cash Equivalents, Marketable Securities, and Restricted Cash The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive gain (loss) in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs. Restricted cash consists primarily of funds held to satisfy the requirements of certain agreements that are restricted in their use and is included as a component of other non-current assets on the Company's Consolidated Balance Sheets. Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities. The Company is subject to credit risk from its accounts receivable primarily related to its product sales of Galafold ® . The Company's accounts receivable at December 31, 2024 have arisen from product sales primarily in Europe, the U.S., and Japan. The Company will periodically assess the financial strength of its customers to establish allowances for anticipated losses, if any. For accounts receivable that have arisen from named patient sales, the payment terms are predetermined, and the Company evaluates the creditworthiness of each customer on a regular basis. As of December 31, 2024, the Company's allowance for doubtful accounts was $3.0 million. Property and Equipment Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are expensed as incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy. The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the long-lived asset over its estimated fair value in the period at which such a determination is made. Revenue Recognition The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2024, one customer accounted for 26% of net product sales and 18% of accounts receivable from product sales while another customer accounted for 13% of accounts receivable from product sales. In 2023, one customer accounted for 28% of net product sales and 16% of accounts receivable from product sales while another customer accounted for 17% of accounts receivable from product sales. Revenue attributed to U.K. and Germany each accounted for 13% of net product sales in 2024, 10% and 13% respectively in 2023, and 12% each in 2022. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes. The following table summarizes the Company's net product sales disaggregated by product and geographic area: For the Year (in thousands) 2024 2023 2022 Galafold ® U.S. $ 176,024 $ 145,147 $ 115,759 Galafold ® Ex-U.S. 282,030 242,630 213,287 Total Galafold ® sales $ 458,054 $ 387,777 $ 329,046 Pombiliti ® + Opfolda ® U.S. $ 30,123 1,791 — Pombiliti ® + Opfolda ® Ex-U.S. 40,118 9,788 187 Total Pombiliti ® + Opfolda ® sales $ 70,241 $ 11,579 $ 187 Total net product sales $ 528,295 $ 399,356 $ 329,233 Inventories and Cost of Goods Sold Until regulatory approval of Pombiliti ® + Opfolda ® , the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Pombiliti ® + Opfolda ® . Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of inventory available for sale was expensed as research and development costs prior to regulatory approval and, as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin. Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the Financial Accounting Standard Board ("FASB") guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participant's assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability. Research and Development Costs Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development. Interest Income and Interest Expense Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases. Income Taxes The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized. The Company’s tax returns are subject to examination by U.S. Federal, state, and foreign taxing jurisdictions. The impact of an uncertain tax position taken or expected to be taken on an income tax return must be recognized in the financial statements at the largest amount that is more likely than not to be sustained. An uncertain income tax position will not be recognized in the financial statements unless it is more likely than not to be sustained. Other Comprehensive Gain (Loss) Components of other comprehensive gain (loss) include unrealized gains and losses on available-for-sale securities and gains and losses on foreign currency transactions, and are included in the Consolidated Statements of Comprehensive Loss. Leases The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets. ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. 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. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories. The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories. Nonqualified Cash Deferral Plan The Company's Cash Deferral Plan (the "Deferral Plan") provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code. All of the investments held in the Deferral Plan are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets. Equity-based Compensation At December 31, 2024, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 8. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award. Loss per Common Share The Company calculates net loss per share as a measurement of the Company's performance while giving effect to all dilutive potential common shares that were outstanding during the reporting period. The Company had a net loss for all periods presented; accordingly, the inclusion of common stock options and unvested restricted stock units would be anti-dilutive. Therefore, the weighted average shares used to calculate both basic and diluted earnings per share are the same. Segment Information The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker ("CODM"), its Chief Executive Officer, who comprehensively manages the entire business. The Company and its CODM evaluate performance and allocate resources primarily based on Net product sales and Net loss within the Consolidated Statement of Operations he is regularly provided. Net loss is used to monitor budget to actual results and actuals against prior periods. The Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reportable segment. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Consolidated Statements of Operations and Consolidated Balance Sheets provide information regarding significant segment expenses and segment assets. Revenue segregation by product line is presented in the revenue recognition section earlier in this footnote. Intangible Assets and Goodwill The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is concluded. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference. Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made. No indicators of impairment were noted during the years ended December 31, 2024 and 2023. Recent Accounting Developments - Guidance Adopted in 2024 In November 2023, the FASB issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. Recent Accounting Developments - Guidance Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements. In November 2024, the FASB issued ASU 2024-03, "Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses." The standard is intended to require more detailed disclosures about specified categories of expenses (including employee compensation, depreciation, and amortization) included in certain expense captions presented on the face of the income statement. This ASU is effective for fiscal years beginning after December 15, 2026, and for interim periods within fiscal years beginning after December 15, 2027. Early adoption is permitted. The amendments may be applied either prospectively to financial statements issued for reporting periods after the effective date of this ASU or retrospectively to all prior periods presented in the financial statements. The Company is currently assessing the impact this standard will have on its consolidated financial statements.

Goodwill and Intangible Assets

Goodwill and Intangible Assets	12 Months Ended
	Dec. 31, 2024
Goodwill and Intangible Assets Disclosure [Abstract]
Goodwill and Intangible Assets	Goodwill and Intangible Assets Finite-lived Intangible Assets As of December 31, 2024, the Company had intangible assets of $17.2 million. Intangible assets consist of lead enzyme replacement therapy assets acquired with the Callidus Biopharma, Inc. ("Callidus") acquisition in 2013, previously accounted for as in-process research and development. In March 2023, as a result of the European Commission's ("EC") approval of Pombiliti ® , the Company began amortizing the assets over the initial regulatory exclusivity period of 7 years. The Company completed an impairment assessment before changing the classification to finite-lived intangible asset noting no impairment. Amortization expense for finite-lived intangible assets was $3.3 million and $2.5 million for the years ended December 31, 2024 and 2023 respectively. Total estimated amortization expense for the finite-lived intangible assets for each of the next 5 years is approximately $3.3 million and has a remaining amortization period of 5.2 years. Goodwill

Cash, Cash Equivalents, Marketa

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash	12 Months Ended
	Dec. 31, 2024
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash	Cash, Cash Equivalents, Marketable Securities, and Restricted Cash As of December 31, 2024, the Company held $213.8 million in cash and cash equivalents and $36.2 million of marketable securities which are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are generally reported within other comprehensive gain (loss) in the Consolidated Statements of Comprehensive Loss. If a decline in the fair value of a marketable security below the Company's cost basis is determined to be other-than-temporary or if an available-for-sale debt security’s fair value is determined to be less than the amortized cost and the Company intends or is more than likely to sell the security before recovery and it is not considered a credit loss, such security is written down to its estimated fair value as a new cost basis and the amount of the write-down is included in the Consolidated Statements of Operations as an impairment charge. If the unrealized loss of an available-for-sale debt security is determined to be a result of credit loss the Company would recognize an allowance and the corresponding credit loss would be included in the Consolidated Statements of Operations. The Company regularly invests excess operating cash in deposits with major financial institutions, money market funds, notes issued by the U.S. government, as well as fixed income investments and U.S. bond funds, both of which can be readily purchased and sold using established markets. The Company believes that the market risk arising from its holdings of these financial instruments is mitigated as, in accordance with Company policy, securities are of high credit rating. Investments that have original maturities greater than three months but less than one year are classified as current. All marketable securities represent the investment of funds available for current operations, notwithstanding their contractual maturities. Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following: As of December 31, 2024 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 213,752 $ — $ — $ 213,752 Commercial paper 18,082 3 (4) 18,081 U.S. government agency bonds 16,524 1 (7) 16,518 Corporate debt securities 1,446 — (2) 1,444 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 249,955 $ 4 $ (13) $ 249,946 Included in cash and cash equivalents $ 213,752 $ — $ — $ 213,752 Included in marketable securities 36,203 4 (13) 36,194 Total cash, cash equivalents, and marketable securities $ 249,955 $ 4 $ (13) $ 249,946 As of December 31, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Commercial paper 14,651 12 — 14,663 Treasury bill 12,944 2 — 12,946 U.S. government agency bonds 11,450 — (4) 11,446 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 286,190 $ 14 $ (4) $ 286,200 Included in cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Included in marketable securities 39,196 14 (4) 39,206 Total cash, cash equivalents, and marketable securities $ 286,190 $ 14 $ (4) $ 286,200 For the years ended December 31, 2024 and 2023, there were no realized gains or losses. The cost of securities sold is based on the specific identification method. Unrealized loss positions in the marketable securities as of December 31, 2024 reflect temporary impairments and are not a result of credit loss. Additionally, as these positions have been in a loss position for less than twelve months and the Company does not intend to sell these securities before recovery, the losses are recognized in other comprehensive gain (loss) . The fair value of these marketable securities in unrealized loss positions are $23.7 million and $11.4 million as of December 31, 2024 and 2023 respectively. The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows. December 31, (in thousands) 2024 2023 2022 Cash and cash equivalents $ 213,752 $ 246,994 $ 148,813 Restricted cash 2,964 3,083 4,302 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 216,716 $ 250,077 $ 153,115

Inventories

Inventories	12 Months Ended
	Dec. 31, 2024
Inventory Disclosure [Abstract]
Inventories	Inventories Inventories consisted of the following: December 31, (in thousands) 2024 2023 Raw materials $ 87,916 $ 30,230 Work-in-process 21,223 22,597 Finished goods 9,643 6,869 Total inventories $ 118,782 $ 59,696 The Company's reserve for inventory was $3.3 million and $0.5 million as of December 31, 2024 and 2023, respectively. The Company has a number of binding minimum purchase and manufacturing commitments due to the third-party manufacturers. As of December 31, 2024, these purchase and manufacturing obligations totaled $179.7 million, of which $102.7 million and $77.0 million are expected in 2025 and 2026, respectively.

Property and Equipment

Property and Equipment	12 Months Ended
	Dec. 31, 2024
Property, Plant and Equipment [Abstract]
Property and Equipment	Property and Equipment Property and equipment consist of the following: December 31, (in thousands) 2024 2023 Leasehold improvements $ 32,361 $ 28,032 Research equipment 16,220 16,220 Computer equipment 5,198 4,934 Furniture and fixtures 3,715 2,928 Computer software 402 1,106 Construction in progress 262 3,810 Vehicles — 66 Gross property and equipment 58,158 57,096 Less accumulated depreciation (28,775) (25,429) Net property and equipment $ 29,383 $ 31,667

Accrued Expenses and Other Curr

Accrued Expenses and Other Current Liabilities	12 Months Ended
	Dec. 31, 2024
Payables and Accruals [Abstract]
Accrued Expenses and Other Current Liabilities	Accrued Expenses and Other Current Liabilities Accrued expenses and other current liabilities consist of the following: December 31, (in thousands) 2024 2023 Accrued sales rebates and discounts $ 33,546 $ 31,190 Accrued compensation and benefits 32,529 38,305 Accrued taxes 18,105 12,434 Accrued program fees 10,578 13,607 Accrued royalties 9,790 8,238 Accrued professional fees 6,337 5,059 Accrued contract manufacturing & contract research costs 3,924 13,380 Accrued restructuring 3,665 — Accrued interest on debt 120 11,661 Other 8,706 10,371 $ 127,300 $ 144,245

Restructuring

Restructuring	12 Months Ended
	Dec. 31, 2024
Restructuring and Related Activities [Abstract]
Restructuring	Restructuring Restructuring charges are primarily related to an initiative to reduce operating costs by abandoning one of the Company's leases that it no longer believes is necessary to conduct its operations. The restructuring liabilities are recorded in accrued expenses and other current liabilities in the Company’s Consolidated Balance Sheets. Total charges incurred are summarized as follows: (in thousands) Facility and Non-Lease Costs Other Costs Total Balance as of December 31, 2023 $ — $ — $ — Restructuring charges 6,045 3,143 9,188 Non-cash items (2,844) (2,844) Cash settled (1,025) (1,654) (2,679) Balance as of December 31, 2024 $ 2,176 $ 1,489 $ 3,665

Stockholders' Equity

Stockholders' Equity	12 Months Ended
	Dec. 31, 2024
Equity [Abstract]
Stockholders' Equity	Stockholders' Equity Common Stock and Warrants As of December 31, 2024, the Company was authorized to issue 500 million shares of common stock. Dividends on common stock will be paid when, and if, declared by the Board of Directors. Each holder of common stock is entitled to vote on all matters that are appropriate for stockholder voting and is entitled to one vote for each share held. In November 2022, the Company entered into a Sales Agreement with Goldman Sachs & Co. LLC to create an at-the-market equity program ("ATM program"), pursuant to which the Company may offer to sell shares of its common stock having an aggregate offering gross proceeds of up to $250.0 million. During the year ended December 31, 2024, the Company issued and sold an aggregate of 1,732,204 shares through its ATM program at a weighted-average public offering price of $11.69 per share, resulting in net proceeds of $19.6 million. During the year ended December 31, 2023, the Company issued and sold an aggregate of 5,244,936 shares through its ATM program at a weighted-average public offering price of $12.50 per share, resulting in net proceeds of $63.1 million. As of December 31, 2024, an aggregate of $164.2 million worth of shares remain available to be issued and sold under the ATM program. In October 2023, the Company entered into a securities purchase agreement with funds managed by Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”), for the private placement of an aggregate of 2,467,104 shares of the Company’s common stock, at a purchase price of $12.16 per share. Proceeds from the private placement, net of offering costs, were $29.8 million. Nonqualified Cash Deferral Plan The Company's Deferral Plan provides certain key employees and members of the Board of Directors, as selected by the Compensation Committee, with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code of 1986 as amended. The Company had a deferred compensation investment balance of $2.2 million and $7.5 million as of December 31, 2024 and 2023, respectively, with corresponding approximate amounts of liability. Deferral Plan investment assets are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. Deferred compensation liability amounts under the Deferral Plan are included in other long-term liabilities. Equity Incentive Plan The Company's Amended and Restated 2007 Equity Incentive Plan (the "Plan") provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company's Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company's business. Under the provisions of the Plan, no option will have a term in excess of 10 years. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. Options granted pursuant to the Plan generally vest 25% on the first year anniversary date of grant plus an additional 1/48th for each month thereafter and may be exercised in whole or in part for 100% of the shares vested at any time after the date of grant. As of December 31, 2024, the Company has reserved up to 14,228,754 shares for issuance under the Plan.

Stock based Compensation

Stock based Compensation	12 Months Ended
	Dec. 31, 2024
Share-Based Payment Arrangement [Abstract]
Stock based Compensation	Stock-based Compensation The Plan provides for the granting of restricted stock units and options to purchase common stock in the Company to employees, directors, advisors, and consultants at a price to be determined by the Company’s Board of Directors. The Plan is intended to encourage ownership of stock by employees and consultants of the Company and to provide additional incentives for them to promote the success of the Company’s business. The Board of Directors, or its committee, is responsible for determining the individuals to be granted options, the number of options each individual will receive, the option price per share, and the exercise period of each option. The Plan provides for certain benefits to qualifying Plan participants who separate from service with the Company due to death, disability or "retirement" (as such term is defined under the Plan) ("Qualified Participants"). Options granted under the Plan to a Qualified Participant shall continue to vest until the 2nd anniversary of the Qualified Participant’s separation and all vested options held by such Qualified Participant shall remain exercisable until the earlier of the 4th anniversary of the Qualified Participant’s separation or the original expiration date of the option. Options that are not exercised during this exercise period shall be forfeited. Time-based restricted stock units and restricted stock granted to a Qualified Participant under the Plan that was scheduled to vest within the two-year period following the Qualified Participant's separation shall accelerate and be delivered upon such separation. Any time-based restricted stock units or restricted stock that would have vested after such two-year period will be forfeited upon the Qualified Participant's separation. Also, per the Amendment, any performance-based restricted stock units under the Plan ("PRSUs") received by the Qualified Participant, shall remain eligible to vest after the Qualified Participant’s separation based on the actual performance of the Company through the end of the performance period applicable to any such PRSUs. Stock Option Grants The Company uses the fair value method of measuring share-based compensation, using the fair value of each equity award granted. The Company chose the ‘‘straight-line’’ attribution method for allocating compensation costs and recognized the fair value of each stock option on a straight-line basis over the vesting period of the related awards. The Company uses the Black-Scholes option pricing model when estimating the grant date fair value for share-based awards. Use of a valuation model requires management to make certain assumptions with respect to selected model inputs. Expected volatility is based on the historical volatility of the Company's common stock over the look-back period corresponding to the expected life of the options. The average expected life is determined using the Company's actual historical data. The risk-free interest rate is based on U.S. Treasury, zero-coupon issues with a remaining term equal to the expected life assumed at the date of grant. Forfeitures are estimated based on a historical analysis of actual option forfeitures. The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Years ended December 31, 2024 2023 2022 Expected stock price volatility 57.0 % 59.2 % 62.1 % Risk free interest rate 4.0 % 3.9 % 1.7 % Expected life of options (years) 5.60 5.47 5.34 Expected annual dividend per share $ — $ — $ — The weighted average grant-date fair value per share of options granted during 2024, 2023, and 2022 were $7.50, $6.75, and $6.11, respectively. A summary of the Company's stock options is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Options outstanding, December 31, 2021 14,731 $ 11.08 Granted 5,733 $ 11.53 Exercised (660) $ 6.58 Forfeited (495) $ 12.57 Expired (245) $ 12.84 Options outstanding, December 31, 2022 19,064 $ 11.31 Granted 5,733 $ 11.99 Exercised (1,361) $ 7.54 Forfeited (356) $ 11.59 Expired (78) $ 15.09 Options outstanding, December 31, 2023 23,002 $ 11.69 Granted 4,917 $ 13.52 Exercised (957) $ 8.02 Forfeited (622) $ 12.75 Expired (327) $ 14.30 Options outstanding, December 31, 2024 26,013 $ 12.11 5.8 $ 5.8 Vested and unvested expected to vest, December 31, 2024 24,643 $ 12.09 5.7 $ 5.8 Exercisable at December 31, 2024 17,464 $ 11.80 4.6 $ 5.7 The aggregate intrinsic value of options exercised during the years ended December 31, 2024, 2023 and 2022 was $3.8 million, $7.1 million, and $2.5 million, respectively. Cash proceeds from stock options exercised during the years ended December 31, 2024, 2023, and 2022 were $7.7 million, $10.3 million, and $4.3 million, respectively. As of December 31, 2024, the total unrecognized compensation cost related to non-vested stock options granted was $38.6 million and is expected to be recognized over a weighted average period of two years. Restricted Stock Units and Performance-Based Restricted Stock Units (collectively "RSUs") RSUs awarded under the Plan are generally subject to graded vesting and are contingent on an employee's continued service. RSUs are generally subject to forfeiture if employment terminates prior to the release of vesting restrictions. The Company expenses the cost of the RSUs, which is determined to be the fair market value of the shares of common stock underlying the RSUs at the date of grant, ratably over the period during which the vesting restrictions lapse. A summary of non-vested RSU activity under the Plan is as follows: Number of Weighted Average Grant Date Fair Value Weighted Average Remaining Years Aggregate Intrinsic Value (in thousands) (in millions) Non-vested units as of December 31, 2021 7,341 $ 13.90 Granted 5,048 $ 11.93 Vested (2,251) $ 12.48 Forfeited (421) $ 12.06 Non-vested units as of December 31, 2022 9,717 $ 13.07 Granted 4,762 $ 13.02 Vested (3,610) $ 12.21 Forfeited (836) $ 11.63 Non-vested units as of December 31, 2023 10,033 $ 13.37 Granted 4,150 $ 14.32 Vested (5,107) $ 13.07 Forfeited (799) $ 13.74 Non-vested units as of December 31, 2024 8,277 $ 13.59 2.1 $ 78.0 As of December 31, 2024, there was $55.1 million of total unrecognized compensation cost related to unvested RSUs with service-based vesting conditions. These costs are expected to be recognized over a weighted average period of two years. Compensation Expense Related to Equity Awards The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards: Years ended December 31, (in thousands) 2024 2023 2022 Research and development expense $ 15,969 $ 21,470 $ 25,089 Selling, general, and administrative expense 68,936 64,607 51,423 Total equity compensation expense $ 84,905 $ 86,077 $ 76,512

Assets and Liabilities Measured

Assets and Liabilities Measured at Fair Value	12 Months Ended
	Dec. 31, 2024
Fair Value Disclosures [Abstract]
Assets and Liabilities Measured at Fair Value	Assets and Liabilities Measured at Fair Value The Company's financial assets and liabilities are measured at fair value and classified within the fair value hierarchy which is defined as follows: Level 1 — Quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date. Level 2 — Inputs other than quoted prices in active markets that are observable for the asset or liability, either directly or indirectly. Level 3 — Inputs that are unobservable for the asset or liability. A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2024 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 18,081 $ 18,081 U.S. government agency bonds — 16,518 16,518 Corporate debt securities — 1,444 1,444 Money market 2,318 — 2,318 $ 2,318 $ 36,043 $ 38,361 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability $ 2,218 $ — $ 2,218 $ 2,218 $ — $ 2,218 A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2023 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 14,663 $ 14,663 Treasury bill — 12,946 12,946 U.S. government agency bonds — 11,446 11,446 Money market 7,631 — 7,631 $ 7,631 $ 39,055 $ 46,686 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability 7,531 — 7,531 $ 7,531 $ — $ 7,531 The Company's Senior Secured Term Loan due 2029 falls into the Level 2 category within the fair value level hierarchy and the fair value was determined using a discounted cash flow analysis that factors in current market yields for comparable borrowing arrangements under the Company's credit profile. The carrying value of the Senior Secured Term Loan due 2029 approximates the fair value. Deferred compensation plan liability is recorded as a component of other non-current liabilities on the Company's Consolidated Balance Sheets. The Company did not have any Level 3 assets or liabilities as of December 31, 2024 or 2023. Cash, Money Market Funds and Marketable Securities The Company classifies its cash and cash equivalents within the fair value hierarchy as Level 1 as these assets are valued using quoted prices in an active market for identical assets at the measurement date. The Company considers its investments in marketable securities as available-for-sale and classifies these assets within the fair value hierarchy as Level 2 primarily utilizing broker quotes in a non-active market for valuation of these securities. No changes in valuation techniques or inputs occurred during the year ended December 31, 2024. No transfers of assets between Level 1 and Level 2 of the fair value measurement hierarchy occurred during the year ended December 31, 2024.

Debt

Debt	12 Months Ended
	Dec. 31, 2024
Debt Disclosure [Abstract]
Debt	Debt The Company's debt consists of the following: As of December 31, (in thousands) 2024 2023 Senior Secured Term Loan due 2029: Principal $ 400,000 $ 400,000 Less: debt discount (1) (7,863) (9,652) Less: deferred financing (1) (2,026) (2,490) Net carrying value of long-term debt $ 390,111 $ 387,858 _____________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the corresponding Senior Secured Term Loan using the effective interest rate method. Senior Secured Term Loan due 2029 In October 2023, the Company entered into a $400 million loan agreement (the “Senior Secured Term Loan due 2029”) with Blackstone Alternative Credit Advisors LP and Blackstone Life Sciences Advisors L.L.C. (collectively, “Blackstone”) with an interest rate equal to 3-month Term SOFR, subject to a 2.5% floor, plus a Term SOFR adjustment of 0.26161% and a margin of 6.25% that requires interest-only payments until early-2027. The Senior Secured Term Loan due 2029 will be repaid in twelve quarterly payments of $33.3 million, starting on January 2027 with the final balance due on the maturity date in October 2029. This transaction resulted in net proceeds of $387.4 million, after deducting fees and expenses. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2029. The Senior Secured Term Loan due 2029 is subject to mandatory prepayment provisions that require prepayment upon a change of control, the incurrence of certain additional indebtedness, asset sale, or an event of loss, subject to certain conditions set forth in the Senior Secured Term Loan due 2029. The Company may prepay the Senior Secured Term Loan due 2029 in whole or in part, at its option at any time. Any prepayment of the Senior Secured Term Loan due 2029 is subject to certain make-whole premiums and prepayment premiums, the latter of which decrease until the fifth anniversary of the transaction date at which point no prepayment penalty shall exist. The obligations under the Senior Secured Term Loan due 2029 are secured by a first lien security interest in certain assets of the Company. The Senior Secured Term Loan due 2029 contains certain customary representations and warranties, affirmative and negative covenants and events of default applicable to the Company. If an event of default occurs and is continuing, Blackstone may declare all amounts outstanding under the Senior Secured Term Loan due 2029 to be immediately due and payable. Senior Secured Term Loan due 2026 In July 2020, the Company entered into a definitive agreement for a $400 million credit facility with Hayfin Capital Management (“Senior Secured Term Loan due 2026”) with an interest rate equal to 3-month LIBOR, subject to a 1% floor, plus 6.5% per annum and requires interest-only payments until mid-2024. The Senior Secured Term Loan due 2026 was to be repaid in nine quarterly payments of $44.4 million, starting on July 2024 with the final balance due on the maturity date in July 2026. There were no warrants or equity conversion features associated with the Senior Secured Term Loan due 2026. In October 2023, the Company used $408.9 million of the net proceeds from the Senior Secured Term Loan due 2029 and the private placement to prepay the Senior Secured Term Loan due 2026, inclusive of the outstanding principal amount, $0.8 million in accrued interest and $8.0 million as a prepayment premium. In connection with the prepayment, the Company recorded a loss from early extinguishment of debt of $13.9 million in the Company's Consolidated Statements of Operations for the year ended December 31, 2023. Interest Expense The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2024 and 2023, respectively: December 31, (in thousands, except interest rate amounts) 2024 2023 Contractual interest expense $ 47,373 $ 47,626 Amortization of debt discount $ 1,790 $ 1,605 Amortization of deferred financing $ 472 $ 1,011 Effective interest rate of the liability component, Senior Secured Term Loan due 2029 12.0 % 12.8 %

Leases

Leases	12 Months Ended
	Dec. 31, 2024
Leases [Abstract]
Leases	Leases The Company currently has operating leases for office and research laboratory space, equipment, and vehicles under agreements expiring at various dates through 2034, which include renewal options on leases which the Company is reasonably certain to exercise. For the years ended December 31, 2024 and 2023, operating lease expense was $6.7 million and $9.5 million and variable lease expense was $2.2 million and $2.1 million, respectively. For the years ended December 31, 2024 and 2023, the Company paid $9.6 million and $9.5 million, respectively, for amounts included in the measurement of operating lease liabilities and recorded right-of-use assets of $0.7 million and $0.6 million, respectively. Commitments under finance leases are not significant for the year ended December 31, 2024. Supplemental balance sheet information related to operating leases were as follows: December 31, (in thousands, except year and discount rate amounts) 2024 2023 Operating lease ROU assets, net $ 22,278 $ 26,312 Current portion of the operating lease liabilities $ 8,455 $ 8,324 Non-current portion of the operating lease liabilities 45,078 48,877 Total operating lease liability $ 53,533 $ 57,201 Weighted-average remaining lease terms (years) 8.5 9.4 Weighted-average discount rate 9.7 % 9.7 % At December 31, 2024, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2025 $ 9,466 2026 9,356 2027 9,426 2028 9,139 2029 8,172 Thereafter 35,402 Total lease payments 80,961 Less: lease incentives (22,299) Less: imputed interest (5,129) Total operating lease liability $ 53,533

Income Taxes

Income Taxes	12 Months Ended
	Dec. 31, 2024
Income Tax Disclosure [Abstract]
Income Taxes	Income Taxes For financial reporting purposes, income (loss) before income taxes includes the following components: Years ended December 31, (in thousands) 2024 2023 2022 United States $ (210,049) $ (234,482) $ (343,424) Foreign 181,293 84,381 101,385 Total $ (28,756) $ (150,101) $ (242,039) Following were the components of income tax expense (benefit) for the years ended December 31, 2024, 2023, and 2022: Years ended December 31, (in thousands) 2024 2023 2022 Current Federal $ — $ — $ — State 76 14 6 Foreign 27,274 6,408 (5,760) Deferred Federal — (4,801) 274 State — (138) 9 Foreign — — — Total $ 27,350 $ 1,483 $ (5,471) A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2024, 2023, and 2022 are as follows: Years ended December 31, 2024 2023 2022 Statutory rate (21) % (21) % (21) % Tax credits (14) (8) (11) Impact of foreign operations 180 (9) 18 Executive compensation 36 7 3 Other 63 1 — Valuation allowance (149) 31 9 Net 95 % 1 % (2) % On December 22, 2017, the U.S. government enacted the Tax Cuts and Jobs Act ("Tax Act"). The Tax Act significantly revised U.S. tax law by, among other provisions, lowering the U.S. federal statutory income tax rate to 21%, imposing a mandatory one-time transition tax on previously deferred foreign earnings, and eliminating or reducing certain income tax deductions. The Tax Act also introduced an additional U.S. tax on certain non-U.S. subsidiaries’ earnings which are considered to be Global Intangible Low Taxed Income (referred to as "GILTI"). After consideration of the relevant guidance and completing the accounting for the tax effects of the Tax Act, the Company has elected to treat GILTI as a period cost. Beginning in 2022, the Tax Act eliminated the right to deduct research and development expenditures for tax purposes in the period the expenses were incurred and instead requires all U.S. and foreign research and development expenditures to be amortized over five and fifteen tax years, respectively. Tax returns for years 2019 through 2024 are open to examination by tax authorities. The Company is also subject to examination in any period for which it has net operating losses. A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows: Years ended December 31, (in thousands) 2024 2023 2022 Gross unrecognized tax benefits at beginning of year $ 4,888 $ 283 $ — Gross increases for tax positions in prior years 9,542 4,605 283 Decreases for settlements and payments (625) — — Gross unrecognized tax benefits at end of year $ 13,805 $ 4,888 $ 283 For the years ended December 31, 2024 and 2023, approximately $13.8 million and $4.8 million of unrecognized tax benefits respectively, would, if recognized, impact the Company's effective tax rate. The Company recognizes accrued interest related to its uncertain tax positions as part of its income tax expense within its consolidated statement of operations. The amount of accrued interest was $1.9 million for the year ended December 31, 2024. The Company's management determined that no accrual for penalties was required as of December 31, 2024. Deferred income taxes reflect the net effect of temporary difference between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. The significant components of the deferred tax assets and liabilities are as follows: Years ended December 31, (in thousands) 2024 2023 Deferred tax assets Net operating loss carry forwards 302,066 321,004 Research tax credit 234,189 235,532 Capitalized research and development costs 34,883 34,425 Share-based compensation 20,045 17,314 Lease liability 10,026 10,567 Interest carry forward limitation 9,097 17,627 Inventory 1,599 1,931 Other 5,267 20,858 Gross deferred tax assets 617,172 659,258 Deferred tax liabilities Other (8,042) (10,004) Total net deferred tax assets 609,130 649,254 Less: valuation allowance (609,130) (649,254) Net deferred tax liability $ — $ — The Company records a valuation allowance for temporary differences for which it is more likely than not that the Company will not receive future tax benefits. At December 31, 2024 and 2023, the Company recorded valuation allowances of $609.1 million and $649.3 million, respectively, representing a decrease in the valuation allowance of $40.2 million in 2024, due to the uncertainty regarding the realization of such deferred tax assets, to offset the benefits of net operating losses generated during those years. The deferred tax liability related to business acquisitions pertains to the basis difference in intangible assets acquired by the Company. The Company's policy is to record a deferred tax liability related to acquired intangible assets that may eventually be realized either upon amortization of the asset when the research is completed, and a product is successfully launched or the write-off of the asset if it is abandoned or unsuccessful. As of December 31, 2024, the Company had U.S. federal and state net operating loss carry forwards ("NOLs") of approximately $1.1 billion and $1.0 billion, respectively. The federal carry forward for losses generated prior to 2018 will expire in 2031 through 2037. Federal net operating losses incurred in 2018 and onward have an indefinite expiration under the Tax Act. Most of the state net operating loss carry forwards generated prior to 2009 have expired through 2016. The remaining state net operating loss carry forwards including those generated in 2009 through 2024 will expire in 2028 through 2042. State research and development credits will expire 2024 through 2033. Utilization of NOLs may be subject to a substantial limitation pursuant to Section 382 of the Internal Revenue Code of 1986, as amended ("Section 382"), as well as similar state statutes in the event of an ownership change. Such ownership changes have occurred in the past and could occur again in the future. Under Section 382, if a corporation undergoes an "ownership change," generally defined as a greater than 50% change (by value) in its equity ownership over a three-year period, the corporation's ability to use its pre-change NOLs and other pre-change tax attributes (such as research and development tax credits) to offset its post-change income may be limited. The Company may experience ownership changes in the future as a result of shifts in the stock ownership some of which are outside the Company's control. The Company completed a detailed study of the NOLs for the tax year 2024 and determined that there was not an ownership change in excess of 50%. Ownership changes in future periods may place additional limits on the Company's ability to utilize net operating loss and tax credit carry forwards. In addition, at the state level, there may be periods during which the use of NOLs is suspended or otherwise limited, which could accelerate or permanently decrease the amount of state attributes and increase state taxes owed. The Company also has U.S. federal research and experimentation and orphan drug credit carryforwards of approximately $19.1 million and $201.6 million, respectively, which will expire in the years 2030 through 2043. The Company also has state research and development tax credit carryforwards of $13.5 million. Deferred tax assets for these carryforwards are subject to a full valuation allowance. The Company permanently reinvests the earnings of its foreign subsidiaries except for its U.K. operations. The determination of the amount of the unrecognized of the deferred tax liability related to the undistributed earnings is not practicable because of the complexities associated with its hypothetical calculation.

Collaborative Agreements

Collaborative Agreements	12 Months Ended
	Dec. 31, 2024
Collaborative Agreements
Collaborative Agreements	Collaborative Agreements GlaxoSmithKline In July 2012, as amended in November 2013, the Company entered into an agreement with GlaxoSmithKline ("GSK"), pursuant to which Amicus obtained global rights to develop and commercialize Galafold ® as a monotherapy and in combination with ERT for Fabry disease (“Collaboration Agreement”). Under the terms of the Collaboration Agreement, GSK is eligible to receive post-approval and sales-based milestones up to $40 million, as well as tiered royalties in the mid-teens in eight major markets outside the U.S. As of December 31, 2024, the contingent milestone payments due to GSK were $4.7 million and are recorded within the other current and other non-current liabilities accounts on the Consolidated Balance Sheets. Sales based tiered royalties due to GSK are recorded within the cost of goods sold within the Consolidated Statements of Operations. For the year ended December 31, 2024, under the GSK collaboration agreement, the Company incurred approximately $32.7 million of royalty expenses, of which $9.8 million is recorded within accrued expenses in the Consolidated Balance Sheets.

Legal Proceedings

Legal Proceedings	12 Months Ended
	Dec. 31, 2024
Commitments and Contingencies Disclosure [Abstract]
Legal Proceedings	Legal Proceedings In the fourth quarter of 2022, the Company received Paragraph IV Certification Notice Letters from Teva Pharmaceuticals USA, Inc. ("Teva"), Aurobindo Pharma Limited ("Aurobindo"), and Lupin Limited ("Lupin") in connection with Abbreviated New Drug Applications (“ANDA”) filed with the FDA requesting approval to market generic Galafold®. In November 2022, the Company filed four lawsuits against Teva, Lupin, and Aurobindo in the U.S. District Court for the District of Delaware (the "Court") for infringement of its Orange Book-listed patents. In the fourth quarter of 2023, a stipulation order to stay litigation with respect to Lupin was ordered. Additionally, in the first quarter of 2024, a stipulation was filed with the court and approved by the presiding judge, whereby the parties agreed to accept the Company’s definition of the terms that were in dispute. As such, the scheduled Markman hearing was deemed unneeded and cancelled. In October 2024, the Company entered into a non-exclusive, non-transferable, royalty-free, fully paid-up license with Teva which will allow Teva to market its generic version of Galafold ® in the United States beginning on January 30, 2037, or earlier in certain circumstances. In accordance with the license agreement, a consent judgment and permanent injunction was entered with the Court and all Hatch-Waxman litigation between Amicus and Teva has been terminated. As required by law, Amicus and Teva have submitted the confidential license agreement to the U.S. Federal Trade Commission and the U.S. Department of Justice for review. The litigation will continue against Aurobindo as the remaining active party, and the litigation stay remains in place for Lupin. The Company has, and will continue to, vigorously enforce its Galafold ® intellectual property rights.

Basic and Diluted Net Loss per

Basic and Diluted Net Loss per Common Share	12 Months Ended
	Dec. 31, 2024
Earnings Per Share [Abstract]
Basic and Diluted Net Loss per Common Share	Basic and Diluted Net Loss per Common Share The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share: Years ended December 31, (in thousands, except per share amounts) 2024 2023 2022 Numerator: Net loss attributable to common stockholders $ (56,106) $ (151,584) $ (236,568) Denominator: Weighted average common shares outstanding — basic and diluted 304,380,502 295,164,515 289,057,198 Dilutive common stock equivalents would include the dilutive effect of outstanding common stock options and unvested RSUs. Potentially dilutive common stock equivalents were excluded from the diluted earnings per share denominator for all periods because of their anti-dilutive effect. Weighted average common shares outstanding includes outstanding pre-funded warrants with an exercise price of $0.01. The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method: Years ended December 31, (in thousands) 2024 2023 2022 Options to purchase common stock 26,013 23,002 19,064 Unvested restricted stock units 8,277 10,033 9,717 Total number of potentially issuable shares 34,290 33,035 28,781

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Pay vs Performance Disclosure
Net loss	$ (56,106)	$ (151,584)	$ (236,568)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended
	Dec. 31, 2024
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

Insider Trading Policies and Pr

Insider Trading Policies and Procedures	12 Months Ended
	Dec. 31, 2024
Insider Trading Policies and Procedures [Line Items]
Insider Trading Policies and Procedures Adopted	true

Cybersecurity Risk Management a

Cybersecurity Risk Management and Strategy Disclosure	12 Months Ended
	Dec. 31, 2024
Cybersecurity Risk Management, Strategy, and Governance [Line Items]
Cybersecurity Risk Management Processes for Assessing, Identifying, and Managing Threats [Text Block]	Risk Management and Strategy As one of the critical elements of the Company’s overall enterprise risk management approach, the Company’s cybersecurity program is focused on the following key areas: • Governance: As discussed in more detail under the heading “Governance,” the Board’s oversight of cybersecurity is delegated to the Audit and Compliance Committee of the Board, which oversees the Company’s entire ERMP, reporting up to the full board on a periodic basis. The Company’s Chief Information Officer (“CIO”), the Chief Compliance Officer and other members of management regularly report to the Audit and Compliance Committee, with cybersecurity representing a standing meeting agenda topic. • Collaborative Approach: The Company has implemented a systematic, cross-functional approach to identifying, preventing and mitigating cybersecurity threats and incidents, while also implementing controls and procedures that provide for the prompt escalation of certain cybersecurity incidents so that decisions regarding the public disclosure and reporting of such incidents can be made by management in a timely manner. Technical Safeguards: The Company deploys technical safeguards that are designed to protect the Company’s information systems from cybersecurity threats, including firewalls, intrusion prevention and detection systems, anti-malware functionality and access controls, 24x7 security monitoring, and other controls which are evaluated and improved through vulnerability assessments and cybersecurity threat intelligence. • Incident Response and Recovery Planning: The Company has established and maintains systematic incident response and recovery plans that address the Company’s response to a cybersecurity incident, and such plans are tested and evaluated on a periodic basis. • Third-Party Risk Management: The Company maintains a systematic, risk-based approach to identifying and overseeing cybersecurity risks presented by third parties, including vendors, service providers and other external users of the Company’s systems, as well as the systems of third parties that could adversely impact our business in the event of a cybersecurity incident affecting those third-party systems. • Education and Awareness: The Company provides regular, mandatory cybersecurity training for all personnel as a means to equip the Company’s workforce with effective tools to recognize, address and communicate potential or actual threats to the Company’s cybersecurity systems. Moreover, these trainings also allow Company personnel to remain up-to-date with evolving information security policies, standards, processes and best practices. The Company engages in the periodic assessment and testing of the Company’s policies, standards, processes and practices that are designed to address cybersecurity threats and incidents. These efforts include a wide range of activities, including audits, assessments, tabletop exercises, threat modeling, vulnerability testing and other exercises focused on evaluating the effectiveness of our cybersecurity measures and planning. The Company has also engaged third parties to perform assessments on our cybersecurity measures, including information security maturity assessments, audits and independent reviews of our information security control environment and operating effectiveness. The results and findings of these exercises are reported to the Audit and Compliance Committee, who in turn updates the Board as appropriate. Management will then evaluate such findings and, with input from the Audit and Compliance Committee, take the appropriate steps to adjust the Company’s cybersecurity policies, standards, processes and practices, as may be applicable, to strengthen or address any weaknesses, gaps or findings as the case may be. As of the date of this Annual Report on Form 10-K, we are not aware of any risks from the cybersecurity threats that have materially affected or are reasonably likely to materially affect us, including our business strategy, results of operations and financial condition.
Cybersecurity Risk Management Processes Integrated [Flag]	true
Cybersecurity Risk Management Processes Integrated [Text Block]	The Company’s Board of Directors (the “Board”) recognizes the critical importance of maintaining the trust and confidence of our customers, clients, business partners and employees. The Board is actively involved in oversight of the Company’s Enterprise Risk Management Program (“ERMP”), and cybersecurity represents an important component of the Company’s overall approach to enterprise risk management. The Company’s cybersecurity policies, standards, processes and practices are integrated into the Company’s ERMP and are based on recognized frameworks established by the National Institute of Standards and Technology and other applicable industry standards. In general, the Company seeks to address cybersecurity risks through a systematic, cross-functional approach that is focused on preserving the confidentiality, security and availability of the information that the Company collects and stores by identifying, preventing and mitigating cybersecurity threats and responding to cybersecurity incidents if they should occur.
Cybersecurity Risk Management Third Party Engaged [Flag]	true
Cybersecurity Risk Third Party Oversight and Identification Processes [Flag]	true
Cybersecurity Risk Materially Affected or Reasonably Likely to Materially Affect Registrant [Flag]	false
Cybersecurity Risk Board of Directors Oversight [Text Block]	The Board has delegated their oversight of cybersecurity to the Company’s Audit and Compliance Committee which oversees the entire ERMP process. As detailed above, cybersecurity is a standing agenda topic for the Audit and Compliance Committee which receives regular presentations and reports from the Company’s CIO on cybersecurity risks, detection protocols, disaster recovery readiness, the threat environment, recent developments in the cybersecurity space (including known incidents affecting the Company or key Company suppliers), evolving standards, vulnerability assessments, third-party and independent reviews, technological trends and information security considerations arising with respect to the Company’s peers and third parties. Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident. The Audit and Compliance Committee will keep the full board informed, as may be appropriate, until any such threat has been addressed to their satisfaction. Additionally, cybersecurity is also a standing agenda topic for the Global Risk Committee, with periodic updates to the Executive Committee. The CIO, in coordination with the Chief Executive Officer, Chief Financial Officer, Chief Compliance Officer, Chief Legal Officer, and Chief People Officer works collaboratively across the Company to implement a program designed to protect the Company’s information systems from cybersecurity threats and to promptly respond to any cybersecurity incidents in accordance with the Company’s incident response and recovery plans. To facilitate the success of the Company’s cybersecurity risk management program, multidisciplinary teams, comprised of our cybersecurity experts, are deployed throughout the Company to identify and address cybersecurity threats and to respond to cybersecurity incidents. The CIO has served in various leadership roles in information technology and information security for over 24 years, including serving as the vice president of information technology, with direct responsibility over the cyber security program, for a large publicly-traded company and as the chief information security officer of several large healthcare organizations. The CIO holds a Certified Information Systems Security Professional certification, an undergraduate degree in engineering, an MBA and a PhD in engineering.
Cybersecurity Risk Board Committee or Subcommittee Responsible for Oversight [Text Block]	As detailed above, cybersecurity is a standing agenda topic for the Audit and Compliance Committee which receives regular presentations and reports from the Company’s CIO on cybersecurity risks, detection protocols, disaster recovery readiness, the threat environment, recent developments in the cybersecurity space (including known incidents affecting the Company or key Company suppliers), evolving standards, vulnerability assessments, third-party and independent reviews, technological trends and information security considerations arising with respect to the Company’s peers and third parties. Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident. The Audit and Compliance Committee will keep the full board informed, as may be appropriate, until any such threat has been addressed to their satisfaction. Additionally, cybersecurity is also a standing agenda topic for the Global Risk Committee, with periodic updates to the Executive Committee.
Cybersecurity Risk Process for Informing Board Committee or Subcommittee Responsible for Oversight [Text Block]	Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident.
Cybersecurity Risk Role of Management [Text Block]	As detailed above, cybersecurity is a standing agenda topic for the Audit and Compliance Committee which receives regular presentations and reports from the Company’s CIO on cybersecurity risks, detection protocols, disaster recovery readiness, the threat environment, recent developments in the cybersecurity space (including known incidents affecting the Company or key Company suppliers), evolving standards, vulnerability assessments, third-party and independent reviews, technological trends and information security considerations arising with respect to the Company’s peers and third parties. Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident. The Audit and Compliance Committee will keep the full board informed, as may be appropriate, until any such threat has been addressed to their satisfaction. Additionally, cybersecurity is also a standing agenda topic for the Global Risk Committee, with periodic updates to the Executive Committee. The CIO, in coordination with the Chief Executive Officer, Chief Financial Officer, Chief Compliance Officer, Chief Legal Officer, and Chief People Officer works collaboratively across the Company to implement a program designed to protect the Company’s information systems from cybersecurity threats and to promptly respond to any cybersecurity incidents in accordance with the Company’s incident response and recovery plans. To facilitate the success of the Company’s cybersecurity risk management program, multidisciplinary teams, comprised of our cybersecurity experts, are deployed throughout the Company to identify and address cybersecurity threats and to respond to cybersecurity incidents. The CIO has served in various leadership roles in information technology and information security for over 24 years, including serving as the vice president of information technology, with direct responsibility over the cyber security program, for a large publicly-traded company and as the chief information security officer of several large healthcare organizations. The CIO holds a Certified Information Systems Security Professional certification, an undergraduate degree in engineering, an MBA and a PhD in engineering.
Cybersecurity Risk Management Positions or Committees Responsible [Flag]	true
Cybersecurity Risk Management Positions or Committees Responsible [Text Block]	The Board has delegated their oversight of cybersecurity to the Company’s Audit and Compliance Committee which oversees the entire ERMP process. As detailed above, cybersecurity is a standing agenda topic for the Audit and Compliance Committee which receives regular presentations and reports from the Company’s CIO on cybersecurity risks, detection protocols, disaster recovery readiness, the threat environment, recent developments in the cybersecurity space (including known incidents affecting the Company or key Company suppliers), evolving standards, vulnerability assessments, third-party and independent reviews, technological trends and information security considerations arising with respect to the Company’s peers and third parties. Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident. The Audit and Compliance Committee will keep the full board informed, as may be appropriate, until any such threat has been addressed to their satisfaction. Additionally, cybersecurity is also a standing agenda topic for the Global Risk Committee, with periodic updates to the Executive Committee. The CIO, in coordination with the Chief Executive Officer, Chief Financial Officer, Chief Compliance Officer, Chief Legal Officer, and Chief People Officer works collaboratively across the Company to implement a program designed to protect the Company’s information systems from cybersecurity threats and to promptly respond to any cybersecurity incidents in accordance with the Company’s incident response and recovery plans. To facilitate the success of the Company’s cybersecurity risk management program, multidisciplinary teams, comprised of our cybersecurity experts, are deployed throughout the Company to identify and address cybersecurity threats and to respond to cybersecurity incidents. The CIO has served in various leadership roles in information technology and information security for over 24 years, including serving as the vice president of information technology, with direct responsibility over the cyber security program, for a large publicly-traded company and as the chief information security officer of several large healthcare organizations. The CIO holds a Certified Information Systems Security Professional certification, an undergraduate degree in engineering, an MBA and a PhD in engineering.
Cybersecurity Risk Management Expertise of Management Responsible [Text Block]	The CIO has served in various leadership roles in information technology and information security for over 24 years, including serving as the vice president of information technology, with direct responsibility over the cyber security program, for a large publicly-traded company and as the chief information security officer of several large healthcare organizations. The CIO holds a Certified Information Systems Security Professional certification, an undergraduate degree in engineering, an MBA and a PhD in engineering.
Cybersecurity Risk Process for Informing Management or Committees Responsible [Text Block]	Under the current cybersecurity framework, the Audit and Compliance Committee receives prompt and timely information regarding any cybersecurity incident that meets established reporting thresholds, as well as ongoing updates regarding any such incident.
Cybersecurity Risk Management Positions or Committees Responsible Report to Board [Flag]	true

Summary of Significant Accoun_2

Summary of Significant Accounting Policies (Policies)	12 Months Ended
	Dec. 31, 2024
Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The Company has prepared the accompanying Consolidated Financial Statements in accordance with U.S. generally accepted accounting principles ("U.S. GAAP") and include all adjustments necessary for the fair presentation of the Company's financial position for the periods presented. Certain prior year amounts have been reclassified for comparative purposes. The reclassifications did not affect results of operations, net assets or cash flows.
Consolidation	Consolidation The Consolidated Financial Statements include the accounts of the Company and its subsidiaries. Intercompany accounts and transactions are eliminated in consolidation.
Foreign Currency Transactions	Foreign Currency Transactions The functional currency for most of the Company's foreign subsidiaries is their local currency. For non-U.S. subsidiaries that transact in a functional currency other than the U.S. dollar, assets and liabilities are translated at current rates of exchange at the balance sheet date. Income and expense items are translated at the weighted average foreign exchange rates for the period. Adjustments resulting from the translation of the financial statements of the Company's foreign operations into U.S. dollars are excluded from the determination of net income and are recorded in accumulated other comprehensive income, a separate component of stockholders' equity. Transactions which are not in the functional currency of the entity are remeasured into the functional currency with gains or losses resulting from the remeasurement recorded in other (expense) income.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements, and the reported amounts of revenues and expenses during the reporting periods. Actual results could differ from those estimates.
Cash, Cash Equivalents, Marketable Securities, and Restricted Cash	Cash, Cash Equivalents, Marketable Securities, and Restricted Cash The Company considers all highly liquid investments purchased with a maturity of three months or less at the date of acquisition to be cash equivalents. Marketable securities consist of fixed income investments with a maturity of greater than three months and other highly liquid investments that can be readily purchased or sold using established markets. These investments are classified as available-for-sale and are reported at fair value on the Company's Consolidated Balance Sheets. Unrealized holding gains and losses are reported within other comprehensive gain (loss) in the Company's Consolidated Statements of Comprehensive Loss. Fair value is based on available market information including quoted market prices, broker or dealer quotations, or other observable inputs.
Concentration of Credit Risk	Concentration of Credit Risk The Company's financial instruments that are exposed to concentration of credit risk consist primarily of cash, cash equivalents, and marketable securities. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company invests its marketable securities in high-quality commercial financial instruments. The Company has not recognized any losses from credit risks on such accounts during any of the periods presented. The Company believes it is not exposed to significant credit risk on its cash, cash equivalents, or marketable securities. The Company is subject to credit risk from its accounts receivable primarily related to its product sales of Galafold ®
Property and Equipment	Property and Equipment Property and equipment are stated at cost, less accumulated depreciation. Depreciation is calculated over the estimated useful lives of the respective assets, which range from three The initial cost of property and equipment consists of its purchase price and any directly attributable costs of bringing the asset to its working condition and location for its intended use. Expenditures incurred after the fixed assets have been put into operation, such as repairs and maintenance, are expensed as incurred. Major replacements, improvements, and additions are capitalized in accordance with Company policy. The Company evaluates long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset group may not be recoverable. If indications of impairment exist, projected future undiscounted cash flows associated with the asset or asset group are compared to the carrying value of the asset to determine whether the asset or asset group's value is recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the long-lived asset over its estimated fair value in the period at which such a determination is made.
Revenue Recognition	Revenue Recognition The Company has recorded revenue on sales where its products are available either on a commercial basis or through a reimbursed early access program. Product orders are generally received from distributors and pharmacies, with the ultimate payor often a government authority. In 2024, one customer accounted for 26% of net product sales and 18% of accounts receivable from product sales while another customer accounted for 13% of accounts receivable from product sales. In 2023, one customer accounted for 28% of net product sales and 16% of accounts receivable from product sales while another customer accounted for 17% of accounts receivable from product sales. Revenue attributed to U.K. and Germany each accounted for 13% of net product sales in 2024, 10% and 13% respectively in 2023, and 12% each in 2022. The Company recognizes revenue when its performance obligations to its customers have been satisfied, which occurs at a point in time when the pharmacies or distributors obtain control of the products. The transaction price is determined based on fixed consideration in the Company's customer contracts and is recorded net of estimates for variable consideration, which primarily consist of third-party discounts and rebates. The identified variable consideration is recorded as a reduction of revenue at the time revenue from the sale is recognized. The Company recognizes revenue to the extent that it is probable that a significant revenue reversal will not occur in a future period. These estimates may differ from actual consideration received. The Company evaluates these estimates each reporting period to reflect known changes.
Inventories and Cost of Goods Sold	Inventories and Cost of Goods Sold Until regulatory approval of Pombiliti ® + Opfolda ® , the Company expensed all manufacturing costs as research and development expense. Upon regulatory approval, the Company began capitalizing costs related to the purchase and manufacture of Pombiliti ® + Opfolda ® . Inventories are stated at the lower of cost and net realizable value, determined by the first-in, first-out method. Inventories are reviewed periodically to identify slow-moving or obsolete inventory based on projected sales activity as well as product shelf-life. In evaluating the recoverability of inventories produced, the probability that revenue will be obtained from the future sale of the related inventory is considered and inventory value is written down for inventory quantities in excess of expected requirements. Expired inventory is disposed of and the related costs are recognized as cost of goods sold in the Company's Consolidated Statements of Operations. Cost of goods sold includes the cost of inventory sold, manufacturing and supply chain costs, product shipping and handling costs, provisions for excess and obsolete inventory, as well as royalties payable. A portion of inventory available for sale was expensed as research and development costs prior to regulatory approval and, as such, the cost of goods sold and related gross margins are not necessarily indicative of future costs of goods sold and gross margin.
Fair Value Measurements	Fair Value Measurements The Company records certain asset and liability balances under the fair value measurements as defined by the Financial Accounting Standard Board ("FASB") guidance. Current FASB fair value guidance emphasizes that fair value is a market-based measurement, not an entity-specific measurement. Therefore, a fair value measurement should be determined based on the assumptions that market participants would use in pricing the asset or liability. As a basis for considering market participant assumptions in fair value measurements, current FASB guidance establishes a fair value hierarchy that distinguishes between market participant assumptions based on market data obtained from sources independent of the reporting entity (observable inputs that are classified within Levels 1 and 2 of the hierarchy) and the reporting entity's own assumptions that market participant's assumptions would use in pricing assets or liabilities (unobservable inputs classified within Level 3 of the hierarchy). Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access at measurement date. Level 2 inputs are inputs other than quoted prices included in Level 1 that are observable for the asset or liability, either directly or indirectly. Level 2 inputs may include quoted prices for similar assets and liabilities in active markets, as well as inputs that are observable for the asset or liability (other than quoted prices), such as interest rates, foreign exchange rates, and yield curves that are observable at commonly quoted intervals. Level 3 inputs are unobservable inputs for the asset or liability, which is typically based on an entity's own assumptions, as there is little, if any, related market activity. In instances where the determination of the fair value measurement is based on inputs from different levels of the fair value hierarchy, the level in the fair value hierarchy within which the entire fair value measurement falls is based on the lowest level input that is significant to the fair value measurement in its entirety. The Company's assessment of the significance of a particular input to the fair value measurement in its entirety requires judgment and considers factors specific to the asset or liability.
Research and Development Costs	Research and Development Costs Research and development costs are expensed as incurred. Research and development expense consist primarily of costs related to personnel, including salaries and other personnel related expenses, consulting fees, and the cost of facilities and support services used in drug development. Assets acquired that are used for research and development and have no future alternative use are expensed as in-process research and development.
Interest Income and Interest Expense	Interest Income and Interest Expense Interest income consists of interest earned on the Company's cash, cash equivalents, and marketable securities. Interest expense consists of interest incurred on debt and finance leases.
Income Taxes	Income Taxes The Company accounts for income taxes under the liability method. Under this method deferred income tax liabilities and assets are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities and for operating losses and tax credit carry forwards, using enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance is recorded if it is "more likely than not" that a portion or all of a deferred tax asset will not be realized. The Company’s tax returns are subject to examination by U.S. Federal, state, and foreign taxing jurisdictions. The impact of an uncertain tax position taken or expected to be taken on an income tax return must be recognized in the financial statements at the largest amount that is more likely than not to be sustained. An uncertain income tax position will not be recognized in the financial statements unless it is more likely than not to be sustained.
Other Comprehensive Gain (Loss)	Other Comprehensive Gain (Loss) Components of other comprehensive gain (loss) include unrealized gains and losses on available-for-sale securities and gains and losses on foreign currency transactions, and are included in the Consolidated Statements of Comprehensive Loss.
Leases	Leases The Company primarily enters into lease agreements for office space, equipment, and vehicles. The leases have varying terms, some of which could include options to renew, extend, and early terminate. The Company determines if an arrangement is a lease at contract inception. Operating leases are included in right-of-use ("ROU") assets and lease liabilities on the Consolidated Balance Sheets. ROU assets represent the Company's right to control the use of an explicitly or implicitly identified fixed asset for a period of time and lease liabilities represent the Company's obligation to make lease payments arising from the lease. Control of an underlying asset is conveyed if the Company obtains the rights to direct the use of and to obtain substantially all of the economic benefits from using the underlying asset. ROU assets and liabilities are recognized at commencement date based on the present value of lease payments over the lease term. 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. Lease payments included in the measurement of the lease liability are comprised of fixed payments. Variable lease payments are excluded from the ROU asset and lease liability and are recognized in the period in which the obligation for those payments is incurred. Variable lease payments are presented in the Consolidated Statements of Operations in the same line item as expenses arising from fixed lease payments for operating leases. The Company has lease agreements that include lease and non-lease components, which the Company accounts for as a single lease component for all underlying asset categories. The lease term for all of the Company's leases includes the non-cancellable period of the lease plus any additional periods covered by either a Company option to extend (or not to terminate) the lease that the Company is reasonably certain to exercise, or an option to extend (or not to terminate) the lease controlled by the lessor. Leases with an initial term of 12 months or less are not recorded on the Consolidated Balance Sheets. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. The Company applies this policy to all underlying asset categories.
Nonqualified Cash Deferral Plan	Nonqualified Cash Deferral Plan The Company's Cash Deferral Plan (the "Deferral Plan") provides certain key employees and members of the Board of Directors as selected by the Compensation Committee of the Board of Directors of the Company (the "Compensation Committee"), with an opportunity to defer the receipt of such participant's base salary, bonus, and director's fees, as applicable. The Deferral Plan is intended to be a nonqualified deferred compensation plan that complies with the provisions of Section 409A of the Internal Revenue Code. All of the investments held in the Deferral Plan are classified as trading securities and recorded at fair value with changes in the investments' fair value recognized as earnings in the period they occur. The corresponding liability for the Deferral Plan is included in other non-current liability in the Consolidated Balance Sheets.
Equity-based Compensation	Equity-based Compensation At December 31, 2024, the Company had one equity-based employee compensation plan, which is described more fully in "— Note 8. Stockholders' Equity." The Company applies the fair value method of measuring equity-based compensation, which requires a public entity to measure the cost of employee services received in exchange for an award of equity instruments based on the grant-date fair value of the award.
Loss per Common Share	Loss per Common Share
Segment Information	Segment Information The Company currently operates in one business segment focused on the discovery, development, and commercialization of advanced therapies to treat a range of devastating rare and orphan diseases. The Company is not organized by market and is managed and operated as one business. A single management team reports to the chief operating decision maker ("CODM"), its Chief Executive Officer, who comprehensively manages the entire business. The Company and its CODM evaluate performance and allocate resources primarily based on Net product sales and Net loss within the Consolidated Statement of Operations he is regularly provided. Net loss is used to monitor budget to actual results and actuals against prior periods. The Company does not accumulate discrete financial information with respect to separate service lines, and thus there is one reportable segment. The Company does not operate any separate lines of business or separate business entities with respect to its products. Accordingly, the Consolidated Statements of Operations and Consolidated Balance Sheets provide information regarding significant segment expenses and segment assets. Revenue segregation by product line is presented in the revenue recognition section earlier in this footnote.
Intangible Assets and Goodwill	Intangible Assets and Goodwill The Company records goodwill in a business combination when the total consideration exceeds the fair value of the net tangible and identifiable intangible assets acquired. Goodwill is assessed annually for impairment on October 1 and whenever events or circumstances indicate that the carrying amount of an asset may not be recoverable. The Company first assesses the qualitative factors to determine if a quantitative test is necessary. If required, or if the Company elects to bypass the qualitative assessment, a quantitative goodwill impairment test is concluded. If it is determined the Company's single reporting unit's carrying value, including goodwill, exceeds its fair value, an impairment loss is recorded for the difference. Finite-lived intangible assets are recorded at cost, net of accumulated amortization, and, if applicable, impairment charges. Amortization of finite-lived intangible assets is recorded over the assets’ estimated useful lives on a straight-line basis or based on the pattern in which economic benefits are consumed, if reliably determinable. The Company reviews finite-lived intangible assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. If impairment is determined, the Company writes down the asset to its estimated fair value and records an impairment loss equal to the excess of the carrying value of the asset over its estimated fair value in the period at which such a determination is made.
Recent Accounting Developments	Recent Accounting Developments - Guidance Adopted in 2024 In November 2023, the FASB issued the Accounting Standards Update ("ASU") No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures. The amendments expand reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses. The amendments require, among other things, disclosure of the title and position of the chief operating decision maker and require that public entities with a single reportable segment provide all disclosures required by this update and existing segment disclosures in Topic 280. Annual disclosures are required for fiscal years beginning after December 15, 2023 and interim disclosures are required for periods within fiscal years beginning after December 15, 2024. Retrospective application is required unless it is impracticable, and early adoption is permitted. The Company adopted this guidance on January 1, 2024. Recent Accounting Developments - Guidance Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures. The ASU requires disclosure of disaggregated income taxes paid, prescribes standard categories for the components of the effective tax rate reconciliation, and modifies other income tax-related disclosures. ASU 2023-09 is effective for fiscal years beginning after December 15, 2024, must be applied prospectively with an option to apply retrospectively, and early adoption is permitted. The adoption of this guidance is not expected to have a significant impact on the Company's consolidated financial statements. In November 2024, the FASB issued ASU 2024-03, "Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses." The standard is intended to require more detailed disclosures about specified categories of expenses (including employee compensation, depreciation, and amortization) included in certain expense captions presented on the face of the income statement. This ASU is effective for fiscal years beginning after December 15, 2026, and for interim periods within fiscal years beginning after December 15, 2027. Early adoption is permitted. The amendments may be applied either prospectively to financial statements issued for reporting periods after the effective date of this ASU or retrospectively to all prior periods presented in the financial statements. The Company is currently assessing the impact this standard will have on its consolidated financial statements.

Summary of Significant Accoun_3

Summary of Significant Accounting Policies (Tables)	12 Months Ended
	Dec. 31, 2024
Accounting Policies [Abstract]
Schedule of Revenue Disaggregated by Product and Geographical Area	The following table summarizes the Company's net product sales disaggregated by product and geographic area: For the Year (in thousands) 2024 2023 2022 Galafold ® U.S. $ 176,024 $ 145,147 $ 115,759 Galafold ® Ex-U.S. 282,030 242,630 213,287 Total Galafold ® sales $ 458,054 $ 387,777 $ 329,046 Pombiliti ® + Opfolda ® U.S. $ 30,123 1,791 — Pombiliti ® + Opfolda ® Ex-U.S. 40,118 9,788 187 Total Pombiliti ® + Opfolda ® sales $ 70,241 $ 11,579 $ 187 Total net product sales $ 528,295 $ 399,356 $ 329,233

Cash, Cash Equivalents, Marke_2

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash (Tables)	12 Months Ended
	Dec. 31, 2024
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Schedule of Cash and Available for Sale Securities	Cash, cash equivalents, and marketable securities are classified as current unless mentioned otherwise below and consisted of the following: As of December 31, 2024 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 213,752 $ — $ — $ 213,752 Commercial paper 18,082 3 (4) 18,081 U.S. government agency bonds 16,524 1 (7) 16,518 Corporate debt securities 1,446 — (2) 1,444 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 249,955 $ 4 $ (13) $ 249,946 Included in cash and cash equivalents $ 213,752 $ — $ — $ 213,752 Included in marketable securities 36,203 4 (13) 36,194 Total cash, cash equivalents, and marketable securities $ 249,955 $ 4 $ (13) $ 249,946 As of December 31, 2023 (in thousands) Cost Gross Gross Fair Cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Commercial paper 14,651 12 — 14,663 Treasury bill 12,944 2 — 12,946 U.S. government agency bonds 11,450 — (4) 11,446 Money market 100 — — 100 Certificates of deposit 51 — — 51 $ 286,190 $ 14 $ (4) $ 286,200 Included in cash and cash equivalents $ 246,994 $ — $ — $ 246,994 Included in marketable securities 39,196 14 (4) 39,206 Total cash, cash equivalents, and marketable securities $ 286,190 $ 14 $ (4) $ 286,200
Schedule of Reconciliation of Cash and Cash Equivalents and Restricted Cash	The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Consolidated Balance Sheets that sum to the total of the same such amounts shown in the Consolidated Statements of Cash Flows. December 31, (in thousands) 2024 2023 2022 Cash and cash equivalents $ 213,752 $ 246,994 $ 148,813 Restricted cash 2,964 3,083 4,302 Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows $ 216,716 $ 250,077 $ 153,115

Inventories (Tables)

Inventories (Tables)	12 Months Ended
	Dec. 31, 2024
Inventory Disclosure [Abstract]
Schedule of Inventories	Inventories consisted of the following: December 31, (in thousands) 2024 2023 Raw materials $ 87,916 $ 30,230 Work-in-process 21,223 22,597 Finished goods 9,643 6,869 Total inventories $ 118,782 $ 59,696

Property and Equipment (Tables)

Property and Equipment (Tables)	12 Months Ended
	Dec. 31, 2024
Property, Plant and Equipment [Abstract]
Schedule of Property and Equipment	Property and equipment consist of the following: December 31, (in thousands) 2024 2023 Leasehold improvements $ 32,361 $ 28,032 Research equipment 16,220 16,220 Computer equipment 5,198 4,934 Furniture and fixtures 3,715 2,928 Computer software 402 1,106 Construction in progress 262 3,810 Vehicles — 66 Gross property and equipment 58,158 57,096 Less accumulated depreciation (28,775) (25,429) Net property and equipment $ 29,383 $ 31,667

Accrued Expenses and Other Cu_2

Accrued Expenses and Other Current Liabilities (Tables)	12 Months Ended
	Dec. 31, 2024
Payables and Accruals [Abstract]
Schedule of Accounts Payable and Accrued Expenses	Accrued expenses and other current liabilities consist of the following: December 31, (in thousands) 2024 2023 Accrued sales rebates and discounts $ 33,546 $ 31,190 Accrued compensation and benefits 32,529 38,305 Accrued taxes 18,105 12,434 Accrued program fees 10,578 13,607 Accrued royalties 9,790 8,238 Accrued professional fees 6,337 5,059 Accrued contract manufacturing & contract research costs 3,924 13,380 Accrued restructuring 3,665 — Accrued interest on debt 120 11,661 Other 8,706 10,371 $ 127,300 $ 144,245

Restructuring (Tables)

Restructuring (Tables)	12 Months Ended
	Dec. 31, 2024
Restructuring and Related Activities [Abstract]
Schedule of Total Charges Incurred	Total charges incurred are summarized as follows: (in thousands) Facility and Non-Lease Costs Other Costs Total Balance as of December 31, 2023 $ — $ — $ — Restructuring charges 6,045 3,143 9,188 Non-cash items (2,844) (2,844) Cash settled (1,025) (1,654) (2,679) Balance as of December 31, 2024 $ 2,176 $ 1,489 $ 3,665

Stock based Compensation (Table

Stock based Compensation (Tables)	12 Months Ended
	Dec. 31, 2024
Share-Based Payment Arrangement [Abstract]
Schedule of Fair Value of Options	The fair value of the stock options granted were estimated on the date of grant using a Black-Scholes option pricing model with the following weighted-average assumptions: Years ended December 31, 2024 2023 2022 Expected stock price volatility 57.0 % 59.2 % 62.1 % Risk free interest rate 4.0 % 3.9 % 1.7 % Expected life of options (years) 5.60 5.47 5.34 Expected annual dividend per share $ — $ — $ —
Schedule of Stock Options Activity	A summary of the Company's stock options is as follows: Number of Weighted Weighted Aggregate (in thousands) (in millions) Options outstanding, December 31, 2021 14,731 $ 11.08 Granted 5,733 $ 11.53 Exercised (660) $ 6.58 Forfeited (495) $ 12.57 Expired (245) $ 12.84 Options outstanding, December 31, 2022 19,064 $ 11.31 Granted 5,733 $ 11.99 Exercised (1,361) $ 7.54 Forfeited (356) $ 11.59 Expired (78) $ 15.09 Options outstanding, December 31, 2023 23,002 $ 11.69 Granted 4,917 $ 13.52 Exercised (957) $ 8.02 Forfeited (622) $ 12.75 Expired (327) $ 14.30 Options outstanding, December 31, 2024 26,013 $ 12.11 5.8 $ 5.8 Vested and unvested expected to vest, December 31, 2024 24,643 $ 12.09 5.7 $ 5.8 Exercisable at December 31, 2024 17,464 $ 11.80 4.6 $ 5.7
Schedule of Non-Vested RSU Activity under the Plan	A summary of non-vested RSU activity under the Plan is as follows: Number of Weighted Average Grant Date Fair Value Weighted Average Remaining Years Aggregate Intrinsic Value (in thousands) (in millions) Non-vested units as of December 31, 2021 7,341 $ 13.90 Granted 5,048 $ 11.93 Vested (2,251) $ 12.48 Forfeited (421) $ 12.06 Non-vested units as of December 31, 2022 9,717 $ 13.07 Granted 4,762 $ 13.02 Vested (3,610) $ 12.21 Forfeited (836) $ 11.63 Non-vested units as of December 31, 2023 10,033 $ 13.37 Granted 4,150 $ 14.32 Vested (5,107) $ 13.07 Forfeited (799) $ 13.74 Non-vested units as of December 31, 2024 8,277 $ 13.59 2.1 $ 78.0
Schedule of Equity Compensation Expenses	The following table summarizes information related to compensation expense recognized in the Company's Consolidated Statements of Operations related to the equity awards: Years ended December 31, (in thousands) 2024 2023 2022 Research and development expense $ 15,969 $ 21,470 $ 25,089 Selling, general, and administrative expense 68,936 64,607 51,423 Total equity compensation expense $ 84,905 $ 86,077 $ 76,512

Assets and Liabilities Measur_2

Assets and Liabilities Measured at Fair Value (Tables)	12 Months Ended
	Dec. 31, 2024
Fair Value Disclosures [Abstract]
Schedule of Assets and Liabilities Subject to Fair Value Measurements	A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2024 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 18,081 $ 18,081 U.S. government agency bonds — 16,518 16,518 Corporate debt securities — 1,444 1,444 Money market 2,318 — 2,318 $ 2,318 $ 36,043 $ 38,361 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability $ 2,218 $ — $ 2,218 $ 2,218 $ — $ 2,218 A summary of the fair value of the Company's recurring assets and liabilities aggregated by the level in the fair value hierarchy within which those measurements fall as of December 31, 2023 are identified in the following tables: (in thousands) Level 1 Level 2 Total Assets: Commercial paper $ — $ 14,663 $ 14,663 Treasury bill — 12,946 12,946 U.S. government agency bonds — 11,446 11,446 Money market 7,631 — 7,631 $ 7,631 $ 39,055 $ 46,686 (in thousands) Level 1 Level 2 Total Liabilities: Deferred compensation plan liability 7,531 — 7,531 $ 7,531 $ — $ 7,531

Debt (Tables)

Debt (Tables)	12 Months Ended
	Dec. 31, 2024
Debt Disclosure [Abstract]
Schedule of Liability Components of the Convertible Notes	The Company's debt consists of the following: As of December 31, (in thousands) 2024 2023 Senior Secured Term Loan due 2029: Principal $ 400,000 $ 400,000 Less: debt discount (1) (7,863) (9,652) Less: deferred financing (1) (2,026) (2,490) Net carrying value of long-term debt $ 390,111 $ 387,858 _____________________________ (1) Included in the Consolidated Balance Sheets within long-term debt and amortized to interest expense over the remaining life of the corresponding Senior Secured Term Loan using the effective interest rate method.
Schedule of Components of Total Interest Expense	The following table sets forth interest expense recognized related to the Company's debt for the years ended December 31, 2024 and 2023, respectively: December 31, (in thousands, except interest rate amounts) 2024 2023 Contractual interest expense $ 47,373 $ 47,626 Amortization of debt discount $ 1,790 $ 1,605 Amortization of deferred financing $ 472 $ 1,011 Effective interest rate of the liability component, Senior Secured Term Loan due 2029 12.0 % 12.8 %

Leases (Tables)

Leases (Tables)	12 Months Ended
	Dec. 31, 2024
Leases [Abstract]
Schedule of Supplemental Balance Sheet Information	Supplemental balance sheet information related to operating leases were as follows: December 31, (in thousands, except year and discount rate amounts) 2024 2023 Operating lease ROU assets, net $ 22,278 $ 26,312 Current portion of the operating lease liabilities $ 8,455 $ 8,324 Non-current portion of the operating lease liabilities 45,078 48,877 Total operating lease liability $ 53,533 $ 57,201 Weighted-average remaining lease terms (years) 8.5 9.4 Weighted-average discount rate 9.7 % 9.7 %
Schedule of Future Minimum Operating Lease Payments	At December 31, 2024, the future minimum operating lease payments were as follows: (in thousands) Operating Lease 2025 $ 9,466 2026 9,356 2027 9,426 2028 9,139 2029 8,172 Thereafter 35,402 Total lease payments 80,961 Less: lease incentives (22,299) Less: imputed interest (5,129) Total operating lease liability $ 53,533

Income Taxes (Tables)

Income Taxes (Tables)	12 Months Ended
	Dec. 31, 2024
Income Tax Disclosure [Abstract]
Schedule of Income (Loss) Before Income Taxes	For financial reporting purposes, income (loss) before income taxes includes the following components: Years ended December 31, (in thousands) 2024 2023 2022 United States $ (210,049) $ (234,482) $ (343,424) Foreign 181,293 84,381 101,385 Total $ (28,756) $ (150,101) $ (242,039)
Schedule of Income Tax Expense (Benefit)	Following were the components of income tax expense (benefit) for the years ended December 31, 2024, 2023, and 2022: Years ended December 31, (in thousands) 2024 2023 2022 Current Federal $ — $ — $ — State 76 14 6 Foreign 27,274 6,408 (5,760) Deferred Federal — (4,801) 274 State — (138) 9 Foreign — — — Total $ 27,350 $ 1,483 $ (5,471)
Schedule of Reconciliation of the Statutory Tax Rates and the Effective Tax Rates	A reconciliation of the statutory tax rates and the effective tax rates for the years ended December 31, 2024, 2023, and 2022 are as follows: Years ended December 31, 2024 2023 2022 Statutory rate (21) % (21) % (21) % Tax credits (14) (8) (11) Impact of foreign operations 180 (9) 18 Executive compensation 36 7 3 Other 63 1 — Valuation allowance (149) 31 9 Net 95 % 1 % (2) %
Reconciliation of Unrecognized Tax Benefits	A reconciliation of the beginning and ending amount of unrecognized tax benefits is as follows: Years ended December 31, (in thousands) 2024 2023 2022 Gross unrecognized tax benefits at beginning of year $ 4,888 $ 283 $ — Gross increases for tax positions in prior years 9,542 4,605 283 Decreases for settlements and payments (625) — — Gross unrecognized tax benefits at end of year $ 13,805 $ 4,888 $ 283
Schedule of Significant Components of the Deferred Tax Assets and Liabilities	The significant components of the deferred tax assets and liabilities are as follows: Years ended December 31, (in thousands) 2024 2023 Deferred tax assets Net operating loss carry forwards 302,066 321,004 Research tax credit 234,189 235,532 Capitalized research and development costs 34,883 34,425 Share-based compensation 20,045 17,314 Lease liability 10,026 10,567 Interest carry forward limitation 9,097 17,627 Inventory 1,599 1,931 Other 5,267 20,858 Gross deferred tax assets 617,172 659,258 Deferred tax liabilities Other (8,042) (10,004) Total net deferred tax assets 609,130 649,254 Less: valuation allowance (609,130) (649,254) Net deferred tax liability $ — $ —

Basic and Diluted Net Loss pe_2

Basic and Diluted Net Loss per Common Share (Tables)	12 Months Ended
	Dec. 31, 2024
Earnings Per Share [Abstract]
Schedule of Reconciliation of the Numerator and Denominator Used in Computing Basic and Diluted Net Loss Per Common Share	The following table provides a reconciliation of the numerator and denominator used in computing basic and diluted net loss attributable to common stockholders per common share: Years ended December 31, (in thousands, except per share amounts) 2024 2023 2022 Numerator: Net loss attributable to common stockholders $ (56,106) $ (151,584) $ (236,568) Denominator: Weighted average common shares outstanding — basic and diluted 304,380,502 295,164,515 289,057,198
Schedule of Potential Shares of Common Stock that were Excluded from the Computation as they were Anti-Dilutive Using the Treasury Stock Method	The table below presents potential shares of common stock that were excluded from the computation as they were anti-dilutive using the treasury stock method: Years ended December 31, (in thousands) 2024 2023 2022 Options to purchase common stock 26,013 23,002 19,064 Unvested restricted stock units 8,277 10,033 9,717 Total number of potentially issuable shares 34,290 33,035 28,781

Description of Business (Detail

Description of Business (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Accumulated deficit	$ 2,740,180	$ 2,684,074

Summary of Significant Accoun_4

Summary of Significant Accounting Policies - Concentration of Credit Risk and Revenue Recognition (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Concentration Risk [Line Items]
Allowance for doubtful accounts receivable	$ 3
Net product sales | Customer Concentration Risk | Customer One
Concentration Risk [Line Items]
Concentration risk, percentage	26%	28%
Net product sales | Geographic Concentration Risk | United Kingdom
Concentration Risk [Line Items]
Concentration risk, percentage	13%	10%	12%
Net product sales | Geographic Concentration Risk | Germany
Concentration Risk [Line Items]
Concentration risk, percentage	13%	13%	12%
Accounts receivable | Customer Concentration Risk | Customer One
Concentration Risk [Line Items]
Concentration risk, percentage	18%	16%
Accounts receivable | Customer Concentration Risk | Customer Two
Concentration Risk [Line Items]
Concentration risk, percentage	13%	17%

Summary of Significant Accoun_5

Summary of Significant Accounting Policies - Property and Equipment (Details)	Dec. 31, 2024
Minimum
Property and Equipment
Estimated useful lives	3 years
Maximum
Property and Equipment
Estimated useful lives	5 years

Summary of Significant Accoun_6

Summary of Significant Accounting Policies - Revenue by Product and Geographical Area (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Disaggregation of Revenue [Line Items]
Net product sales	$ 528,295	$ 399,356	$ 329,233
Galafold
Disaggregation of Revenue [Line Items]
Net product sales	458,054	387,777	329,046
Pombiliti + Opfolda
Disaggregation of Revenue [Line Items]
Net product sales	70,241	11,579	187
UNITED STATES | Galafold
Disaggregation of Revenue [Line Items]
Net product sales	176,024	145,147	115,759
UNITED STATES | Pombiliti + Opfolda
Disaggregation of Revenue [Line Items]
Net product sales	30,123	1,791	0
Non-US | Galafold
Disaggregation of Revenue [Line Items]
Net product sales	282,030	242,630	213,287
Non-US | Pombiliti + Opfolda
Disaggregation of Revenue [Line Items]
Net product sales	$ 40,118	$ 9,788	$ 187

Summary of Significant Accoun_7

Summary of Significant Accounting Policies - Equity-Based Compensation (Details)	Dec. 31, 2024 plan
Equity-based Compensation
Number of equity-based employee compensation plans	1

Summary of Significant Accoun_8

Summary of Significant Accounting Policies - Segment Information (Details)	12 Months Ended
	Dec. 31, 2024 segment
Accounting Policies [Abstract]
Number of business segments	1
Number of reportable segments	1

Goodwill and Intangible Assets

Goodwill and Intangible Assets (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Goodwill and Intangible Assets Disclosure [Abstract]
Intangible assets	$ 17,198	$ 20,490
Initial regulatory exclusivity period	7 years
Amortization expense	$ 3,300	2,500
Finite-lived intangible assets, year one	3,300
Finite-lived intangible assets, year two	3,300
Finite-lived intangible assets, year three	3,300
Finite-lived intangible assets, year four	3,300
Finite-lived intangible assets, year five	$ 3,300
Finite-lived intangible assets, remaining amortization period (in years)	5 years 2 months 12 days
Goodwill	$ 197,797	$ 197,797

Cash, Cash Equivalents, Marke_3

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Narrative (Details) - USD ($)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 213,752,000	$ 246,994,000	$ 148,813,000
Debt securities, available-for-sale	36,194,000	39,206,000
Debt securities, realized gain (loss)	0	0
Fair value of available-for-sale debt securities in unrealized loss positions	$ 23,700,000	$ 11,400,000

Cash, Cash Equivalents, Marke_4

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Cash and Available for Sale Securities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Debt Securities, Available-for-sale [Line Items]
Cash and cash equivalents	$ 213,752	$ 246,994	$ 148,813
Fair value, cash balances	213,752	246,994
Cost, available-for-sale securities	36,203	39,196
Gross unrealized gain, available-for-sale securities	4	14
Gross unrealized loss, available-for-sale securities	(13)	(4)
Fair value, available-for-sale debt securities	36,194	39,206
Cost, cash balances and available-for-sale securities	249,955	286,190
Gross unrealized gain, cash balances and available-for-sale securities	4	14
Gross unrealized loss, cash balances and available-for-sale securities	(13)	(4)
Fair value, cash balances and available-for-sale securities	249,946	286,200
Commercial paper
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	18,082	14,651
Gross unrealized gain, available-for-sale securities	3	12
Gross unrealized loss, available-for-sale securities	(4)	0
Fair value, available-for-sale debt securities	18,081	14,663
Treasury bill
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities		12,944
Gross unrealized gain, available-for-sale securities		2
Gross unrealized loss, available-for-sale securities		0
Fair value, available-for-sale debt securities		12,946
U.S. government agency bonds
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	16,524	11,450
Gross unrealized gain, available-for-sale securities	1	0
Gross unrealized loss, available-for-sale securities	(7)	(4)
Fair value, available-for-sale debt securities	16,518	11,446
Corporate debt securities
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	1,446
Gross unrealized gain, available-for-sale securities	0
Gross unrealized loss, available-for-sale securities	(2)
Fair value, available-for-sale debt securities	1,444
Money market
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	100	100
Gross unrealized gain, available-for-sale securities	0	0
Gross unrealized loss, available-for-sale securities	0	0
Fair value, available-for-sale debt securities	100	100
Certificates of deposit
Debt Securities, Available-for-sale [Line Items]
Cost, available-for-sale securities	51	51
Gross unrealized gain, available-for-sale securities	0	0
Gross unrealized loss, available-for-sale securities	0	0
Fair value, available-for-sale debt securities	$ 51	$ 51

Cash, Cash Equivalents, Marke_5

Cash, Cash Equivalents, Marketable Securities, and Restricted Cash - Reconciliation of Cash and Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Cash, Cash Equivalents, and Short-Term Investments [Abstract]
Cash and cash equivalents	$ 213,752	$ 246,994	$ 148,813
Restricted cash	2,964	3,083	4,302
Cash, cash equivalents, and restricted cash shown in the Consolidated Statements of Cash Flows	$ 216,716	$ 250,077	$ 153,115	$ 249,456

Inventories (Details)

Inventories (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 87,916	$ 30,230
Work-in-process	21,223	22,597
Finished goods	9,643	6,869
Total inventories	$ 118,782	$ 59,696

Inventories - Narrative (Detail

Inventories - Narrative (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Reserve for inventory	$ 3.3	$ 0.5
Purchase obligation	179.7
Purchase obligation, to be paid in 2025	102.7
Purchase obligation, to be paid in 2026	$ 77

Property and Equipment (Details

Property and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Property and Equipment
Gross property and equipment	$ 58,158	$ 57,096
Less accumulated depreciation	(28,775)	(25,429)
Net property and equipment	29,383	31,667
Leasehold improvements
Property and Equipment
Gross property and equipment	32,361	28,032
Research equipment
Property and Equipment
Gross property and equipment	16,220	16,220
Computer equipment
Property and Equipment
Gross property and equipment	5,198	4,934
Furniture and fixtures
Property and Equipment
Gross property and equipment	3,715	2,928
Computer software
Property and Equipment
Gross property and equipment	402	1,106
Construction in progress
Property and Equipment
Gross property and equipment	262	3,810
Vehicles
Property and Equipment
Gross property and equipment	$ 0	$ 66

Property and Equipment - Narrat

Property and Equipment - Narrative (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Property, Plant and Equipment [Abstract]
Depreciation expense	$ 5	$ 4.8

Accrued Expenses and Other Cu_3

Accrued Expenses and Other Current Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Accounts payable and accrued expenses
Accrued sales rebates and discounts	$ 33,546	$ 31,190
Accrued compensation and benefits	32,529	38,305
Accrued taxes	18,105	12,434
Accrued program fees	10,578	13,607
Accrued royalties	9,790	8,238
Accrued professional fees	6,337	5,059
Accrued contract manufacturing & contract research costs	3,924	13,380
Accrued restructuring	3,665	0
Accrued interest on debt	120	11,661
Other	8,706	10,371
Accrued expenses and other current liabilities	$ 127,300	$ 144,245

Restructuring (Details)

Restructuring (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Restructuring Reserve [Roll Forward]
Beginning balance	$ 0
Restructuring charges	9,188	$ 0	$ 0
Non-cash items	(2,844)
Cash settled	(2,679)
Ending balance	3,665	0
Facility and Non-Lease Costs
Restructuring Reserve [Roll Forward]
Beginning balance	0
Restructuring charges	6,045
Non-cash items	(2,844)
Cash settled	(1,025)
Ending balance	2,176	0
Other Costs
Restructuring Reserve [Roll Forward]
Beginning balance	0
Restructuring charges	3,143
Non-cash items
Cash settled	(1,654)
Ending balance	$ 1,489	$ 0

Stockholders' Equity - Common S

Stockholders' Equity - Common Stock and Warrants (Details) $ / shares in Units, $ in Millions	1 Months Ended		12 Months Ended
	Oct. 31, 2023 USD ($) $ / shares shares	Nov. 30, 2022 USD ($)	Dec. 31, 2024 USD ($) vote $ / shares shares	Dec. 31, 2023 USD ($) $ / shares shares
Convertible Debt
Common stock, shares authorized (in shares)			500,000,000	500,000,000
Voting right for each share held, number | vote			1
Proceeds from issuance of private placement | $	$ 29.8
Common Stock
Convertible Debt
Stock issued from private placement (in shares)	2,467,104
At-The-Market
Convertible Debt
Sale of stock, maximum aggregate gross offering proceeds | $		$ 250
Number of shares issued, private placement (in shares)			1,732,204	5,244,936
Offering price per share (in dollars per share) | $ / shares			$ 11.69	$ 12.50
Aggregate net proceeds from stock offering | $			$ 19.6	$ 63.1
Amount remaining available to be issued and sold | $			$ 164.2
At-The-Market | Common Stock
Convertible Debt
Stock issued from private placement (in shares)			1,732,204	5,244,936
Private Placement
Convertible Debt
Offering price per share (in dollars per share) | $ / shares	$ 12.16
Private Placement | Common Stock
Convertible Debt
Stock issued from private placement (in shares)				2,467,104

Stockholders' Equity - Nonquali

Stockholders' Equity - Nonqualified Cash Deferral Plan and Equity Incentive Plans (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Deferred compensation investment	$ 2.2	$ 7.5
Expiration period	10 years
Vesting percentage	100%
Maximum | Options to purchase common stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of shares reserved for issuance (in shares)	14,228,754
First Anniversary
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting percentage	25%
Monthly vesting percentage	2.08%

Stock based Compensation - Narr

Stock based Compensation - Narrative (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Vesting option, vesting, anniversary period	2 years
Vesting option, exercisable, anniversary period	4 years
Weighted average grant-date fair value, options (in dollars per share)	$ 7.50	$ 6.75	$ 6.11
Aggregate intrinsic value of options exercised	$ 3,800	$ 7,100	$ 2,500
Proceeds from stock options exercised, net	7,677	$ 10,261	$ 4,310
Total unrecognized compensation costs	$ 38,600
Unvested restricted stock units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Period following separation for accelerated vesting	2 years
Unrecognized compensation costs, period for recognition (in years)	2 years
Unrecognized compensation cost	$ 55,100
Options to purchase common stock
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized compensation costs, period for recognition (in years)	2 years

Stock based Compensation - Weig

Stock based Compensation - Weighted-average Assumptions (Details) - Options to purchase common stock - $ / shares	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Expected stock price volatility	57%	59.20%	62.10%
Risk free interest rate	4%	3.90%	1.70%
Expected life of options (years)	5 years 7 months 6 days	5 years 5 months 19 days	5 years 4 months 2 days
Expected annual dividend per share (in dollars per share)	$ 0	$ 0	$ 0

Stock based Compensation - Stoc

Stock based Compensation - Stock Option Activity (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Number of Shares
Balance at the beginning of the period (in shares)	23,002	19,064	14,731
Options, Granted (in shares)	4,917	5,733	5,733
Options, Exercised (in shares)	(957)	(1,361)	(660)
Options, Forfeited (in shares)	(622)	(356)	(495)
Options, Expired (in shares)	(327)	(78)	(245)
Balance at the end of the period (in shares)	26,013	23,002	19,064
Vested and unvested expected to vest as of the end of the period (in shares)	24,643
Exercisable at the end of the period (in shares)	17,464
Weighted Average Exercise Price
Balance at the beginning of the period (in dollars per share)	$ 11.69	$ 11.31	$ 11.08
Options, Granted (in dollars per share)	13.52	11.99	11.53
Options, Exercised (in dollars per share)	8.02	7.54	6.58
Options, Forfeited (in dollars per share)	12.75	11.59	12.57
Options, Expired (in dollars per share)	14.30	15.09	12.84
Balance at the end of the period (in dollars per share)	12.11	$ 11.69	$ 11.31
Vested and unvested expected to vest as of the end of the period (in dollars per share)	12.09
Exercisable at the end of the period (in dollars per share)	$ 11.80
Weighted Average Remaining Years and Aggregate Intrinsic Value
Balance at the end of the period	5 years 9 months 18 days
Vested and unvested expected to vest at the end of the period	5 years 8 months 12 days
Exercisable at the end of the period	4 years 7 months 6 days
Aggregate intrinsic value of options outstanding	$ 5.8
Aggregate intrinsic value of options vested and unvested expected to vest	5.8
Aggregate intrinsic value of options exercisable	$ 5.7

Stock based Compensation - RSUs

Stock based Compensation - RSUs and PBRSUs Summary (Details) - Unvested restricted stock units - USD ($) $ / shares in Units, shares in Thousands, $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Number of Shares
Non-vested units as of the beginning of the period (in shares)	10,033	9,717	7,341
Granted (in shares)	4,150	4,762	5,048
Vested (in shares)	(5,107)	(3,610)	(2,251)
Forfeited (in shares)	(799)	(836)	(421)
Non-vested units as of the end of the period (in shares)	8,277	10,033	9,717
Weighted Average Grant Date Fair Value
Non-vested units as of the beginning of the period (in dollars per share)	$ 13.37	$ 13.07	$ 13.90
Granted (in dollars per share)	14.32	13.02	11.93
Vested (in dollars per share)	13.07	12.21	12.48
Forfeited (in dollars per share)	13.74	11.63	12.06
Non-vested units as of the end of the period (in dollars per share)	$ 13.59	$ 13.37	$ 13.07
Weighted Average Remaining Years and Aggregate Intrinsic Value
Non-vested units, weighted average remaining years	2 years 1 month 6 days
Non-vested units, aggregate intrinsic value	$ 78

Stock based Compensation - Expe

Stock based Compensation - Expense Summary (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 84,905	$ 86,077	$ 76,512
Research and development expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	15,969	21,470	25,089
Selling, general, and administrative expense
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total equity compensation expense	$ 68,936	$ 64,607	$ 51,423

Assets and Liabilities Measur_3

Assets and Liabilities Measured at Fair Value (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Assets:
Fair value of assets	$ 38,361	$ 46,686
Liabilities:
Deferred compensation plan liability	2,218	7,531
Fair value of liabilities	2,218	7,531
Commercial paper
Assets:
Fair value of assets	18,081	14,663
Treasury bill
Assets:
Fair value of assets		12,946
U.S. government agency bonds
Assets:
Fair value of assets	16,518	11,446
Corporate debt securities
Assets:
Fair value of assets	1,444
Money market
Assets:
Fair value of assets	2,318	7,631
Level 1
Assets:
Fair value of assets	2,318	7,631
Liabilities:
Deferred compensation plan liability	2,218
Fair value of liabilities	2,218
Level 1 | Commercial paper
Assets:
Fair value of assets	0	0
Level 1 | Treasury bill
Assets:
Fair value of assets		0
Level 1 | U.S. government agency bonds
Assets:
Fair value of assets	0	0
Level 1 | Corporate debt securities
Assets:
Fair value of assets	0
Level 1 | Money market
Assets:
Fair value of assets	2,318	7,631
Level 2
Assets:
Fair value of assets	36,043	39,055
Liabilities:
Deferred compensation plan liability	0	7,531
Fair value of liabilities	0	7,531
Level 2 | Commercial paper
Assets:
Fair value of assets	18,081	14,663
Level 2 | Treasury bill
Assets:
Fair value of assets		12,946
Level 2 | U.S. government agency bonds
Assets:
Fair value of assets	16,518	11,446
Level 2 | Corporate debt securities
Assets:
Fair value of assets	1,444
Level 2 | Money market
Assets:
Fair value of assets	$ 0	0
Level 2
Liabilities:
Deferred compensation plan liability		0
Fair value of liabilities		$ 0

Debt - Summary of Long Term Deb

Debt - Summary of Long Term Debt (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Debt Instruments
Net carrying value of long-term debt	$ 390,111	$ 387,858
Senior Loans | Senior Secured Term Loan due 2029:
Debt Instruments
Principal	400,000	400,000
Less: debt discount	(7,863)	(9,652)
Less: deferred financing	(2,026)	(2,490)
Net carrying value of long-term debt	$ 390,111	$ 387,858

Debt - Narrative (Details)

Debt - Narrative (Details)	1 Months Ended		12 Months Ended
	Oct. 31, 2023 USD ($) payment	Jul. 31, 2020 USD ($) payment	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)
Convertible Debt
Loss on extinguishment of debt			$ 0	$ (13,933,000)	$ 0
Secured Debt | Senior Loans
Convertible Debt
Number of payments | payment	12
Secured Debt | Senior Secured Term Loan due 2029: | Senior Loans
Convertible Debt
Maximum borrowing capacity	$ 400,000,000
Basis spread on variable rate	6.25%
Periodic payment	$ 33,300,000
Proceeds from secured lines of credit	$ 387,400,000
Secured Debt | Senior Secured Term Loan due 2029: | Senior Loans | Secured Overnight Financing Rate Floor
Convertible Debt
Basis spread on variable rate	2.50%
Secured Debt | Senior Secured Term Loan due 2029: | Senior Loans | Secured Overnight Financing Rate Adjustment
Convertible Debt
Basis spread on variable rate	0.26161%
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans
Convertible Debt
Maximum borrowing capacity		$ 400,000,000
Number of payments | payment		9
Periodic payment		$ 44,400,000
Proceeds from secured lines of credit	$ 408,900,000
Interest expense, debt	800,000
Early settlement premiums	$ 8,000,000
Secured Debt | Senior Secured Term Loan due 2026 | Senior Loans | LIBOR
Convertible Debt
Basis spread on variable rate		6.50%
Floor rate		1%

Debt - Interest Expense (Detail

Debt - Interest Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Interest expense
Contractual interest expense	$ 47,373	$ 47,626
Amortization of debt discount	1,790	1,605
Amortization of deferred financing	$ 472	$ 1,011
Senior Notes | Senior Secured Term Loan due 2029:
Interest expense
Effective interest rate of the liability component, Senior Secured Term Loan due 2029	12%	12.80%

Leases - Narrative (Details)

Leases - Narrative (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Leases [Abstract]
Operating lease expense	$ 6.7	$ 9.5
Variable lease cost	2.2	2.1
Operating lease payments	9.6	9.5
Right-of-use assets obtained in exchange for new operating lease liabilities	$ 0.7	$ 0.6

Leases - Supplemental Balance S

Leases - Supplemental Balance Sheet Information (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Leases [Abstract]
Operating lease ROU assets, net	$ 22,278	$ 26,312
Current portion of the operating lease liabilities	8,455	8,324
Non-current portion of the operating lease liabilities	45,078	48,877
Total operating lease liability	$ 53,533	$ 57,201
Weighted-average remaining lease terms (years)	8 years 6 months	9 years 4 months 24 days
Weighted-average discount rate	9.70%	9.70%

Leases - Future Minimum Operati

Leases - Future Minimum Operating Lease Payments (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Operating Lease
2025	$ 9,466
2026	9,356
2027	9,426
2028	9,139
2029	8,172
Thereafter	35,402
Total lease payments	80,961
Less: lease incentives	(22,299)
Less: imputed interest	(5,129)
Total operating lease liability	$ 53,533	$ 57,201

Income Taxes - Income Tax Expen

Income Taxes - Income Tax Expense (Benefit) and Reconciliation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Income (loss) before income taxes:
United States	$ (210,049)	$ (234,482)	$ (343,424)
Foreign	181,293	84,381	101,385
Loss before income tax	(28,756)	(150,101)	(242,039)
Current
Federal	0	0	0
State	76	14	6
Foreign	27,274	6,408	(5,760)
Deferred
Federal	0	(4,801)	274
State	0	(138)	9
Foreign	0	0	0
Total	$ 27,350	$ 1,483	$ (5,471)
Reconciliation of the statutory tax rates and the effective tax rates
Statutory rate	(21.00%)	(21.00%)	(21.00%)
Tax credits	(14.00%)	(8.00%)	(11.00%)
Impact of foreign operations	180%	(9.00%)	18%
Executive compensation	36%	7%	3%
Other	63%	1%	0%
Valuation allowance	(149.00%)	31%	9%
Net	95%	1%	(2.00%)

Income Taxes - Reconciliation o

Income Taxes - Reconciliation of Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Unrecognized Tax Benefits [Roll Forward]
Gross unrecognized tax benefits at beginning of year	$ 4,888	$ 283	$ 0
Gross increases for tax positions in prior years	9,542	4,605	283
Decreases for settlements and payments	(625)	0	0
Gross unrecognized tax benefits at end of year	$ 13,805	$ 4,888	$ 283

Income Taxes - Narrative (Detai

Income Taxes - Narrative (Details) - USD ($)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Operating Loss Carryforwards [Line Items]
Unrecognized tax benefits that would impact effective tax rate	$ 13,800,000	$ 4,800,000
Accrued interest	1,900,000
Accrual for penalties	0
Valuation allowance	609,130,000	$ 649,254,000
Decrease in the valuation allowance	40,200,000
Federal
Operating Loss Carryforwards [Line Items]
Amount of net operating loss carry forwards	1,100,000,000
Federal | Research and experimentation tax credit carryforward
Operating Loss Carryforwards [Line Items]
Tax credit carryforward amount	19,100,000
Federal | Orphan drug tax credit carryforwards
Operating Loss Carryforwards [Line Items]
Tax credit carryforward amount	201,600,000
State
Operating Loss Carryforwards [Line Items]
Amount of net operating loss carry forwards	1,000,000,000
State | Research and experimentation tax credit carryforward
Operating Loss Carryforwards [Line Items]
Tax credit carryforward amount	$ 13,500,000

Income Taxes - Deferred and Val

Income Taxes - Deferred and Valuation Allowance (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Deferred tax assets
Net operating loss carry forwards	$ 302,066	$ 321,004
Research tax credit	234,189	235,532
Capitalized research and development costs	34,883	34,425
Share-based compensation	20,045	17,314
Lease liability	10,026	10,567
Interest carry forward limitation	9,097	17,627
Inventory	1,599	1,931
Other	5,267	20,858
Gross deferred tax assets	617,172	659,258
Deferred tax liabilities
Other	(8,042)	(10,004)
Total net deferred tax assets	609,130	649,254
Less: valuation allowance	(609,130)	(649,254)
Net deferred tax liability	$ 0	$ 0

Collaborative Agreements (Detai

Collaborative Agreements (Details) $ in Thousands	1 Months Ended	12 Months Ended
	Nov. 30, 2013 USD ($) market	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)
Collaborative Agreements
Net product sales		$ 528,295	$ 399,356	$ 329,233
Accrued expenses and other current liabilities		127,300	$ 144,245
Revised Agreement | GSK
Collaborative Agreements
Potential milestone payments upon achievement of post-approval and sales-based milestones	$ 40,000
Number of major markets outside the U.S. from whom parties to contractual arrangement is eligible to receive post-approval and sales-based milestones | market	8
Deferred credits and other liabilities, noncurrent		4,700
Net product sales		32,700
Accrued expenses and other current liabilities		$ 9,800

Legal Proceedings (Details)

Legal Proceedings (Details)	1 Months Ended
	Nov. 30, 2022 lawsuit
Commitments and Contingencies Disclosure [Abstract]
Number of lawsuits	4

Basic and Diluted Net Loss pe_3

Basic and Diluted Net Loss per Common Share (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Numerator:
Net loss attributable to common stockholders	$ (56,106)	$ (151,584)	$ (236,568)
Denominator:
Weighted-average common shares outstanding - basic (in shares)	304,380,502	295,164,515	289,057,198
Weighted-average common shares outstanding - diluted (in shares)	304,380,502	295,164,515	289,057,198
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	34,290,000	33,035,000	28,781,000
Options to purchase common stock
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	26,013,000	23,002,000	19,064,000
Unvested restricted stock units
Antidilutive securities
Antidilutive securities excluded from computation of diluted earnings per share (in shares)	8,277,000	10,033,000	9,717,000

Basic and Diluted Net Loss pe_4

Basic and Diluted Net Loss per Common Share - Narrative (Details)	Dec. 31, 2024 $ / shares
Private Placement
Antidilutive securities
Warrants exercise price (in dollars per share)	$ 0.01