Document_and_Entity_Informatio
| Document and Entity Information | 9 Months Ended | |
| Sep. 30, 2014 | Nov. 04, 2014 | |
| Document and Entity Information [Abstract] | ' | ' |
| Entity Registrant Name | 'Raptor Pharmaceutical Corp | ' |
| Entity Central Index Key | '0001070698 | ' |
| Current Fiscal Year End Date | '--12-31 | ' |
| Entity Well-known Seasoned Issuer | 'No | ' |
| Entity Voluntary Filers | 'No | ' |
| Entity Current Reporting Status | 'Yes | ' |
| Entity Filer Category | 'Accelerated Filer | ' |
| Entity Common Stock, Shares Outstanding | ' | 62,742,871 |
| Document Fiscal Year Focus | '2014 | ' |
| Document Fiscal Period Focus | 'Q3 | ' |
| Document Type | '10-Q | ' |
| Amendment Flag | 'false | ' |
| Document Period End Date | 30-Sep-14 | ' |
CONDENSED_CONSOLIDATED_BALANCE
| CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (USD $) | Sep. 30, 2014 | Dec. 31, 2013 |
| In Thousands, unless otherwise specified | ||
| Current assets: | ' | ' |
| Cash and cash equivalents | $114,678 | $83,052 |
| Restricted cash | 894 | 500 |
| Accounts receivable | 10,479 | 6,181 |
| Inventories | 12,747 | 3,000 |
| Prepaid expenses and other | 4,464 | 3,566 |
| Total current assets | 143,262 | 96,299 |
| Noncurrent assets: | ' | ' |
| Fixed assets, net | 5,602 | 1,810 |
| Goodwill | 3,034 | 3,275 |
| Intangible assets, net | 3,275 | 3,213 |
| Other assets | 6,196 | 4,129 |
| Total Assets | 161,369 | 108,726 |
| Current liabilities: | ' | ' |
| Accounts payable | 3,376 | 5,264 |
| Accrued liabilities | 19,560 | 13,128 |
| Common stock warrant liability | 654 | 7,066 |
| Deferred revenue | 0 | 4,698 |
| Note payable, current portion | 6,000 | 0 |
| Total current liabilities | 29,590 | 30,156 |
| Noncurrent liabilities: | ' | ' |
| Note payable, net of current portion | 54,000 | 50,000 |
| Convertible debt | 60,000 | 0 |
| Total liabilities | 143,590 | 80,156 |
| Stockholders' equity: | ' | ' |
| Preferred stock, $0.001 par value per share,15,000,000 shares authorized, zero shares issued and outstanding | 0 | 0 |
| Common stock, $0.001 par value per share, 150,000,000 shares authorized, 63,268,534 and 61,614,576 shares issued and outstanding at September 30, 2014 and December 31, 2013, respectively | 63 | 62 |
| Additional paid-in capital | 256,810 | 234,286 |
| Accumulated other comprehensive loss | -122 | -423 |
| Accumulated deficit | -238,972 | -205,355 |
| Total stockholders' equity | 17,779 | 28,570 |
| Total Liabilities and Stockholders' Equity | $161,369 | $108,726 |
CONDENSED_CONSOLIDATED_BALANCE1
| CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited) (Parenthetical) (USD $) | Sep. 30, 2014 | Dec. 31, 2013 |
| Stockholders' Equity | ' | ' |
| Preferred stock, par value (in dollars per share) | $0.00 | $0.00 |
| Preferred stock, shares authorized (in shares) | 15,000,000 | 15,000,000 |
| Preferred stock, shares issued (in shares) | 0 | 0 |
| Preferred stock, shares outstanding (in shares) | 0 | 0 |
| Common stock, par value (in dollars per share) | $0.00 | $0.00 |
| Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 |
| Common stock issued (in shares) | 63,268,534 | 61,614,576 |
| Common stock, shares outstanding (in shares) | 63,268,534 | 61,614,576 |
CONDENSED_CONSOLIDATED_STATEME
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) (USD $) | 3 Months Ended | 9 Months Ended | ||
| In Thousands, except Share data, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 |
| CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited) [Abstract] | ' | ' | ' | ' |
| Revenues | $23,764 | $6,603 | $52,211 | $6,624 |
| Cost of sales | 3,917 | 442 | 6,226 | 867 |
| Gross profit | 19,847 | 6,161 | 45,985 | 5,757 |
| Operating expenses: | ' | ' | ' | ' |
| Research and development | 9,001 | 6,791 | 29,625 | 21,418 |
| Selling, general and administrative | 15,077 | 8,334 | 40,468 | 25,576 |
| Total operating expenses | 24,078 | 15,125 | 70,093 | 46,994 |
| Loss from operations | -4,231 | -8,964 | -24,108 | -41,237 |
| Interest income | 14 | 3 | 54 | 174 |
| Interest expense | -4,380 | -2,287 | -10,856 | -4,088 |
| Foreign currency transaction gain (loss) | 5 | 15 | 42 | -20 |
| Loss on short-term investments | 0 | 0 | 0 | -129 |
| Adjustment to fair value of common stock warrants | 206 | -6,044 | -1,091 | -12,025 |
| Other income | 2,346 | 0 | 2,346 | 0 |
| Net loss before provision for income taxes | -6,040 | -17,277 | -33,613 | -57,325 |
| Benefit from (provision for) income taxes | 9 | 0 | -4 | 0 |
| Net Loss | ($6,031) | ($17,277) | ($33,617) | ($57,325) |
| Net loss per share: | ' | ' | ' | ' |
| Basic and diluted (in dollars per share) | ($0.10) | ($0.29) | ($0.54) | ($1.01) |
| Weighted-average shares outstanding: | ' | ' | ' | ' |
| Basic and diluted (in shares) | 62,896,379 | 59,964,308 | 62,562,865 | 56,657,937 |
CONDENSED_CONSOLIDATED_STATEME1
| CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) (USD $) | 3 Months Ended | 9 Months Ended | ||
| In Thousands, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 |
| CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (Unaudited) [Abstract] | ' | ' | ' | ' |
| Net loss | ($6,031) | ($17,277) | ($33,617) | ($57,325) |
| Other comprehensive gain (loss): | ' | ' | ' | ' |
| Foreign currency translation gain (loss) | 103 | 11 | 301 | -78 |
| Comprehensive Loss | ($5,928) | ($17,266) | ($33,316) | ($57,403) |
CONDENSED_CONSOLIDATED_STATEME2
| CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited) (USD $) | 9 Months Ended | |
| In Thousands, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 |
| Cash flows from operating activities: | ' | ' |
| Net loss | ($33,617) | ($57,325) |
| Adjustments to reconcile net loss to net cash used in operating activities: | ' | ' |
| Stock-based compensation expense | 9,224 | 5,420 |
| Fair value adjustment of common stock warrants | 1,091 | 12,025 |
| Amortization of intangible assets | 179 | 134 |
| Depreciation of fixed assets | 411 | 152 |
| Realized gain on sale of fixed assets | 0 | -12 |
| Loss on short-term investments | 0 | 129 |
| Amortization of debt issuance cost | 966 | 289 |
| Changes in assets and liabilities: | ' | ' |
| Accounts receivable | -4,298 | -5,519 |
| Inventories | -9,747 | -2,218 |
| Prepaid expenses and other assets | -264 | -1,281 |
| Accounts payable | -1,888 | -2,752 |
| Accrued liabilities | 6,443 | 7,237 |
| Deferred revenue | -4,650 | 3,010 |
| Net cash used in operating activities | -36,150 | -40,711 |
| Cash flows from investing activities: | ' | ' |
| Net purchase of fixed assets | -4,262 | -965 |
| Purchase of short-term investments | 0 | -147 |
| Sale of short-term investments | 0 | 22,114 |
| Intangible assets | 0 | -1,250 |
| Change in restricted cash | -394 | -337 |
| Net cash (used in) provided by investing activities | -4,656 | 19,415 |
| Cash flows from financing activities: | ' | ' |
| Proceeds from sale of common stock under an ATM agreement | 0 | 38,392 |
| Proceeds from the exercise of common stock warrants | 1,826 | 9,809 |
| Proceeds from the exercise of common stock options | 4,023 | 1,516 |
| Proceeds from issuance of debt | 70,000 | 25,000 |
| Debt issuance costs | -3,521 | -1,259 |
| Offering costs | -147 | -63 |
| Net cash provided by financing activities | 72,181 | 73,395 |
| Effect of exchange rates on cash and cash equivalents | 251 | -78 |
| Net increase in cash and cash equivalents | 31,626 | 52,021 |
| Cash and cash equivalents, beginning of period | 83,052 | 36,313 |
| Cash and Cash Equivalents, End of Period | 114,678 | 88,334 |
| Supplemental cash flow information: | ' | ' |
| Interest paid | 9,356 | 2,314 |
| Income taxes paid | 250 | 0 |
| Supplemental disclosure of non-cash financing activities: | ' | ' |
| Fair value of warrant liability reclassified to equity upon exercise | $7,503 | $0 |
DESCRIPTION_OF_BUSINESS_AND_SU
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES [Abstract] | ' | ||||||||
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | ' | ||||||||
| 1. DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES | |||||||||
| The accompanying condensed consolidated financial statements reflect the financial position and results of operations of Raptor Pharmaceutical Corp. (the "Company" or "Raptor") and have been prepared in accordance with the accounting principles generally accepted in the United States of America ("GAAP") pursuant to the rules and regulations of the Securities and Exchange Commission (the "SEC"). Certain information and footnote disclosures have been condensed or omitted pursuant to such rules and regulations. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and in the opinion of management reflect all adjustments, which include only normal recurring adjustments, necessary for a fair presentation of the periods presented. The condensed consolidated balance sheet as of December 31, 2013 has been derived from the Company’s audited financial statements as of such date but does not include all disclosures required by GAAP. This Form 10-Q should be read in conjunction with the audited financial statements and accompanying notes in the Company's Annual Report on Form 10-K for the year ended December 31, 2013. | |||||||||
| Raptor is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat debilitating and often fatal diseases. The Company's first product, PROCYSBI® (cysteamine bitartrate) delayed-release capsules ("PROCYSBI"), received marketing approval from the U.S. Food and Drug Administration ("FDA") on April 30, 2013 for the management of nephropathic cystinosis in adults and children six years and older. In Europe, PROCYSBI® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), received marketing authorization on September 6, 2013 from the European Commission ("EC") as an orphan medicinal product for the management of proven nephropathic cystinosis for marketing in the European Union ("EU"). PROCYSBI received seven years and 10 years of market exclusivity as an orphan drug in the U.S. and the EU, respectively. The Company commenced commercial sales of PROCYSBI in the U.S. in mid-June 2013, in Germany in April 2014, and in Austria in August 2014. For at least the near term, the Company's ability to generate revenues is entirely dependent upon sales of PROCYSBI in the U.S. for the management of nephropathic cystinosis in adults and children six years and older and in the EU for the management of proven nephropathic cystinosis. | |||||||||
| Raptor's pipeline includes its proprietary delayed-release form of cysteamine, or RP103 and its proprietary oral 4-methylpyrazole, or Convivia™. Raptor currently has product candidates in clinical development designed to potentially treat Huntington's disease ("HD"), non-alcoholic steatohepatitis ("NASH"), Leigh syndrome and other mitochondrial disorders and aldehyde dehydrogenase deficiency ("ALDH2"). Raptor's preclinical programs are based upon bioengineered novel drug candidates that are designed to target cancer and other diseases. | |||||||||
| The Company is subject to a number of risks, including: the level of commercial sales of PROCYSBI in the U.S., Germany and Austria; the ability to successfully launch PROCYSBI in other international markets; uncertainty whether the Company's research and development efforts will result in expanded labeling for PROCYSBI and commercialization for RP103 in various indications or additional commercial products; competition from other organizations; reliance on licensing the proprietary technology of others; dependence on key personnel; uncertain patent protection; and the need to raise capital through equity and/or debt financings. Funding may not be available when needed if at all or on terms acceptable to the Company. If the Company exhausts its cash reserves and is unable to obtain adequate financing, it may be required to curtail planned operating expenditures, including its development programs. | |||||||||
| Basis of Presentation | |||||||||
| The Company's condensed consolidated financial statements include the accounts of the Company's direct and indirect wholly owned subsidiaries, Raptor Pharmaceuticals Inc. and Raptor European Products, LLC, such subsidiaries incorporated in Delaware on August 1, 2007, and February 14, 2012, respectively, and Raptor Pharmaceuticals Europe B.V. (“BV”), Raptor Pharmaceuticals France SAS (“SAS”), Raptor Pharmaceuticals Germany GmbH (“GMBH”) and RPTP European Holdings C.V. (“CV”), domiciled in the Netherlands on December 15, 2009, in France on October 30, 2012, in Germany on October 16, 2013 and in the Cayman Islands on February 16, 2012, respectively. All inter-company accounts have been eliminated. | |||||||||
| Use of Estimates | |||||||||
| The preparation of financial statements in conformity with U.S. GAAP requires management to make certain estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the condensed consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. | |||||||||
| Functional Currency | |||||||||
| The Company's consolidated functional currency is the U.S. dollar. BV, SAS, GMBH, and CV, the Company's Dutch, French, German and Cayman-based subsidiaries, respectively, use the European Euro as their functional currency. At each quarter end, each foreign subsidiary's balance sheet is translated into U.S. dollars based upon the quarter-end exchange rate, while their statements of operations are translated into U.S. dollars based upon an average exchange rate during the period. | |||||||||
| Segment Information | |||||||||
| The Company has determined that it operates in only one segment, as it only reports profit and loss information on an aggregate basis to its chief operating decision maker. The Company's long-lived assets maintained outside the U.S. are not material. | |||||||||
| Cash and Cash Equivalents | |||||||||
| The Company considers all highly liquid investments with original maturities of three months or less, when purchased, to be cash equivalents. The Company maintains cash and cash equivalents, which consist principally of money market funds with high credit quality financial institutions. Such amounts exceed Federal Deposit Insurance Corporation insurance limits. Restricted cash represents compensating balances required by the Company's U.S. and European banks as collateral for credit cards and for access to a value-added tax deferral program. As of September 30, 2014, the Company had $114.7 million in cash and cash equivalents, of which $4.1 million was held by its foreign subsidiaries. | |||||||||
| Fair Value of Financial Instruments | |||||||||
| The carrying amounts of certain of the Company's financial instruments including cash equivalents, restricted cash, accounts payable, accrued liabilities, note payable and capital lease liability approximate fair value due either to length of maturity or interest rates that approximate prevailing market rates. The warrant liability is carried at fair value, which is determined using the Black-Scholes option valuation model at the end of each reporting period. | |||||||||
| Revenue Recognition and Accounts Receivable | |||||||||
| The Company recognizes revenue in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification (“ASC”) 605, Revenue Recognition, when the following criteria have been met: persuasive evidence of an arrangement exists; delivery has occurred and risk of loss has passed; the seller's price to the buyer is fixed or determinable and collectability is reasonably assured. The Company determines that persuasive evidence of an arrangement exists based on written contracts that define the terms of the arrangements. Pursuant to the contract terms, the Company determines when title to products and associated risk of loss has passed on to the customer. The Company assesses whether the fee is fixed or determinable based on the payment terms associated with the transaction and whether the sales price is subject to refund or adjustment. The Company assesses collectability based primarily on the customer's payment history and creditworthiness. | |||||||||
| PROCYSBI is currently available for U.S. distribution from the Company's U.S. specialty pharmacy partner, the Accredo Health Group, Inc. ("Accredo") which is currently the Company's only U.S. customer and ships directly to patients. The Company's distributor in the EU is the Almac Group, Ltd. PROCYSBI is not available in U.S. retail pharmacies. Prior authorization of coverage by patients' commercial insurance plans, Raptor's patient assistance program ("PAP") or government payors is a prerequisite to the shipment of PROCYSBI to U.S. patients. Prior to the third quarter of 2014, revenue was recognized in the U.S. once the product had been shipped by the specialty pharmacy to patients because the Company had not yet been able to reasonably estimate the third-party payor mix and resulting rebates based on its lack of sufficient historical data. Beginning July 2014, the Company was able to reasonably estimate and determine sales allowances; therefore the Company began recognizing PROCYSBI revenue at the point of sale to the specialty distributor. As a result of the change, we recognized a net $4.3 million of net revenue in the third quarter of 2014, composed of $6.9 million of net revenue recognized in the third quarter of 2014 that would have been recognized in the fourth quarter of 2014, offset by $2.6 million of net revenue recognized in the third quarter of 2014 that would have been recognized in the second quarter of 2014. Revenue is currently recognized in the EU once confirmed orders from the German pharmacies have been placed and invoiced for payment by the distributor. | |||||||||
| The Company records revenue net of expected discounts, distributor fees, returns and rebates, including those paid to Medicare and Medicaid in the U.S. Allowances are recorded as a reduction of revenue at the time product sales are recognized. Allowances for government rebates and discounts are established based on the actual payor information, which is known in the U.S. at the time of shipment to patients, and the government mandated discount rates applicable to government-funded programs in the U.S. and Germany. The allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter the changes are known. | |||||||||
| Trade accounts receivable are recorded net of product sales allowances for prompt-payment discounts and chargebacks. Estimates for chargebacks and prompt-payment discounts are based on contractual terms and the Company's expectations regarding the utilization rates. | |||||||||
| Inventories and Cost of Sales | |||||||||
| Inventories are stated at the lower of cost or market price, with cost determined on a first-in, first-out basis. Inventories are reviewed periodically to identify slow-moving inventory based on sales activity, both projected and historical, as well as product shelf-life. Prior to the approval of PROCYSBI by the FDA on April 30, 2013 and in Europe, prior to the approval by the EC on September 6, 2013, the Company recorded the purchase of raw materials and the manufacturing costs relating to PROCYSBI as research and development expense. Subsequent to FDA and EC approval, the Company began capitalizing these costs and manufacturing overhead as commercial inventory. Upon launching PROCYSBI in mid-June 2013 in the U.S. and April 2014 in the EU, the Company began recognizing cost of sales. Cost of sales includes the cost of inventory sold or reserved; manufacturing, manufacturing overhead and supply chain costs; product shipping and handling costs; amortization of licensing approval milestone payments and licensing royalties payable to the University of California, San Diego ("UCSD"). | |||||||||
| The Company capitalizes inventory produced in preparation for product launches when positive results have been obtained for the clinical trials that the Company believes are necessary to support regulatory approval and the Company has determined it is probable that these capitalized costs will provide some future economic benefit in excess of capitalized costs or when the inventory is identical in nature to existing commercial inventory and thus has an "alternative future use". For these inventories, the Company also considers the expected approval date in assessing realizability. To the extent that inventory is expected to expire prior to being sold, the Company will write down the value of inventory. If actual results differ from those estimates, additional inventory write-offs may be required. | |||||||||
| Impairment of Goodwill and Intangible Assets | |||||||||
| Goodwill represents the excess of the purchase price over the fair value of tangible and identified intangible net assets of businesses acquired. Goodwill is not amortized, but is evaluated for impairment on an annual basis or more often when impairment indicators are present. The Company has one reporting unit. Therefore, the Company's consolidated net assets, including existing goodwill and other intangible assets, are considered to be the carrying value of the reporting unit. If the carrying value of the reporting unit is in excess of its fair value, an impairment may exist, and the Company must perform the second step of the analysis, in which the implied fair value of the goodwill is compared to its carrying value to determine the impairment charge, if any. If the estimated fair value of the reporting unit exceeds the carrying value of the reporting unit, goodwill is not impaired and no further analysis is required. | |||||||||
| The Company makes judgments about the recoverability of purchased intangible assets with finite lives whenever events or changes in circumstances indicate that impairment may exist. Recoverability of purchased intangible assets with finite lives is measured by comparing the carrying amount of the asset to the future undiscounted cash flows the asset is expected to generate. Impairment, if any, is measured as the amount by which the carrying value exceeds the fair value of the impaired asset. | |||||||||
| Fixed Assets | |||||||||
| Fixed assets, which mainly consist of leasehold improvements, office furniture, manufacturing and lab equipment and computer hardware and software, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements and capital lease equipment, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred. | |||||||||
| Common Stock Warrant Liabilities | |||||||||
| The Company issued warrants that contain conditional obligations that may require the Company to transfer cash to settle the warrants upon the occurrence of certain fundamental transactions. Therefore, the Company has classified the warrants as liabilities. The Company re-measures the liability at the end of every reporting period with the change in value reported in the Company's condensed consolidated statements of operations. At the exercise date, the fair values of these warrants are re-measured and reclassified to equity. | |||||||||
| Note Payable | |||||||||
| Note payable consists of a loan agreement with HealthCare Royalty Partners II, L.P. ("HC Royalty"), as lender, which was amended effective July 1, 2014. The amendment qualified as a modification of debt in accordance with ASC 470-50, Debt – Modifications and Extinguishments, as we determined it did not result in substantially different terms. The amended loan requires quarterly interest payments at an annual fixed interest rate of 8.0% of outstanding principal and includes a synthetic royalty component based on net product sales, including PROCYSBI, in a calendar year. Prior to July 1, 2014, the original loan bore interest at an annual fixed rate of 10.75% of outstanding principal and included a synthetic royalty component based on net product sales, including PROCYSBI, in a calendar year. | |||||||||
| Note payable is carried at its unpaid principal balance. The fixed and royalty interest under both agreements were recognized as interest expense as incurred. | |||||||||
| Convertible Notes | |||||||||
| Convertible notes include unsecured convertible senior notes and are carried at their unpaid principal balance. Interest on the notes is payable quarterly and the notes mature on August 1, 2019. If converted by a holder, upon conversion, the holder of the notes would receive shares of the Company’s common stock. | |||||||||
| Debt Issuance Costs | |||||||||
| Debt issuance costs are expenses associated with the issuance of the loan agreements with HC Royalty and the convertible notes. Debt issuance costs which were capitalized are being amortized over the life of the respective debt to interest expense using the interest method. Debt issuance costs are a component of other assets on our condensed consolidated balance sheets. | |||||||||
| Research and Development Costs | |||||||||
| Research and development costs are charged to expense as incurred. Research and development expenses primarily include salaries and benefits for medical, clinical, regulatory, quality, pharmacovigilance and research personnel, preclinical studies, clinical trials and certain commercial drug manufacturing expenses prior to obtaining marketing approval. | |||||||||
| Net Loss per Share | |||||||||
| Net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include: | |||||||||
| Nine Months Ended September 30, | |||||||||
| 2014 | 2013 | ||||||||
| Warrants to purchase common stock | 334,764 | 1,145,170 | |||||||
| Options to purchase common stock | 9,287,302 | 8,229,927 | |||||||
| Convertible debt | 3,428,571 | - | |||||||
| Total Potentially Dilutive Securities | 13,050,637 | 9,375,097 | |||||||
| Comprehensive Loss | |||||||||
| The components of comprehensive loss include net loss and foreign currency translation adjustments. | |||||||||
| Stock-Based Compensation | |||||||||
| Stock Option Plan | |||||||||
| Compensation costs related to the Company's stock option plan are measured at the grant date based on the fair value of the equity instruments awarded and are recognized over the period during which an employee is required to provide service in exchange for the award, or the requisite service period, which is usually the vesting period. The compensation expense for stock-based compensation awards is reduced by an estimate for forfeitures. | |||||||||
| The Company recognizes expense associated with stock options issued to third parties, including consultants, based upon the fair value of such awards on the date the options vest. | |||||||||
| Employee Stock Purchase Plan | |||||||||
| In July 2014, the Company’s shareholders approved the Raptor Pharmaceutical Corp. 2013 Employee Stock Purchase Plan (“ESPP”). Up to 1,000,000 shares may be issued pursuant to the ESPP. The purpose of the ESPP is to give the Company’s employees an opportunity to acquire an equity interest in the Company through the purchase of shares of common stock at a discount. The ESPP allows eligible employees to purchase common stock at 85% of its fair value, subject to certain limits. Fair value as defined under the ESPP is the lesser of the closing market price of the common stock on the first day of the offering period or the last day of the offering period. | |||||||||
| Income Taxes | |||||||||
| Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized. Based on the weight of available evidence, including cumulative losses since inception and expected future losses, the Company has determined that it is more likely than not that the deferred tax asset amount will not be realized and therefore a full valuation allowance has been provided on the Company's net deferred tax assets. | |||||||||
| The Company identifies uncertain tax positions and discloses any potential tax liability on its financial statements. The Company recognizes interest and/or penalties related to income tax matters as a component of income tax expense. As of September 30 2014, there were no accrued uncertain tax positions or interest and penalties related to uncertain tax positions. | |||||||||
| The Company files U.S. Federal, California, various other state and other income tax returns and various foreign country income tax returns. The Company is currently not subject to any income tax examinations. Due to the Company's net operating losses ("NOLs"), generally all tax years remain open. | |||||||||
| Reclassifications | |||||||||
| Certain amounts previously reported under specific financial statement captions have been reclassified to be consistent with the current year presentation. | |||||||||
| Recent Accounting Pronouncements | |||||||||
| In May 2014, the FASB issued Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers, which outlines a single comprehensive model for entities to use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance, including industry-specific guidance. The core principle of the revenue model is that “an entity recognizes revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services.” In applying the revenue model to contracts within its scope, the Company will: identify the contract(s) with a customer, identify the performance obligations in the contract, determine the transaction price, allocate the transaction price to the performance obligations in the contract, and recognize revenue when (or as) the entity satisfies a performance obligation. This ASU is effective for interim and annual periods beginning after December 15, 2016 and early adoption is not permitted. The Company does not believe the adoption of this ASU will have a material impact on its consolidated financial statements. | |||||||||
| In June 2014, the FASB issued ASU 2014-12, Accounting for Share-Based Payments When the Terms of an Award Provide That a Performance Target Could Be Achieved after the Requisite Service Period. The ASU requires that a performance target that affects vesting, and that could be achieved after the requisite service period, be treated as a performance condition. A reporting entity should apply existing guidance in Topic 718 as it relates to awards with performance conditions that affect vesting to account for such awards. This ASU is effective for interim and annual periods beginning after December 15, 2015 and early adoption is permitted. The Company does not anticipate the adoption of this ASU will have a material impact on its consolidated financial statements. | |||||||||
FAIR_VALUE_MEASUREMENT
| FAIR VALUE MEASUREMENT | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| FAIR VALUE MEASUREMENT [Abstract] | ' | ||||||||||||||||
| FAIR VALUE MEASUREMENT | ' | ||||||||||||||||
| 2. FAIR VALUE MEASUREMENT | |||||||||||||||||
| The Company uses a fair value approach to value certain assets and liabilities. Fair value is the price that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. The Company uses a fair value hierarchy, which distinguishes between assumptions based on market data (observable inputs) and an entity's own assumptions (unobservable inputs). The hierarchy consists of three levels: | |||||||||||||||||
| • | Level 1 – Quoted market prices in active markets for identical assets or liabilities; | ||||||||||||||||
| • | Level 2 – Inputs other than level one inputs that are either directly or indirectly observable; and | ||||||||||||||||
| • | Level 3 – Unobservable inputs developed using estimates and assumptions, which are developed by the reporting entity and reflect those assumptions that a market participant would use. | ||||||||||||||||
| Determining which category an asset or liability falls within the hierarchy requires significant judgment. The Company evaluates its hierarchy disclosures each quarter. Assets and liabilities measured at fair value on a recurring basis at September 30, 2014 and December 31, 2013 are summarized as follows: | |||||||||||||||||
| (In thousands) | |||||||||||||||||
| 30-Sep-14 | Level 1 | Level 2 | Level 3 | Total | |||||||||||||
| Assets | |||||||||||||||||
| Cash equivalents (1) | $ | 109,610 | $ | - | $ | - | $ | 109,610 | |||||||||
| Total | $ | 109,610 | $ | - | $ | - | $ | 109,610 | |||||||||
| Liabilities | |||||||||||||||||
| Common stock warrants | $ | - | $ | - | $ | 654 | $ | 654 | |||||||||
| Total | $ | - | $ | - | $ | 654 | $ | 654 | |||||||||
| 31-Dec-13 | Level 1 | Level 2 | Level 3 | Total | |||||||||||||
| Assets | |||||||||||||||||
| Cash equivalents (1) | $ | 70,627 | $ | - | $ | - | $ | 70,627 | |||||||||
| Total | $ | 70,627 | $ | - | $ | - | $ | 70,627 | |||||||||
| Liabilities | |||||||||||||||||
| Common stock warrants | $ | - | $ | - | $ | 7,066 | $ | 7,066 | |||||||||
| Total | $ | - | $ | - | $ | 7,066 | $ | 7,066 | |||||||||
| (1) Cash equivalents represent the fair value of the Company's investments in money market funds at September 30, 2014 and December 31, 2013. | |||||||||||||||||
| Certain of the Company's common stock warrants are classified as liabilities and are therefore re-measured using the Black-Scholes option valuation model at the end of each reporting period with the change in value reported in the Company's condensed consolidated statements of operations. | |||||||||||||||||
| The following table presents a reconciliation of the Company's recurring fair value measurements categorized within Level 3 of the fair value hierarchy (liability-classified common stock warrants): | |||||||||||||||||
| Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Common Stock Warrants | |||||||||||||||||
| (In thousands) | Nine Months Ended | ||||||||||||||||
| 30-Sep-14 | |||||||||||||||||
| Fair value as of December 31, 2013 | $ | 7,066 | |||||||||||||||
| Change in fair value recognized in earnings | 1,091 | ||||||||||||||||
| Exercises | (7,503 | ) | |||||||||||||||
| Fair Value as of September 30, 2014 | $ | 654 | |||||||||||||||
| Effect of Raptor's Stock Price and Volatility Assumptions on the Calculation of Fair Value of Warrant Liabilities | |||||||||||||||||
| As discussed above, the Company uses the Black-Scholes option pricing model as its method of valuation for warrants that are subject to warrant liability accounting. The determination of fair value as of the reporting date is affected by Raptor's stock price as well as assumptions regarding a number of subjective variables. These variables include, but are not limited to, expected stock price volatility over the term of the security and risk-free interest rate. The primary factors affecting the fair value of the warrant liability are the Company's stock price and volatility. | |||||||||||||||||
INTANGIBLE_ASSETS_AND_GOODWILL
| INTANGIBLE ASSETS AND GOODWILL | 9 Months Ended | ||||||||||||
| Sep. 30, 2014 | |||||||||||||
| INTANGIBLE ASSETS AND GOODWILL [Abstract] | ' | ||||||||||||
| INTANGIBLE ASSETS AND GOODWILL | ' | ||||||||||||
| 3. INTANGIBLE ASSETS AND GOODWILL | |||||||||||||
| On December 14, 2007, the Company acquired the intellectual property and other rights to develop RP103 to treat various clinical indications from UCSD through a merger with Encode Pharmaceuticals, Inc., a privately held development stage company ("Encode"), which held the intellectual property license with UCSD. The fair value of the intangible assets at the time of acquisition was approximately $2.6 million. | |||||||||||||
| Pursuant to the license agreement with UCSD, the Company is obligated to pay an annual maintenance fee until the commencement of commercial sales of any licensed products developed. The Company is also obligated to pay milestone payments upon the occurrence of certain events, royalties on net sales from products developed pursuant to the license agreement and a percentage of sublicense fees or royalties, if any. The Company is obligated to fulfill predetermined milestones within a specified number of years from the effective date of the license agreement, depending on the indication. To the extent that the Company fails to perform any of the obligations, UCSD may terminate the license or otherwise cause the license to become non-exclusive. | |||||||||||||
| In April 2013, the Company received FDA approval of PROCYSBI (cysteamine bitartrate) delayed release capsules for the management of nephropathic cystinosis in adults and children 6 years and older. Subsequently, the Company announced that the EC had approved PROCYSBI gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) as an orphan medicinal product for the management of proven nephropathic cystinosis for marketing in the EU. In conjunction with these approvals, the Company paid milestone payments to UCSD during the second and third quarters of 2013 of $0.8 million and $0.5 million, respectively, pursuant to this license, which were capitalized as intangible assets. | |||||||||||||
| A summary of intangibles acquired is as follows: | |||||||||||||
| Useful Life | September 30, | December 31, | |||||||||||
| (In thousands) | (Years) | 2014 | 2013 | ||||||||||
| Intangible asset (IP license for RP103) related to the Encode merger | 20 | $ | 2,620 | $ | 2,620 | ||||||||
| Intangible assets (UCSD license - FDA and EC approval milestones) | 20 | 1,250 | 1,250 | ||||||||||
| Other intangible assets | 16 | 240 | 240 | ||||||||||
| Total intangible assets | 4,110 | 4,110 | |||||||||||
| Less accumulated amortization | (1,076 | ) | (897 | ) | |||||||||
| Intangible Assets, Net | $ | 3,034 | $ | 3,213 | |||||||||
| The intangible assets related to RP103 are being amortized over an estimated useful life of 20 years, which is the life of the intellectual property patents. The 20-year estimated useful life is also based upon the typical development, approval, marketing and life cycle management timelines of pharmaceutical drug products. Other intangible assets are being amortized using the straight-line method over an estimated useful life of 16 years, which is the life of the intellectual property patents. | |||||||||||||
| During the nine months ended September 30, 2014 and year ended December 31, 2013, there was no intangible asset impairment recognized. During the three and nine months ended September 30, 2014, the Company amortized approximately $37 thousand and $110 thousand, respectively, of intangible assets to research and development expense. During the three and nine months ended September 30, 2013, the Company amortized approximately $47 thousand and $134 thousand, respectively, of intangible assets to research and development expense. | |||||||||||||
| Amortization expense for intangible assets for each of the next five years is expected to be as follows: | |||||||||||||
| (In thousands) | Amortization Expense | ||||||||||||
| 2014 (remaining 3 months) | $ | 60 | |||||||||||
| 2015 | 238 | ||||||||||||
| 2016 | 238 | ||||||||||||
| 2017 | 238 | ||||||||||||
| 2018 | 238 | ||||||||||||
| The Company tested the carrying value of goodwill for impairment as of December 31, 2013 and determined that there was no impairment. | |||||||||||||
INVENTORIES
| INVENTORIES | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| INVENTORIES [Abstract] | ' | ||||||||
| INVENTORIES | ' | ||||||||
| 4. INVENTORIES | |||||||||
| Inventories consist of raw materials, work-in-process and finished goods related to the manufacture of PROCYSBI and early access program inventory. Raw materials include the Company's active pharmaceutical ingredient ("API"), cysteamine bitartrate. Work-in-process includes third party manufacturing and associated labor costs relating to the Company's personnel directly involved in the production process. Also included in inventories are raw materials and work-in-process that may be used for clinical trials, which are charged to research and development (“R&D”) expense when consumed. | |||||||||
| Inventories are summarized as follows: | |||||||||
| September 30, | December 31, | ||||||||
| (In thousands) | 2014 | 2013 | |||||||
| Raw materials | $ | 8,540 | $ | 2,469 | |||||
| Work-in-process | 444 | - | |||||||
| Early access program | 1,801 | - | |||||||
| Finished goods | 1,962 | 531 | |||||||
| Total Inventories | $ | 12,747 | $ | 3,000 | |||||
FIXED_ASSETS
| FIXED ASSETS | 9 Months Ended | ||||||||||||
| Sep. 30, 2014 | |||||||||||||
| FIXED ASSETS [Abstract] | ' | ||||||||||||
| FIXED ASSETS | ' | ||||||||||||
| 5. FIXED ASSETS | |||||||||||||
| Fixed assets consisted of: | |||||||||||||
| September 30, | December 31, | Estimated | |||||||||||
| (In thousands) | 2014 | 2013 | useful lives | ||||||||||
| Laboratory equipment | $ | 1,484 | $ | 1,132 | 5 years | ||||||||
| Assets under construction | 2,388 | 102 | - | ||||||||||
| Computer hardware and software | 780 | 646 | 3 years | ||||||||||
| Office furniture | 1,835 | 605 | 7 years | ||||||||||
| Leasehold improvements | 260 | - | Lease term | ||||||||||
| Total at cost | 6,747 | 2,485 | |||||||||||
| Less: accumulated depreciation | (1,145 | ) | (675 | ) | |||||||||
| Total Fixed Assets, Net | $ | 5,602 | $ | 1,810 | |||||||||
NOTE_PAYABLE
| NOTE PAYABLE | 9 Months Ended |
| Sep. 30, 2014 | |
| NOTE PAYABLE [Abstract] | ' |
| NOTE PAYABLE | ' |
| 6. NOTE PAYABLE | |
| On December 20, 2012, the Company entered into a loan agreement with HC Royalty, as lender, under which it agreed to borrow $50.0 million in two $25.0 million tranches. The Company received $23.4 million in net proceeds from the first tranche of the loan at closing in December 2012 and an additional $23.7 million in net proceeds in May 2013 from the second tranche upon FDA approval of PROCYSBI. | |
| In July 2014, the Company entered into an amended and restated loan agreement with HC Royalty which revises the terms of the 2012 loan agreement between the Company and HC Royalty and also provided for an additional $10 million in term loan funding. The interest rate was revised to an annual fixed rate of 8.0%, compared to the original interest rate of 10.75%. The loan also contains a synthetic royalty component based on net product revenues, including PROCYSBI, in a calendar year, and such royalty is payable quarterly. The variable royalty rate under the amended and restated loan agreement has been revised to 8.0% on the first $50 million of revenue and 2.0% on revenue in excess of $50 million. The first principal payment of $3 million is due in June 2015. All term loans under the amended and restated loan agreement mature on March 31, 2020. The loan and the Company's obligation to make payments thereunder shall terminate immediately when all payments received by HC Royalty equal $120.0 million. | |
| Prior to July 1, 2014, with respect to the first $25.0 million tranche, for each calendar year (prorated for any portion thereof), the loan bore a royalty rate of 6.25% of the first $25.0 million of product net revenues, 3.0% of product net revenues for such calendar year in excess of $25.0 million and up to $50.0 million, and 1.0% of product net revenues for such calendar year in excess of $50.0 million, payable quarterly. Prior to July 1, 2014, with respect to the second $25.0 million tranche, for each calendar year (prorated for any portion thereof), the loan bore a royalty rate of 6.0% of the first $25.0 million of net product revenues for such calendar year, 3.0% of product net revenues for such calendar year in excess of $25.0 million and up to $50.0 million, and 1.0% of product net revenues for such calendar year in excess of $50.0 million, payable quarterly. | |
| The Company's amended and restated loan agreement with HC Royalty includes affirmative and negative covenants, including the use of commercially reasonable efforts to exploit RP103 in specific markets and compliance with laws, as well as restrictions on mergers and sales of assets, incurrence of liens, incurrence of indebtedness and transactions with affiliates and other requirements. To secure the performance of the Company's obligations under the loan, the Company granted a security interest to HC Royalty in substantially all of its assets, the assets of its domestic subsidiaries and a pledge of stock of certain of its domestic subsidiaries. The Company's failure to comply with the terms of the loan and related documents, the occurrence of a change of control of the Company or the occurrence of an uncured material adverse effect on the Company or the occurrence of certain other specified events, will result in an event of default under the loan that, if not cured or waived, could result in the acceleration of the payment of all of its indebtedness, as well as prepayment penalties, to HC Royalty and interest thereon. Under the terms of the security agreement, in an event of default, the lender can potentially take possession of, foreclose on, sell, assign or grant a license to use, the Company's pledged collateral and assign and transfer the pledged stock of certain of its subsidiaries. | |
| The Company received marketing approval of PROCYSBI from the FDA on April 30, 2013 and commenced shipment of PROCYSBI during June 2013, and as a result, royalties became payable to HC Royalty based upon net revenues of PROCYSBI. Interest expense on the loan, excluding amortization of debt issuance costs, for the three months ended September 30, 2014 and 2013 was $2.8 million and $2.1 million, respectively. Interest expense on the loan, excluding amortization of debt issuance costs, for the nine months ended September 30, 2014 and 2013 was $9.0 million and $3.8 million, respectively. | |
| Unamortized debt issuance costs on the loan agreement totaled $2.4 million and $2.8 million as of September 30, 2014 and December 31, 2013, respectively. Amortization expense for the nine months ended September 30, 2014 and 2013 was $0.9 million and $0.3 million, respectively. |
CONVERTIBLE_NOTES
| CONVERTIBLE NOTES | 9 Months Ended |
| Sep. 30, 2014 | |
| CONVERTIBLE NOTES [Abstract] | ' |
| CONVERTIBLE NOTES | ' |
| 7. CONVERTIBLE NOTES | |
| In July 2014, the Company sold $60 million aggregate principal amount of 8.0% convertible senior notes due 2019. These convertible notes require quarterly interest distributions at a fixed coupon rate equal of 8.0% until maturity or conversion, which will be no later than August 1, 2019. The convertible senior notes are convertible at the option of the holder at a conversion rate of 57.14 common shares per $1,000 principal amount of convertible senior notes at issuance (equivalent to a conversion price of $17.50 per common share), subject to adjustment in certain events. Upon conversion of these convertible senior notes by a holder, the holder will receive shares of the Company's common stock. | |
| In addition, the Company may elect to exercise the optional redemption, as defined in the note purchase agreement, in which case the convertible senior notes will convert into shares of common stock if the price of the common stock is at or above 175% of the applicable conversion price over a 30 consecutive day period. Upon the occurrence of a "change of control", as defined in the note purchase agreement, the holders may require the Company to repurchase all or a portion of the notes for cash at 100% of the principal amount of the notes being purchased, plus a repayment premium and any accrued and unpaid interest. To secure the performance of the Company's obligations under the convertible notes agreement, the Company has assigned certain of its assets as collateral. | |
| Interest expense on convertible notes, excluding amortization of debt issuance costs, was $0.9 million for the three months ended September 30, 2014. Unamortized debt issuance costs on these convertible notes totaled $2.9 million at September 30, 2014. Amortization expense for the three months ended September 30, 2014 was $0.1 million. |
ACCRUED_LIABILITIES
| ACCRUED LIABILITIES | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| ACCRUED LIABILITIES [Abstract] | ' | ||||||||
| ACCRUED LIABILITIES | ' | ||||||||
| 8. ACCRUED LIABILITIES | |||||||||
| Accrued liabilities consisted of: | |||||||||
| September 30, | December 31, | ||||||||
| (In thousands) | 2014 | 2013 | |||||||
| Personnel-related costs | $ | 4,770 | $ | 4,443 | |||||
| Rebates and other sales deductions | 3,545 | 2,325 | |||||||
| Clinical trials and research and development costs | 2,153 | 1,661 | |||||||
| Royalty-based interest payable | 1,437 | 1,255 | |||||||
| License royalty payable | 925 | 564 | |||||||
| Manufacturing costs | 2,611 | 294 | |||||||
| Other | 2,616 | 2,586 | |||||||
| Total Accrued Liabilities | $ | 18,057 | $ | 13,128 | |||||
CAPITAL_STRUCTURE
| CAPITAL STRUCTURE | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| CAPITAL STRUCTURE [Abstract] | ' | ||||||||||||||||
| CAPITAL STRUCTURE | ' | ||||||||||||||||
| 9. CAPITAL STRUCTURE | |||||||||||||||||
| Preferred Stock | |||||||||||||||||
| At September 30, 2014, the Company was authorized to issue 15,000,000 shares of $0.001 par value per share of preferred stock. There were no shares issued and outstanding. | |||||||||||||||||
| Common Stock | |||||||||||||||||
| At September 30, 2014, the Company was authorized to issue 150,000,000 shares of $0.001 par value per share of common stock. Each holder of common stock is entitled to vote on all matters and is entitled to one vote for each share held. As of September 30, 2014 and December 31, 2013, there were 63,268,534 and 61,614,576 shares, respectively, of the Company's common stock issued and outstanding. | |||||||||||||||||
| Stockholder Rights Plan | |||||||||||||||||
| The Company’s stockholder rights plan entitles the holder of each outstanding share of common stock of the Company to one stock purchase right (a “Right”). Each Right entitles the registered holder to purchase from the Company one thousandth of a share of the Company’s Series A Participating Preferred Stock (the “Preferred Shares”) at a price of $15 per one one-thousandth of a Preferred Share (the “Purchase Price”), once the Rights become exercisable. The Rights will not be exercisable until the earlier of either (a) 10 days after the public announcement that a person, together with all affiliates or associates of such person, has become an “Acquiring Person” by obtaining beneficial ownership of 15% or more of the Company’s outstanding common stock, or (b) 10 business days (or a later date determined by the Board before any person or group becomes an Acquiring Person) after a person or group of affiliated or associated persons begins a tender or exchange offer which, if completed, would result in that person or group of affiliated or associated persons becoming an Acquiring Person. Each one one-thousandth of a share preferred stock, if issued, will have the same voting power as one one-hundred thirty-sixth (1/136th) of a share of common stock and will entitle holders to a per share payment equal to the payment made on one one-hundred thirty-sixth (1/136th) of a share of common stock, so that one full share of preferred stock would be entitled to receive a payment one one-hundred thirty-sixth (1/136th) of 1,000 times the per share payment to a share of common stock, provided that shares of the Company’s common stock are exchanged via merger, consolidation or a similar transaction. The Rights will expire on May 13, 2015 or on an earlier date if the Company redeems or exchanges the Rights. | |||||||||||||||||
| Common Stock Issuance under At-The-Market ("ATM") Agreement | |||||||||||||||||
| On April 30, 2012, the Company entered into an "At-the-Market" ("ATM") Sales Agreement, with Cowen and Company, LLC ("Cowen"), under which the Company may, at its discretion, sell its common stock with a sales value of up to a maximum of $40.0 million through ATM offerings on the NASDAQ Stock Market. On July 3, 2013, the Company and Cowen amended and restated the Sales Agreement (the "Amended and Restated Sales Agreement") to increase the aggregate gross sales proceeds that may be raised to $100 million. Cowen is the sole sales agent for any sales made under the ATM for a 3.0% commission on gross proceeds. The common stock is sold at prevailing market prices at the time of the sale of common stock, and, as a result, prices will vary. During the nine months ended September 30, 2014, there were no shares sold under the ATM. As of September 30, 2014, the Company had used approximately $53.8 million under the ATM. | |||||||||||||||||
| Common Stock Warrants | |||||||||||||||||
| During the nine months ended September 30, 2014, the Company received approximately $1.8 million from the exercise of warrants in exchange for the issuance of 611,606 shares of the Company's common stock. | |||||||||||||||||
| The number of common stock warrants outstanding as of September 30, 2014 were as follows: | |||||||||||||||||
| Number of Shares Exercisable | |||||||||||||||||
| Exercise | Expiration | ||||||||||||||||
| Price | Date | ||||||||||||||||
| Issued in connection with Encode merger | 233,309 | $ | 2.87 | 12/13/15 | |||||||||||||
| TorreyPines warrants assumed in 2009 Merger | 3,502 | 157.08 | 9/26/15 | ||||||||||||||
| Issued to registered direct investors in Dec. 2009 | 1 | 2.45 | 12/22/14 | ||||||||||||||
| Issued to placement agent in Aug. 2010 | 97,952 | 3.08 | 8/12/15 | ||||||||||||||
| Total Warrants Outstanding | 334,764 | $ | 4.54 | -1 | |||||||||||||
| (1) Weighted average exercise price | |||||||||||||||||
| The warrants issued by the Company in the August 2010 private placement and the December 2009 equity financing contain a conditional obligation that may require the Company to transfer assets to repurchase the warrants upon the occurrence of potential future events. Under ASC 480, a financial instrument that may require the issuer to settle the obligation by transferring assets is classified as a liability. Therefore, the Company has classified the warrants from both financings as liabilities and marks them to fair value at each period end. | |||||||||||||||||
| A Black-Scholes option-pricing model was used to obtain the fair value of the warrant liabilities using the following assumptions at September 30, 2014 and December 31, 2013: | |||||||||||||||||
| December 2009 Equity Financing Series A | August 2010 Private Placement Investors and Placement Agent | ||||||||||||||||
| September 30, | December 31, | September 30, | December 31, | ||||||||||||||
| 2014 | 2013 | 2014 | 2013 | ||||||||||||||
| Fair value (in thousands) | $ | - | $ | 133 | $ | 654 | $ | 6,933 | |||||||||
| Black-Scholes inputs: | |||||||||||||||||
| Stock price | $ | 9.59 | $ | 13.02 | $ | 9.59 | $ | 13.02 | |||||||||
| Exercise price | $ | 2.45 | $ | 2.45 | $ | 3.08 | $ | 3.08 | |||||||||
| Risk free interest rate | 0.01 | % | 0.13 | % | 0.08 | % | 0.33 | % | |||||||||
| Volatility | 95 | % | 95 | % | 95 | % | 95 | % | |||||||||
| Expected term (years) | 0.25 | 1 | 0.75 | 1.75 | |||||||||||||
| Dividend | - | - | - | - | |||||||||||||
STOCKBASED_COMPENSATION
| STOCK-BASED COMPENSATION | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| STOCK-BASED COMPENSATION [Abstract] | ' | ||||||||||||||||
| STOCK-BASED COMPENSATION | ' | ||||||||||||||||
| 10. STOCK-BASED COMPENSATION | |||||||||||||||||
| 2010 Stock Incentive Plan | |||||||||||||||||
| The Company's 2010 Stock Incentive Plan, as amended, provides for stock options, restricted shares or restricted share units to be granted to its employees, independent contractors, consultants or directors. During the three and nine months ended September 30, 2014, the Company received approximately $2.4 million and $4.0 million, respectively, from the exercise of stock options. At September 30, 2014, there were 1,746,862 shares remaining available for issuance. | |||||||||||||||||
| The Company recorded employee stock-based compensation expense as follows: | |||||||||||||||||
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | ||||||||||||||||
| (In thousands) | 2014 | 2013 | 2014 | 2013 | |||||||||||||
| Cost of goods sold | $ | 56 | $ | - | $ | 143 | $ | - | |||||||||
| Research and development | 427 | 381 | 1,725 | 1,123 | |||||||||||||
| General and administrative | 3,092 | 1,442 | 7,356 | 4,297 | |||||||||||||
| Total Stock-Based Compensation Expense | $ | 3,575 | $ | 1,823 | $ | 9,224 | $ | 5,420 | |||||||||
| A summary of the activity in the 2010 Equity Incentive Plan, the 2006 Equity Compensation Plan, as amended and the Company's other stock option plans, is as follows: | |||||||||||||||||
| For the Three Months Ended September 30, 2014 | For the Nine Months | ||||||||||||||||
| Ended September 30, 2014 | |||||||||||||||||
| Option | Weighted-average Exercise Price | Option | Weighted-average Exercise Price | ||||||||||||||
| Shares | Shares | ||||||||||||||||
| Beginning balance | 9,952,407 | $ | 7.72 | 8,217,674 | $ | 6.05 | |||||||||||
| Granted | 539,386 | 8.73 | 2,923,104 | 12.35 | |||||||||||||
| Exercised | (578,147 | ) | 4.17 | (1,042,352 | ) | 3.86 | |||||||||||
| Canceled | (626,344 | ) | 9.69 | (811,124 | ) | 10.83 | |||||||||||
| Outstanding at September 30, 2014 | 9,287,302 | 7.86 | 9,287,302 | 7.86 | |||||||||||||
| Employee Stock Purchase Plan | |||||||||||||||||
| The ESPP allows a maximum of 1,000,000 shares of common stock to be purchased in aggregate for all employees. The first purchase of stock will occur in the fourth quarter of 2014 and there was a negligible amount of uninvested employee contributions in the ESPP at September 30, 2014. | |||||||||||||||||
COMMITMENTS_AND_CONTINGENCIES
| COMMITMENTS AND CONTINGENCIES | 9 Months Ended |
| Sep. 30, 2014 | |
| COMMITMENTS AND CONTINGENCIES [Abstract] | ' |
| COMMITMENTS AND CONTINGENCIES | ' |
| 11. COMMITMENTS AND CONTINGENCIES | |
| The Company maintains several contracts with drug labelers and distributors, research organizations, contract manufacturers, clinical organizations and clinical sites, primarily to assist with clinical research and clinical and commercial manufacturing and distribution of PROCYSBI and clinical manufacturing of drug product for the Company's HD and NASH clinical collaborations. The Company's contractual obligations did not change significantly during the nine months ended September 30, 2014 compared to those disclosed as of December 31, 2013. |
SUBSEQUENT_EVENTS
| SUBSEQUENT EVENTS | 9 Months Ended |
| Sep. 30, 2014 | |
| SUBSEQUENT EVENTS [Abstract] | ' |
| SUBSEQUENT EVENTS | ' |
| 12. SUBSEQUENT EVENTS | |
| [OPEN] |
DESCRIPTION_OF_BUSINESS_AND_SU1
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Policies) | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES [Abstract] | ' | ||||||||
| Basis of Presentation | ' | ||||||||
| Basis of Presentation | |||||||||
| The Company's condensed consolidated financial statements include the accounts of the Company's direct and indirect wholly owned subsidiaries, Raptor Pharmaceuticals Inc. and Raptor European Products, LLC, such subsidiaries incorporated in Delaware on August 1, 2007, and February 14, 2012, respectively, and Raptor Pharmaceuticals Europe B.V. (“BV”), Raptor Pharmaceuticals France SAS (“SAS”), Raptor Pharmaceuticals Germany GmbH (“GMBH”) and RPTP European Holdings C.V. (“CV”), domiciled in the Netherlands on December 15, 2009, in France on October 30, 2012, in Germany on October 16, 2013 and in the Cayman Islands on February 16, 2012, respectively. All inter-company accounts have been eliminated. | |||||||||
| Use of Estimates | ' | ||||||||
| Use of Estimates | |||||||||
| The preparation of financial statements in conformity with U.S. GAAP requires management to make certain estimates and assumptions that affect the reported amounts of assets and liabilities, disclosure of contingent assets and liabilities as of the dates of the condensed consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. Actual results could differ from those estimates. | |||||||||
| Functional Currency | ' | ||||||||
| Functional Currency | |||||||||
| The Company's consolidated functional currency is the U.S. dollar. BV, SAS, GMBH, and CV, the Company's Dutch, French, German and Cayman-based subsidiaries, respectively, use the European Euro as their functional currency. At each quarter end, each foreign subsidiary's balance sheet is translated into U.S. dollars based upon the quarter-end exchange rate, while their statements of operations are translated into U.S. dollars based upon an average exchange rate during the period. | |||||||||
| Segment Information | ' | ||||||||
| Segment Information | |||||||||
| The Company has determined that it operates in only one segment, as it only reports profit and loss information on an aggregate basis to its chief operating decision maker. The Company's long-lived assets maintained outside the U.S. are not material. | |||||||||
| Cash and Cash Equivalents | ' | ||||||||
| Cash and Cash Equivalents | |||||||||
| The Company considers all highly liquid investments with original maturities of three months or less, when purchased, to be cash equivalents. The Company maintains cash and cash equivalents, which consist principally of money market funds with high credit quality financial institutions. Such amounts exceed Federal Deposit Insurance Corporation insurance limits. Restricted cash represents compensating balances required by the Company's U.S. and European banks as collateral for credit cards and for access to a value-added tax deferral program. As of September 30, 2014, the Company had $114.7 million in cash and cash equivalents, of which $4.1 million was held by its foreign subsidiaries. | |||||||||
| Fair Value of Financial Instruments | ' | ||||||||
| Fair Value of Financial Instruments | |||||||||
| The carrying amounts of certain of the Company's financial instruments including cash equivalents, restricted cash, accounts payable, accrued liabilities, note payable and capital lease liability approximate fair value due either to length of maturity or interest rates that approximate prevailing market rates. The warrant liability is carried at fair value, which is determined using the Black-Scholes option valuation model at the end of each reporting period. | |||||||||
| Revenue Recognition and Accounts Receivable | ' | ||||||||
| Revenue Recognition and Accounts Receivable | |||||||||
| The Company recognizes revenue in accordance with the Financial Accounting Standards Board ("FASB") Accounting Standards Codification (“ASC”) 605, Revenue Recognition, when the following criteria have been met: persuasive evidence of an arrangement exists; delivery has occurred and risk of loss has passed; the seller's price to the buyer is fixed or determinable and collectability is reasonably assured. The Company determines that persuasive evidence of an arrangement exists based on written contracts that define the terms of the arrangements. Pursuant to the contract terms, the Company determines when title to products and associated risk of loss has passed on to the customer. The Company assesses whether the fee is fixed or determinable based on the payment terms associated with the transaction and whether the sales price is subject to refund or adjustment. The Company assesses collectability based primarily on the customer's payment history and creditworthiness. | |||||||||
| PROCYSBI is currently available for U.S. distribution from the Company's U.S. specialty pharmacy partner, the Accredo Health Group, Inc. ("Accredo") which is currently the Company's only U.S. customer and ships directly to patients. The Company's distributor in the EU is the Almac Group, Ltd. PROCYSBI is not available in U.S. retail pharmacies. Prior authorization of coverage by patients' commercial insurance plans, Raptor's patient assistance program ("PAP") or government payors is a prerequisite to the shipment of PROCYSBI to U.S. patients. Prior to the third quarter of 2014, revenue was recognized in the U.S. once the product had been shipped by the specialty pharmacy to patients because the Company had not yet been able to reasonably estimate the third-party payor mix and resulting rebates based on its lack of sufficient historical data. Beginning July 2014, the Company was able to reasonably estimate and determine sales allowances; therefore the Company began recognizing PROCYSBI revenue at the point of sale to the specialty distributor. As a result of the change, we recognized a net $4.3 million of net revenue in the third quarter of 2014, composed of $6.9 million of net revenue recognized in the third quarter of 2014 that would have been recognized in the fourth quarter of 2014, offset by $2.6 million of net revenue recognized in the third quarter of 2014 that would have been recognized in the second quarter of 2014. Revenue is currently recognized in the EU once confirmed orders from the German pharmacies have been placed and invoiced for payment by the distributor. | |||||||||
| The Company records revenue net of expected discounts, distributor fees, returns and rebates, including those paid to Medicare and Medicaid in the U.S. Allowances are recorded as a reduction of revenue at the time product sales are recognized. Allowances for government rebates and discounts are established based on the actual payor information, which is known in the U.S. at the time of shipment to patients, and the government mandated discount rates applicable to government-funded programs in the U.S. and Germany. The allowances are adjusted to reflect known changes in the factors that may impact such allowances in the quarter the changes are known. | |||||||||
| Trade accounts receivable are recorded net of product sales allowances for prompt-payment discounts and chargebacks. Estimates for chargebacks and prompt-payment discounts are based on contractual terms and the Company's expectations regarding the utilization rates. | |||||||||
| Inventories and Cost of Sales | ' | ||||||||
| Inventories and Cost of Sales | |||||||||
| Inventories are stated at the lower of cost or market price, with cost determined on a first-in, first-out basis. Inventories are reviewed periodically to identify slow-moving inventory based on sales activity, both projected and historical, as well as product shelf-life. Prior to the approval of PROCYSBI by the FDA on April 30, 2013 and in Europe, prior to the approval by the EC on September 6, 2013, the Company recorded the purchase of raw materials and the manufacturing costs relating to PROCYSBI as research and development expense. Subsequent to FDA and EC approval, the Company began capitalizing these costs and manufacturing overhead as commercial inventory. Upon launching PROCYSBI in mid-June 2013 in the U.S. and April 2014 in the EU, the Company began recognizing cost of sales. Cost of sales includes the cost of inventory sold or reserved; manufacturing, manufacturing overhead and supply chain costs; product shipping and handling costs; amortization of licensing approval milestone payments and licensing royalties payable to the University of California, San Diego ("UCSD"). | |||||||||
| The Company capitalizes inventory produced in preparation for product launches when positive results have been obtained for the clinical trials that the Company believes are necessary to support regulatory approval and the Company has determined it is probable that these capitalized costs will provide some future economic benefit in excess of capitalized costs or when the inventory is identical in nature to existing commercial inventory and thus has an "alternative future use". For these inventories, the Company also considers the expected approval date in assessing realizability. To the extent that inventory is expected to expire prior to being sold, the Company will write down the value of inventory. If actual results differ from those estimates, additional inventory write-offs may be required. | |||||||||
| Impairment of Goodwill and Intangible Assets | ' | ||||||||
| Impairment of Goodwill and Intangible Assets | |||||||||
| Goodwill represents the excess of the purchase price over the fair value of tangible and identified intangible net assets of businesses acquired. Goodwill is not amortized, but is evaluated for impairment on an annual basis or more often when impairment indicators are present. The Company has one reporting unit. Therefore, the Company's consolidated net assets, including existing goodwill and other intangible assets, are considered to be the carrying value of the reporting unit. If the carrying value of the reporting unit is in excess of its fair value, an impairment may exist, and the Company must perform the second step of the analysis, in which the implied fair value of the goodwill is compared to its carrying value to determine the impairment charge, if any. If the estimated fair value of the reporting unit exceeds the carrying value of the reporting unit, goodwill is not impaired and no further analysis is required. | |||||||||
| The Company makes judgments about the recoverability of purchased intangible assets with finite lives whenever events or changes in circumstances indicate that impairment may exist. Recoverability of purchased intangible assets with finite lives is measured by comparing the carrying amount of the asset to the future undiscounted cash flows the asset is expected to generate. Impairment, if any, is measured as the amount by which the carrying value exceeds the fair value of the impaired asset. | |||||||||
| Fixed Assets | ' | ||||||||
| Fixed Assets | |||||||||
| Fixed assets, which mainly consist of leasehold improvements, office furniture, manufacturing and lab equipment and computer hardware and software, are stated at cost. Depreciation is computed using the straight-line method over the related estimated useful lives, except for leasehold improvements and capital lease equipment, which are depreciated over the shorter of the useful life of the asset or the lease term. Significant additions and improvements that have useful lives estimated at greater than one year are capitalized, while repairs and maintenance are charged to expense as incurred. | |||||||||
| Common Stock Warrant Liabilities | ' | ||||||||
| Common Stock Warrant Liabilities | |||||||||
| The Company issued warrants that contain conditional obligations that may require the Company to transfer cash to settle the warrants upon the occurrence of certain fundamental transactions. Therefore, the Company has classified the warrants as liabilities. The Company re-measures the liability at the end of every reporting period with the change in value reported in the Company's condensed consolidated statements of operations. At the exercise date, the fair values of these warrants are re-measured and reclassified to equity. | |||||||||
| Note Payable | ' | ||||||||
| Note Payable | |||||||||
| Note payable consists of a loan agreement with HealthCare Royalty Partners II, L.P. ("HC Royalty"), as lender, which was amended effective July 1, 2014. The amendment qualified as a modification of debt in accordance with ASC 470-50, Debt – Modifications and Extinguishments, as we determined it did not result in substantially different terms. The amended loan requires quarterly interest payments at an annual fixed interest rate of 8.0% of outstanding principal and includes a synthetic royalty component based on net product sales, including PROCYSBI, in a calendar year. Prior to July 1, 2014, the original loan bore interest at an annual fixed rate of 10.75% of outstanding principal and included a synthetic royalty component based on net product sales, including PROCYSBI, in a calendar year. | |||||||||
| Note payable is carried at its unpaid principal balance. The fixed and royalty interest under both agreements were recognized as interest expense as incurred. | |||||||||
| Convertible Notes | ' | ||||||||
| Convertible Notes | |||||||||
| Convertible notes include unsecured convertible senior notes and are carried at their unpaid principal balance. Interest on the notes is payable quarterly and the notes mature on August 1, 2019. If converted by a holder, upon conversion, the holder of the notes would receive shares of the Company’s common stock. | |||||||||
| Debt Issuance Costs | ' | ||||||||
| Debt Issuance Costs | |||||||||
| Debt issuance costs are expenses associated with the issuance of the loan agreements with HC Royalty and the convertible notes. Debt issuance costs which were capitalized are being amortized over the life of the respective debt to interest expense using the interest method. Debt issuance costs are a component of other assets on our condensed consolidated balance sheets. | |||||||||
| Research and Development Costs | ' | ||||||||
| Research and Development Costs | |||||||||
| Research and development costs are charged to expense as incurred. Research and development expenses primarily include salaries and benefits for medical, clinical, regulatory, quality, pharmacovigilance and research personnel, preclinical studies, clinical trials and certain commercial drug manufacturing expenses prior to obtaining marketing approval. | |||||||||
| Net Loss per Share | ' | ||||||||
| Net Loss per Share | |||||||||
| Net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include: | |||||||||
| Nine Months Ended September 30, | |||||||||
| 2014 | 2013 | ||||||||
| Warrants to purchase common stock | 334,764 | 1,145,170 | |||||||
| Options to purchase common stock | 9,287,302 | 8,229,927 | |||||||
| Convertible debt | 3,428,571 | - | |||||||
| Total Potentially Dilutive Securities | 13,050,637 | 9,375,097 | |||||||
| Comprehensive Loss | ' | ||||||||
| Comprehensive Loss | |||||||||
| The components of comprehensive loss include net loss and foreign currency translation adjustments. | |||||||||
| Stock-Based Compensation | ' | ||||||||
| Stock-Based Compensation | |||||||||
| Stock Option Plan | |||||||||
| Compensation costs related to the Company's stock option plan are measured at the grant date based on the fair value of the equity instruments awarded and are recognized over the period during which an employee is required to provide service in exchange for the award, or the requisite service period, which is usually the vesting period. The compensation expense for stock-based compensation awards is reduced by an estimate for forfeitures. | |||||||||
| The Company recognizes expense associated with stock options issued to third parties, including consultants, based upon the fair value of such awards on the date the options vest. | |||||||||
| Employee Stock Purchase Plan | |||||||||
| In July 2014, the Company’s shareholders approved the Raptor Pharmaceutical Corp. 2013 Employee Stock Purchase Plan (“ESPP”). Up to 1,000,000 shares may be issued pursuant to the ESPP. The purpose of the ESPP is to give the Company’s employees an opportunity to acquire an equity interest in the Company through the purchase of shares of common stock at a discount. The ESPP allows eligible employees to purchase common stock at 85% of its fair value, subject to certain limits. Fair value as defined under the ESPP is the lesser of the closing market price of the common stock on the first day of the offering period or the last day of the offering period. | |||||||||
| Income Taxes | ' | ||||||||
| Income Taxes | |||||||||
| Income taxes are recorded under the liability method, under which deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Valuation allowances are established when necessary to reduce deferred tax assets to the amount expected to be realized. Based on the weight of available evidence, including cumulative losses since inception and expected future losses, the Company has determined that it is more likely than not that the deferred tax asset amount will not be realized and therefore a full valuation allowance has been provided on the Company's net deferred tax assets. | |||||||||
| The Company identifies uncertain tax positions and discloses any potential tax liability on its financial statements. The Company recognizes interest and/or penalties related to income tax matters as a component of income tax expense. As of September 30 2014, there were no accrued uncertain tax positions or interest and penalties related to uncertain tax positions. | |||||||||
| The Company files U.S. Federal, California, various other state and other income tax returns and various foreign country income tax returns. The Company is currently not subject to any income tax examinations. Due to the Company's net operating losses ("NOLs"), generally all tax years remain open. | |||||||||
| Reclassifications | ' | ||||||||
| Reclassifications | |||||||||
| Certain amounts previously reported under specific financial statement captions have been reclassified to be consistent with the current year presentation. | |||||||||
| Recent Accounting Pronouncements | ' | ||||||||
| Recent Accounting Pronouncements | |||||||||
| In May 2014, the FASB issued Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers, which outlines a single comprehensive model for entities to use in accounting for revenue arising from contracts with customers and supersedes most current revenue recognition guidance, including industry-specific guidance. The core principle of the revenue model is that “an entity recognizes revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services.” In applying the revenue model to contracts within its scope, the Company will: identify the contract(s) with a customer, identify the performance obligations in the contract, determine the transaction price, allocate the transaction price to the performance obligations in the contract, and recognize revenue when (or as) the entity satisfies a performance obligation. This ASU is effective for interim and annual periods beginning after December 15, 2016 and early adoption is not permitted. The Company does not believe the adoption of this ASU will have a material impact on its consolidated financial statements. | |||||||||
| In June 2014, the FASB issued ASU 2014-12, Accounting for Share-Based Payments When the Terms of an Award Provide That a Performance Target Could Be Achieved after the Requisite Service Period. The ASU requires that a performance target that affects vesting, and that could be achieved after the requisite service period, be treated as a performance condition. A reporting entity should apply existing guidance in Topic 718 as it relates to awards with performance conditions that affect vesting to account for such awards. This ASU is effective for interim and annual periods beginning after December 15, 2015 and early adoption is permitted. The Company does not anticipate the adoption of this ASU will have a material impact on its consolidated financial statements. | |||||||||
DESCRIPTION_OF_BUSINESS_AND_SU2
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Tables) | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES [Abstract] | ' | ||||||||
| Potentially dilutive securities | ' | ||||||||
| Net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding during the period. Diluted net loss per share is calculated by dividing net loss by the weighted-average shares of common stock outstanding and potential shares of common stock during the period. For all periods presented, potentially dilutive securities are excluded from the computation of fully diluted net loss per share as their effect is anti-dilutive. Potentially dilutive securities include: | |||||||||
| Nine Months Ended September 30, | |||||||||
| 2014 | 2013 | ||||||||
| Warrants to purchase common stock | 334,764 | 1,145,170 | |||||||
| Options to purchase common stock | 9,287,302 | 8,229,927 | |||||||
| Convertible debt | 3,428,571 | - | |||||||
| Total Potentially Dilutive Securities | 13,050,637 | 9,375,097 | |||||||
FAIR_VALUE_MEASUREMENT_Tables
| FAIR VALUE MEASUREMENT (Tables) | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| FAIR VALUE MEASUREMENT [Abstract] | ' | ||||||||||||||||
| Assets and liabilities measured at fair value on a recurring basis | ' | ||||||||||||||||
| Determining which category an asset or liability falls within the hierarchy requires significant judgment. The Company evaluates its hierarchy disclosures each quarter. Assets and liabilities measured at fair value on a recurring basis at September 30, 2014 and December 31, 2013 are summarized as follows: | |||||||||||||||||
| (In thousands) | |||||||||||||||||
| 30-Sep-14 | Level 1 | Level 2 | Level 3 | Total | |||||||||||||
| Assets | |||||||||||||||||
| Cash equivalents (1) | $ | 109,610 | $ | - | $ | - | $ | 109,610 | |||||||||
| Total | $ | 109,610 | $ | - | $ | - | $ | 109,610 | |||||||||
| Liabilities | |||||||||||||||||
| Common stock warrants | $ | - | $ | - | $ | 654 | $ | 654 | |||||||||
| Total | $ | - | $ | - | $ | 654 | $ | 654 | |||||||||
| 31-Dec-13 | Level 1 | Level 2 | Level 3 | Total | |||||||||||||
| Assets | |||||||||||||||||
| Cash equivalents (1) | $ | 70,627 | $ | - | $ | - | $ | 70,627 | |||||||||
| Total | $ | 70,627 | $ | - | $ | - | $ | 70,627 | |||||||||
| Liabilities | |||||||||||||||||
| Common stock warrants | $ | - | $ | - | $ | 7,066 | $ | 7,066 | |||||||||
| Total | $ | - | $ | - | $ | 7,066 | $ | 7,066 | |||||||||
| (1) Cash equivalents represent the fair value of the Company's investments in money market funds at September 30, 2014 and December 31, 2013. | |||||||||||||||||
| Fair value measurement of liabilities using significant unobservable inputs | ' | ||||||||||||||||
| The following table presents a reconciliation of the Company's recurring fair value measurements categorized within Level 3 of the fair value hierarchy (liability-classified common stock warrants): | |||||||||||||||||
| Changes in Level 3 Liabilities Measured at Fair Value on a Recurring Basis – Common Stock Warrants | |||||||||||||||||
| (In thousands) | Nine Months Ended | ||||||||||||||||
| 30-Sep-14 | |||||||||||||||||
| Fair value as of December 31, 2013 | $ | 7,066 | |||||||||||||||
| Change in fair value recognized in earnings | 1,091 | ||||||||||||||||
| Exercises | (7,503 | ) | |||||||||||||||
| Fair Value as of September 30, 2014 | $ | 654 | |||||||||||||||
INTANGIBLE_ASSETS_AND_GOODWILL1
| INTANGIBLE ASSETS AND GOODWILL (Tables) | 9 Months Ended | ||||||||||||
| Sep. 30, 2014 | |||||||||||||
| INTANGIBLE ASSETS AND GOODWILL [Abstract] | ' | ||||||||||||
| Summary of intangibles acquired | ' | ||||||||||||
| A summary of intangibles acquired is as follows: | |||||||||||||
| Useful Life | September 30, | December 31, | |||||||||||
| (In thousands) | (Years) | 2014 | 2013 | ||||||||||
| Intangible asset (IP license for RP103) related to the Encode merger | 20 | $ | 2,620 | $ | 2,620 | ||||||||
| Intangible assets (UCSD license - FDA and EC approval milestones) | 20 | 1,250 | 1,250 | ||||||||||
| Other intangible assets | 16 | 240 | 240 | ||||||||||
| Total intangible assets | 4,110 | 4,110 | |||||||||||
| Less accumulated amortization | (1,076 | ) | (897 | ) | |||||||||
| Intangible Assets, Net | $ | 3,034 | $ | 3,213 | |||||||||
| Actual and estimated amortization expense for intangible assets | ' | ||||||||||||
| Amortization expense for intangible assets for each of the next five years is expected to be as follows: | |||||||||||||
| (In thousands) | Amortization Expense | ||||||||||||
| 2014 (remaining 3 months) | $ | 60 | |||||||||||
| 2015 | 238 | ||||||||||||
| 2016 | 238 | ||||||||||||
| 2017 | 238 | ||||||||||||
| 2018 | 238 | ||||||||||||
INVENTORIES_Tables
| INVENTORIES (Tables) | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| INVENTORIES [Abstract] | ' | ||||||||
| Inventory | ' | ||||||||
| Inventories are summarized as follows: | |||||||||
| September 30, | December 31, | ||||||||
| (In thousands) | 2014 | 2013 | |||||||
| Raw materials | $ | 8,540 | $ | 2,469 | |||||
| Work-in-process | 444 | - | |||||||
| Early access program | 1,801 | - | |||||||
| Finished goods | 1,962 | 531 | |||||||
| Total Inventories | $ | 12,747 | $ | 3,000 | |||||
FIXED_ASSETS_Tables
| FIXED ASSETS (Tables) | 9 Months Ended | ||||||||||||
| Sep. 30, 2014 | |||||||||||||
| FIXED ASSETS [Abstract] | ' | ||||||||||||
| Fixed Assets | ' | ||||||||||||
| Fixed assets consisted of: | |||||||||||||
| September 30, | December 31, | Estimated | |||||||||||
| (In thousands) | 2014 | 2013 | useful lives | ||||||||||
| Laboratory equipment | $ | 1,484 | $ | 1,132 | 5 years | ||||||||
| Assets under construction | 2,388 | 102 | - | ||||||||||
| Computer hardware and software | 780 | 646 | 3 years | ||||||||||
| Office furniture | 1,835 | 605 | 7 years | ||||||||||
| Leasehold improvements | 260 | - | Lease term | ||||||||||
| Total at cost | 6,747 | 2,485 | |||||||||||
| Less: accumulated depreciation | (1,145 | ) | (675 | ) | |||||||||
| Total Fixed Assets, Net | $ | 5,602 | $ | 1,810 | |||||||||
ACCRUED_LIABILITIES_Tables
| ACCRUED LIABILITIES (Tables) | 9 Months Ended | ||||||||
| Sep. 30, 2014 | |||||||||
| ACCRUED LIABILITIES [Abstract] | ' | ||||||||
| Accrued liabilities | ' | ||||||||
| Accrued liabilities consisted of: | |||||||||
| September 30, | December 31, | ||||||||
| (In thousands) | 2014 | 2013 | |||||||
| Personnel-related costs | $ | 4,770 | $ | 4,443 | |||||
| Rebates and other sales deductions | 3,545 | 2,325 | |||||||
| Clinical trials and research and development costs | 2,153 | 1,661 | |||||||
| Royalty-based interest payable | 1,437 | 1,255 | |||||||
| License royalty payable | 925 | 564 | |||||||
| Manufacturing costs | 2,611 | 294 | |||||||
| Other | 2,616 | 2,586 | |||||||
| Total Accrued Liabilities | $ | 18,057 | $ | 13,128 | |||||
CAPITAL_STRUCTURE_Tables
| CAPITAL STRUCTURE (Tables) | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| CAPITAL STRUCTURE [Abstract] | ' | ||||||||||||||||
| Number of outstanding common stock warrants | ' | ||||||||||||||||
| The number of common stock warrants outstanding as of September 30, 2014 were as follows: | |||||||||||||||||
| Number of Shares Exercisable | |||||||||||||||||
| Exercise | Expiration | ||||||||||||||||
| Price | Date | ||||||||||||||||
| Issued in connection with Encode merger | 233,309 | $ | 2.87 | 12/13/15 | |||||||||||||
| TorreyPines warrants assumed in 2009 Merger | 3,502 | 157.08 | 9/26/15 | ||||||||||||||
| Issued to registered direct investors in Dec. 2009 | 1 | 2.45 | 12/22/14 | ||||||||||||||
| Issued to placement agent in Aug. 2010 | 97,952 | 3.08 | 8/12/15 | ||||||||||||||
| Total Warrants Outstanding | 334,764 | $ | 4.54 | -1 | |||||||||||||
| (1) Weighted average exercise price | |||||||||||||||||
| Assumptions for fair value of the warrants | ' | ||||||||||||||||
| A Black-Scholes option-pricing model was used to obtain the fair value of the warrant liabilities using the following assumptions at September 30, 2014 and December 31, 2013: | |||||||||||||||||
| December 2009 Equity Financing Series A | August 2010 Private Placement Investors and Placement Agent | ||||||||||||||||
| September 30, | December 31, | September 30, | December 31, | ||||||||||||||
| 2014 | 2013 | 2014 | 2013 | ||||||||||||||
| Fair value (in thousands) | $ | - | $ | 133 | $ | 654 | $ | 6,933 | |||||||||
| Black-Scholes inputs: | |||||||||||||||||
| Stock price | $ | 9.59 | $ | 13.02 | $ | 9.59 | $ | 13.02 | |||||||||
| Exercise price | $ | 2.45 | $ | 2.45 | $ | 3.08 | $ | 3.08 | |||||||||
| Risk free interest rate | 0.01 | % | 0.13 | % | 0.08 | % | 0.33 | % | |||||||||
| Volatility | 95 | % | 95 | % | 95 | % | 95 | % | |||||||||
| Expected term (years) | 0.25 | 1 | 0.75 | 1.75 | |||||||||||||
| Dividend | - | - | - | - | |||||||||||||
STOCKBASED_COMPENSATION_Tables
| STOCK-BASED COMPENSATION (Tables) | 9 Months Ended | ||||||||||||||||
| Sep. 30, 2014 | |||||||||||||||||
| STOCK-BASED COMPENSATION [Abstract] | ' | ||||||||||||||||
| Employee and consultant stock-based compensation expense | ' | ||||||||||||||||
| The Company recorded employee stock-based compensation expense as follows: | |||||||||||||||||
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | ||||||||||||||||
| (In thousands) | 2014 | 2013 | 2014 | 2013 | |||||||||||||
| Cost of goods sold | $ | 56 | $ | - | $ | 143 | $ | - | |||||||||
| Research and development | 427 | 381 | 1,725 | 1,123 | |||||||||||||
| General and administrative | 3,092 | 1,442 | 7,356 | 4,297 | |||||||||||||
| Total Stock-Based Compensation Expense | $ | 3,575 | $ | 1,823 | $ | 9,224 | $ | 5,420 | |||||||||
| Summary of the activity in stock option plan | ' | ||||||||||||||||
| A summary of the activity in the 2010 Equity Incentive Plan, the 2006 Equity Compensation Plan, as amended and the Company's other stock option plans, is as follows: | |||||||||||||||||
| For the Three Months Ended September 30, 2014 | For the Nine Months | ||||||||||||||||
| Ended September 30, 2014 | |||||||||||||||||
| Option | Weighted-average Exercise Price | Option | Weighted-average Exercise Price | ||||||||||||||
| Shares | Shares | ||||||||||||||||
| Beginning balance | 9,952,407 | $ | 7.72 | 8,217,674 | $ | 6.05 | |||||||||||
| Granted | 539,386 | 8.73 | 2,923,104 | 12.35 | |||||||||||||
| Exercised | (578,147 | ) | 4.17 | (1,042,352 | ) | 3.86 | |||||||||||
| Canceled | (626,344 | ) | 9.69 | (811,124 | ) | 10.83 | |||||||||||
| Outstanding at September 30, 2014 | 9,287,302 | 7.86 | 9,287,302 | 7.86 | |||||||||||||
DESCRIPTION_OF_BUSINESS_AND_SU3
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES (Details) (USD $) | 3 Months Ended | 9 Months Ended | 9 Months Ended | 1 Months Ended | ||||||||
| Sep. 30, 2014 | Sep. 30, 2014 | Sep. 30, 2013 | Dec. 31, 2013 | Dec. 31, 2012 | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Jul. 31, 2014 | |
| ReportingUnit | Warrants to Purchase Common Stock [Member] | Warrants to Purchase Common Stock [Member] | Options to Purchase Common Stock [Member] | Options to Purchase Common Stock [Member] | Convertible Debt [Member] | Convertible Debt [Member] | Loan Agreement with HC Royalty [Member] | |||||
| Segment | ||||||||||||
| DESCRIPTION OF BUSINESS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Minimum age of children for management of nephropathic cystinosis | ' | '6 years | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Period of orphan drug exclusivity for PROCYSBI in U.S. | ' | '7 years | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Period of orphan drug exclusivity for PROCYSBI in EU | ' | '10 years | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Segment Information [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Number of segment | ' | 1 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Cash and Cash Equivalents [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Maximum maturity period for investments considered as highly liquid investments | ' | '3 months | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Cash and cash equivalents | $114,678,000 | $114,678,000 | $88,334,000 | $83,052,000 | $36,313,000 | ' | ' | ' | ' | ' | ' | ' |
| Cash and cash equivalents held by foreign subsidiaries | 4,100,000 | 4,100,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Revenue Recognition and Accounts Receivable [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Revenue recognized, net of offset | 4,300,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Recognized revenue attributable to sales in the quarter | 6,900,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Revenue recognized, net of offset | 2,600,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Impairment of Goodwill and Intangible Assets [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Number of reporting unit | ' | 1 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Fixed Assets [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Useful life of significant additions and improvements in fixed assets for capitalization, minimum | ' | '1 year | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Note Payable [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Annual fixed interest rate (in hundredths) | 10.75% | 10.75% | ' | ' | ' | ' | ' | ' | ' | ' | ' | 8.00% |
| Convertible Notes [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Maturity date | ' | 1-Aug-19 | ' | ' | ' | ' | ' | ' | ' | ' | ' | 30-Jun-15 |
| Employee Stock Purchase Plan [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Employee stock purchase plan maximum shares issued | 1,000,000 | 1,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Common stock purchase price percentage on fair value (in hundredths) | ' | 85.00% | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Income taxes [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Accrued interest or penalties related to uncertain tax positions | $0 | $0 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Total potentially dilutive securities (in shares) | ' | 13,050,637 | 9,375,097 | ' | ' | 334,764 | 1,145,170 | 9,287,302 | 8,229,927 | 3,428,571 | 0 | ' |
FAIR_VALUE_MEASUREMENT_Details
| FAIR VALUE MEASUREMENT (Details) (USD $) | 9 Months Ended | ||||||||||||||||
| In Thousands, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | ||||||||
| Common Stock Warrants [Member] | Recurring [Member] | Recurring [Member] | Level 1 [Member] | Level 1 [Member] | Level 2 [Member] | Level 2 [Member] | Level 3 [Member] | Level 3 [Member] | |||||||||
| Recurring [Member] | Recurring [Member] | Recurring [Member] | Recurring [Member] | Recurring [Member] | Recurring [Member] | ||||||||||||
| Assets [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Cash equivalents | ' | $109,610 | [1] | $70,627 | [1] | $109,610 | [1] | $70,627 | [1] | $0 | [1] | $0 | [1] | $0 | [1] | $0 | [1] |
| Total | ' | 109,610 | 70,627 | 109,610 | 70,627 | 0 | 0 | 0 | 0 | ||||||||
| Liabilities [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Common stock warrants | ' | 654 | 7,066 | 0 | 0 | 0 | 0 | 654 | 7,066 | ||||||||
| Total | ' | 654 | 7,066 | 0 | 0 | 0 | 0 | 654 | 7,066 | ||||||||
| Warrant liabilities activity [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Fair value at beginning of period | 7,066 | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Change in fair value recognized in earnings | 1,091 | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Exercises | -7,503 | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| Fair Value at end of period | $654 | ' | ' | ' | ' | ' | ' | ' | ' | ||||||||
| [1] | Cash equivalents represent the fair value of the Company's investments in money market funds at September 30, 2014 and December 31, 2013. | ||||||||||||||||
INTANGIBLE_ASSETS_AND_GOODWILL2
| INTANGIBLE ASSETS AND GOODWILL (Details) (USD $) | 9 Months Ended | 12 Months Ended | 3 Months Ended | 9 Months Ended | 9 Months Ended | 3 Months Ended | 9 Months Ended | 9 Months Ended | ||||||||||
| Sep. 30, 2014 | Sep. 30, 2013 | Dec. 31, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | Dec. 14, 2007 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2013 | Jun. 30, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | |
| Research and Development Expense [Member] | Research and Development Expense [Member] | Research and Development Expense [Member] | Research and Development Expense [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | IP license for PROCYSBI/RP103 [Member] | IP license for PROCYSBI/RP103 [Member] | UCSD license FDA and EC approval milestones [Member] | UCSD license FDA and EC approval milestones [Member] | UCSD license FDA and EC approval milestones [Member] | UCSD license FDA and EC approval milestones [Member] | Other Intangible Assets [Member] | Other Intangible Assets [Member] | ||||
| Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | Encode Pharmaceuticals, Inc. [Member] | |||||||||||
| Acquired Finite-Lived Intangible Assets [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Minimum age of children for management of nephropathic cystinosis | '6 years | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Milestone payment based on drug approvals | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | $500,000 | $800,000 | ' | ' | ' | ' |
| Intangible asset impairment recognized | 0 | ' | 0 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Amortization charged to research and development expense | 179,000 | 134,000 | ' | 37,000 | 47,000 | 110,000 | 134,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Summary of intangible assets [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Useful Life | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | '20 years | ' | ' | ' | '20 years | ' | '16 years | ' |
| Total intangible assets | ' | ' | ' | ' | ' | ' | ' | 4,110,000 | 4,110,000 | 2,600,000 | 2,620,000 | 2,620,000 | ' | ' | 1,250,000 | 1,250,000 | 240,000 | 240,000 |
| Less accumulated amortization | ' | ' | ' | ' | ' | ' | ' | -1,076,000 | -897,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Intangible assets, Net | ' | ' | ' | ' | ' | ' | ' | 3,034,000 | 3,213,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Actual and estimated amortization expense for intangible assets [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| 2014 (remaining 3 months) | 60,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| 2015 | 238,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| 2016 | 238,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| 2017 | 238,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| 2018 | $238,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
INVENTORIES_Details
| INVENTORIES (Details) (USD $) | Sep. 30, 2014 | Dec. 31, 2013 |
| In Thousands, unless otherwise specified | ||
| INVENTORIES [Abstract] | ' | ' |
| Raw materials | $8,540 | $2,469 |
| Work-in-process | 444 | 0 |
| Early access program | 1,801 | 0 |
| Finished goods | 1,962 | 531 |
| Total Inventories | $12,747 | $3,000 |
FIXED_ASSETS_Details
| FIXED ASSETS (Details) (USD $) | 9 Months Ended | ||
| In Thousands, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 | Dec. 31, 2013 |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | $6,747 | ' | $2,485 |
| Less: accumulated depreciation | -1,145 | ' | -675 |
| Total fixed assets, net | 5,602 | ' | 1,810 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Depreciation expense | 411 | 152 | ' |
| Laboratory Equipment [Member] | ' | ' | ' |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | 1,484 | ' | 1,132 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Estimated useful lives | '5 years | ' | ' |
| Asset under Construction [Member] | ' | ' | ' |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | 2,388 | ' | 102 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Estimated useful lives | '0 years | ' | ' |
| Computer Hardware and Software [Member] | ' | ' | ' |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | 780 | ' | 646 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Estimated useful lives | '3 years | ' | ' |
| Office Furniture [Member] | ' | ' | ' |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | 1,835 | ' | 605 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Estimated useful lives | '7 years | ' | ' |
| Leasehold Improvements [Member] | ' | ' | ' |
| Fixed assets [Abstract] | ' | ' | ' |
| Total at cost | $260 | ' | $0 |
| Estimated useful lives [Abstract] | ' | ' | ' |
| Estimated useful lives | 'Lease term | ' | ' |
NOTE_PAYABLE_Details
| NOTE PAYABLE (Details) (USD $) | 3 Months Ended | 9 Months Ended | 1 Months Ended | 3 Months Ended | 9 Months Ended | 1 Months Ended | 6 Months Ended | 0 Months Ended | 6 Months Ended | |||||
| Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Jul. 31, 2014 | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 | Dec. 31, 2013 | Dec. 31, 2012 | Jun. 30, 2014 | 21-May-13 | Jun. 30, 2014 | |
| Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | Loan Agreement with HC Royalty [Member] | |||||
| Tranche | Tranche | First Tranche [Member] | First Tranche [Member] | Second Tranche [Member] | Second Tranche [Member] | |||||||||
| Debt Instrument [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Total amount of loan | ' | ' | ' | ' | $10,000,000 | $50,000,000 | ' | $50,000,000 | ' | ' | ' | ' | ' | ' |
| Number of tranches in loan | ' | ' | ' | ' | ' | 2 | ' | 2 | ' | ' | ' | ' | ' | ' |
| Amount of loan per tranche | ' | ' | ' | ' | ' | 25,000,000 | ' | 25,000,000 | ' | ' | ' | ' | ' | ' |
| Draw down on loan | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 23,400,000 | ' | 23,700,000 | ' |
| Annual fixed interest rate (in hundredths) | 10.75% | ' | 10.75% | ' | 8.00% | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Original interest rate (in hundredths) | ' | ' | ' | ' | 10.75% | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Revised annual interest rate (in hundredths) | ' | ' | ' | ' | 8.00% | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Amount of first net revenues for calendar year | ' | ' | ' | ' | 50,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Excess revenue (in hundredths) | ' | ' | ' | ' | 2.00% | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Royalty rate of first threshold of PROCYSBI and future approved product net revenues for calendar year (in hundredths) | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 6.25% | ' | 6.00% |
| Amount of first threshold of PROCYSBI and future approved product net revenues for calendar year | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 25,000,000 | ' | 25,000,000 |
| Royalty rate of amount between first and second threshold of PROCYSBI and future approved product net revenues for calendar year (in hundredths) | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 3.00% | ' | 3.00% |
| Amount of second threshold of PROCYSBI and future approved product net revenues for calendar year | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 50,000,000 | ' | 50,000,000 |
| Royalty rate for amount in excess of second threshold of PROCYSBI and future approved product net revenues for calendar year (in hundredths) | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | 1.00% | ' | 1.00% |
| Interest Expense | 4,380,000 | 2,287,000 | 10,856,000 | 4,088,000 | ' | 2,800,000 | 2,100,000 | 9,000,000 | 3,800,000 | ' | ' | ' | ' | ' |
| First payment amount | ' | ' | ' | ' | 3,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Maturity Date | ' | ' | 1-Aug-19 | ' | 30-Jun-15 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Total payment required to terminate loan obligation immediately | ' | ' | ' | ' | 120,000,000 | ' | ' | ' | ' | ' | ' | ' | ' | ' |
| Unamortized debt issuance costs | ' | ' | ' | ' | ' | 2,400,000 | ' | 2,400,000 | ' | 2,800,000 | ' | ' | ' | ' |
| Amortization of debt expense | ' | ' | ' | ' | ' | ' | ' | $900,000 | $300,000 | ' | ' | ' | ' | ' |
CONVERTIBLE_NOTES_Details
| CONVERTIBLE NOTES (Details) (USD $) | 1 Months Ended | 3 Months Ended | 9 Months Ended |
| Jul. 31, 2014 | Sep. 30, 2014 | Sep. 30, 2014 | |
| Debt Instrument [Line Items] | ' | ' | ' |
| Annual fixed interest rate (in hundredths) | ' | 10.75% | 10.75% |
| Maturity Date | ' | ' | 1-Aug-19 |
| 8% convertible senior notes due 2019 [Member] | ' | ' | ' |
| Debt Instrument [Line Items] | ' | ' | ' |
| Aggregate principal amount | $60,000,000 | ' | ' |
| Annual fixed interest rate (in hundredths) | 8.00% | ' | ' |
| Maturity Date | 1-Aug-19 | ' | ' |
| Convertible senior notes conversion rate | 57.14 | ' | ' |
| Debt instrument convertible base principal amount of conversion | 1,000 | ' | ' |
| The price per share of the conversion feature embedded in the debt instrument (in dollars per share) | $17.50 | ' | ' |
| Percentage of applicable conversion price (in hundredths) | 175.00% | ' | ' |
| Consecutive trading days | '30 days | ' | ' |
| Unamortized debt issuance costs | ' | 2,900,000 | 2,900,000 |
| Amortization expense | ' | 100,000 | ' |
| Interest expense excluding amortization of debt issuance costs | ' | ' | $900,000 |
ACCRUED_LIABILITIES_Details
| ACCRUED LIABILITIES (Details) (USD $) | Sep. 30, 2014 | Dec. 31, 2013 |
| In Thousands, unless otherwise specified | ||
| Accrued liabilities [Abstract] | ' | ' |
| Personnel-related costs | $4,770 | $4,443 |
| Rebates and other sales deductions | 4,273 | 2,325 |
| Manufacturing costs | 2,611 | 294 |
| Royalty-based interest payable | 2,164 | 1,255 |
| Clinical trials and research and development costs | 2,153 | 1,661 |
| License royalty payable | 925 | 564 |
| Other | 2,664 | 2,586 |
| Total Accrued Liabilities | $19,560 | $13,128 |
CAPITAL_STRUCTURE_COMMON_STOCK
| CAPITAL STRUCTURE, COMMON STOCK (Details) (USD $) | 9 Months Ended | 9 Months Ended | |||||
| In Millions, except Share data, unless otherwise specified | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Sep. 30, 2014 | Jul. 03, 2013 | Apr. 30, 2012 | |
| Series A Participating Preferred Stock [Member] | Shares Issued under ATM Sales Agreement [Member] | Shares Issued under ATM Sales Agreement [Member] | Shares Issued under ATM Sales Agreement [Member] | ||||
| Class of Stock [Line Items] | ' | ' | ' | ' | ' | ' | |
| Preferred stock, shares authorized (in shares) | 15,000,000 | 15,000,000 | ' | ' | ' | ' | |
| Preferred stock, par value (in dollars per share) | $0.00 | $0.00 | ' | ' | ' | ' | |
| Common stock, shares authorized (in shares) | 150,000,000 | 150,000,000 | ' | ' | ' | ' | |
| Common stock, par value (in dollars per share) | $0.00 | $0.00 | ' | ' | ' | ' | |
| Common stock, voting rights | 'One vote for each share held | ' | ' | ' | ' | ' | |
| Common stock issued (in shares) | 63,268,534 | 61,614,576 | ' | 0 | ' | ' | |
| Common stock outstanding (in shares) | 63,268,534 | 61,614,576 | ' | ' | ' | ' | |
| Maximum amount of common stock under ATM sales agreement | ' | ' | ' | ' | $100 | $40 | |
| Sales commission percentage for sales made under ATM (in hundredths) | ' | ' | ' | 3.00% | ' | ' | |
| Cumulative net proceeds from sale of shares | ' | ' | ' | $53.80 | ' | ' | |
| Class of Warrant or Right [Line Items] | ' | ' | ' | ' | ' | ' | |
| Rights per share of common stock (in shares) | ' | ' | 0.001 | ' | ' | ' | |
| Price per share (in dollars per share) | $4.54 | [1] | ' | $15 | ' | ' | ' |
| Maximum percentage of common stock held for rights to become non-exercisable (in hundredths) | ' | ' | 15.00% | ' | ' | ' | |
| Common share voting power of right (in shares) | ' | ' | 0.007352 | ' | ' | ' | |
| Common share payment of rights (in shares) | ' | ' | 0.007352 | ' | ' | ' | |
| [1] | Weighted-average exercise price | ||||||
CAPITAL_STRUCTURE_COMMON_STOCK1
| CAPITAL STRUCTURE, COMMON STOCK WARRANTS (Details) (USD $) | 9 Months Ended | 12 Months Ended | 9 Months Ended | 12 Months Ended | |||||||
| In Thousands, except Share data, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2014 | Sep. 30, 2014 | Sep. 30, 2014 | Sep. 30, 2014 | Dec. 31, 2013 | Sep. 30, 2014 | Dec. 31, 2013 | |
| Issued in Connection with Encode Merger [Member] | TorreyPines warrants assumed in 2009 Merger [Member] | Issued to Registered Direct Investors in Dec. 2009 [Member] | Issued to Placement Agent in Aug. 2010 [Member] | December 2009 Equity Financing, Series A [Member] | December 2009 Equity Financing, Series A [Member] | August 2010 Private Placement, Investors and Placement Agent [Member] | August 2010 Private Placement, Investors and Placement Agent [Member] | ||||
| Class of Warrant or Right [Line Items] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | |
| Proceeds from the exercise of common stock warrants | $1,826 | $9,809 | ' | ' | ' | ' | ' | ' | ' | ' | |
| Exercise of common stock warrants (in shares) | 611,606 | ' | ' | ' | ' | ' | ' | ' | ' | ' | |
| Common stock warrants outstanding [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | |
| Number of Shares Exercisable (in shares) | 334,764 | ' | 233,309 | 3,502 | 1 | 97,952 | ' | ' | ' | ' | |
| Exercise Price (in dollars per share) | $4.54 | [1] | ' | $2.87 | $157.08 | $2.45 | $3.08 | ' | ' | ' | ' |
| Expiration Date | ' | ' | 13-Dec-15 | 26-Sep-15 | 22-Dec-14 | 12-Aug-15 | ' | ' | ' | ' | |
| Assumptions to obtain the fair value of the warrants [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | |
| Fair value | ' | ' | ' | ' | ' | ' | $0 | $133 | $654 | $6,933 | |
| Black Scholes inputs [Abstract] | ' | ' | ' | ' | ' | ' | ' | ' | ' | ' | |
| Stock price (in dollars per share) | ' | ' | ' | ' | ' | ' | $9.59 | $13.02 | $9.59 | $13.02 | |
| Exercise price (in dollars per share) | ' | ' | ' | ' | ' | ' | $2.45 | $2.45 | $3.08 | $3.08 | |
| Risk free interest rate (in hundredths) | ' | ' | ' | ' | ' | ' | 0.01% | 0.13% | 0.08% | 0.33% | |
| Volatility (in hundredths) | ' | ' | ' | ' | ' | ' | 95.00% | 95.00% | 95.00% | 95.00% | |
| Expected term (years) | ' | ' | ' | ' | ' | ' | '0 years 3 months | '1 year | '0 years 9 months | '1 year 9 months | |
| Dividend (in hundredths) | ' | ' | ' | ' | ' | ' | 0.00% | 0.00% | 0.00% | 0.00% | |
| [1] | Weighted-average exercise price | ||||||||||
STOCKBASED_COMPENSATION_Detail
| STOCK-BASED COMPENSATION (Details) (USD $) | 3 Months Ended | 9 Months Ended | ||
| In Thousands, except Share data, unless otherwise specified | Sep. 30, 2014 | Sep. 30, 2013 | Sep. 30, 2014 | Sep. 30, 2013 |
| Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ' | ' | ' | ' |
| Proceeds from the exercise of common stock options | ' | ' | $4,023 | $1,516 |
| Employee Stock Purchase Plan [Abstract] | ' | ' | ' | ' |
| Number of shares authorized for repurchase (in shares) | 1,000,000 | ' | 1,000,000 | ' |
| Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
| Total Stock-Based Compensation Expense | 3,575 | 1,823 | 9,224 | 5,420 |
| Cost of Goods Sold [Member] | ' | ' | ' | ' |
| Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
| Total Stock-Based Compensation Expense | 56 | 0 | 143 | 0 |
| Research and Development Expense [Member] | ' | ' | ' | ' |
| Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
| Total Stock-Based Compensation Expense | 427 | 381 | 1,725 | 1,123 |
| General and administrative [Member] | ' | ' | ' | ' |
| Employee Service Share-based Compensation, Allocation of Recognized Period Costs [Line Items] | ' | ' | ' | ' |
| Total Stock-Based Compensation Expense | 3,092 | 1,442 | 7,356 | 4,297 |
| Stock Options [Member] | ' | ' | ' | ' |
| Option shares [Roll Forward] | ' | ' | ' | ' |
| Outstanding, beginning of period (in shares) | 9,952,407 | ' | 8,217,674 | ' |
| Granted (in shares) | 539,386 | ' | 2,923,104 | ' |
| Exercised (in shares) | -578,147 | ' | -1,042,352 | ' |
| Canceled (in shares) | -626,344 | ' | -811,124 | ' |
| Outstanding, end of period (in shares) | 9,287,302 | ' | 9,287,302 | ' |
| Weighted-average exercise price [Roll Forward] | ' | ' | ' | ' |
| Outstanding, beginning of period (in dollars per share) | $7.72 | ' | $6.05 | ' |
| Granted (in dollars per share) | $8.73 | ' | $12.35 | ' |
| Exercised (in dollars per share) | $4.17 | ' | $3.86 | ' |
| Canceled (in dollars per share) | $9.69 | ' | $10.83 | ' |
| Outstanding, end of period (in dollars per share) | $7.86 | ' | $7.86 | ' |
| 2010 Equity Incentive Plan [Member] | Stock Options [Member] | ' | ' | ' | ' |
| Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ' | ' | ' | ' |
| Proceeds from the exercise of common stock options | $2,400 | ' | $4,000 | ' |
| Number of shares available for grant (in shares) | 1,746,862 | ' | 1,746,862 | ' |