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Revenue recognition | 9 Months Ended
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Sep. 30, 2025
Revenue from Contract with Customer [Abstract] |
Revenue recognition | 11. Revenue recognition Net product sales The Company views its operations and manages its business in one operating segment: life science. During the three and nine months ended September 30, 2025 and 2024, net product revenues consisted of the following: ​
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | Three Months Ended September 30,
​ | ​ | ​ | 2025 | ​ | 2024
​ | ​ | ​ | United States | ​ | ​ | International | ​ | ​ | Total | ​ | ​ | United States | ​ | ​ | International | ​ | ​ | Total
Translarna | ​ | $ | — | ​ | $ | 50,665 | ​ | $ | 50,665 | ​ | $ | — | ​ | $ | 72,283 | ​ | $ | 72,283
Emflaza | ​ | ​ | 35,160 | ​ | ​ | — | ​ | ​ | 35,160 | ​ | ​ | 51,941 | ​ | ​ | — | ​ | ​ | 51,941
Sephience | ​ | ​ | 14,365 | ​ | ​ | 5,198 | ​ | ​ | 19,563 | ​ | ​ | — | ​ | ​ | — | ​ | ​ | —
Upstaza/Kebilidi | ​ | ​ | 6,898 | ​ | ​ | 8,837 | ​ | ​ | 15,735 | ​ | ​ | — | ​ | ​ | — | ​ | ​ | —
All other products | ​ | ​ | — | ​ | ​ | 9,833 | ​ | ​ | 9,833 | ​ | ​ | — | ​ | ​ | 11,197 | ​ | ​ | 11,197
Total net product revenue | ​ | $ | 56,423 | ​ | $ | 74,533 | ​ | $ | 130,956 | ​ | $ | 51,941 | ​ | $ | 83,480 | ​ | $ | 135,421
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | Nine Months Ended September 30,
​ | ​ | 2025 | ​ | 2024
​ | ​ | ​ | United States | ​ | ​ | International | ​ | ​ | Total | ​ | ​ | United States | ​ | ​ | International | ​ | ​ | Total
Translarna | ​ | $ | — | ​ | $ | 196,289 | ​ | $ | 196,289 | ​ | $ | — | ​ | $ | 246,217 | ​ | $ | 246,217
Emflaza | ​ | ​ | 119,303 | ​ | ​ | — | ​ | ​ | 119,303 | ​ | ​ | 156,718 | ​ | ​ | — | ​ | ​ | 156,718
Sephience | ​ | ​ | 14,365 | ​ | ​ | 5,198 | ​ | ​ | 19,563 | ​ | ​ | — | ​ | ​ | — | ​ | ​ | —
Upstaza/Kebilidi | ​ | ​ | 6,898 | ​ | ​ | 29,385 | ​ | ​ | 36,283 | ​ | ​ | — | ​ | ​ | 9,648 | ​ | ​ | 9,648
All other products | ​ | ​ | — | ​ | ​ | 31,273 | ​ | ​ | 31,273 | ​ | ​ | — | ​ | ​ | 33,662 | ​ | ​ | 33,662
Total net product revenue | ​ | $ | 140,566 | ​ | $ | 262,145 | ​ | $ | 402,711 | ​ | $ | 156,718 | ​ | $ | 289,527 | ​ | $ | 446,245
​ Disaggregated net product revenues by country for the three and nine months ended September 30, 2025 and 2024 are as follows: ​
​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​ | ​
​ | ​ | Three Months Ended September 30, | ​ | Nine Months Ended September 30,
​ | ​ | 2025 | ​ | 2024 | ​ | 2025 | ​ | 2024
United States | ​ | $ | 56,423 | ​ | $ | 51,941 | ​ | $ | 140,566 | ​ | $ | 156,718
Russia | ​ | ​ | 25,107 | ​ | ​ | 965 | ​ | ​ | 81,745 | ​ | ​ | 55,716
Brazil | ​ | ​ | 9,222 | ​ | ​ | 29,219 | ​ | ​ | 59,678 | ​ | ​ | 68,304
All other countries | ​ | ​ | 40,204 | ​ | ​ | 53,296 | ​ | ​ | 120,722 | ​ | ​ | 165,507
Total net product revenue | ​ | $ | 130,956 | ​ | $ | 135,421 | ​ | $ | 402,711 | ​ | $ | 446,245
​ For the three months ended September 30, 2025, four of the Company’s distributors each accounted for over 10% of the Company’s net product sales. For the three months ended September 30, 2024, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales. For the nine months ended September 30, 2025, three of the Company’s distributors each accounted for over 10% of the Company’s net product sales. For the nine months ended September 30, 2024, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales. As of September 30, 2025 and December 31, 2024, the Company does not have a contract liabilities balance related to net product sales, and has not made significant changes to the judgments made in applying ASC Topic 606. Collaboration and License revenue In November 2011, the Company and the SMA Foundation entered into a licensing and collaboration agreement with Roche. Under the terms of the SMA License Agreement, Roche acquired an exclusive worldwide license to the Company’s SMA program. Under the agreement, the Company is eligible to receive additional payments from Roche if specified events are achieved with respect to each licensed product, including up to $135.0 million in research and development event milestones, up to $325.0 million in sales milestones upon achievement of specified sales events, and up to double digit royalties on worldwide annual net sales of a commercial product. The SMA program currently has one approved product, Evrysdi, which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. As of September 30, 2025, the Company does not have any remaining research and development event milestones that can be received. The remaining potential sales milestones that can be received is $150.0 million. For the three and nine months ended September 30, 2025, collaboration revenue related to the SMA License Agreement with Roche was immaterial. For the three and nine months ended September 30, 2024, the Company did not recognize collaboration revenue related to the SMA License Agreement with Roche. ​ In addition to research and development and sales milestones, the Company is eligible to receive up to double-digit royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the three and nine months ended September 30, 2025, the Company has recognized $70.8 million and $164.8 million of royalty revenue, respectively, related to Evrysdi. For the three and nine months ended September 30, 2024, the Company has recognized $61.4 million and $145.7 million of royalty revenue, respectively, related to Evrysdi. In November 2024, the Company entered into the Novartis Agreement with Novartis related to the Company’s votoplam HD program. The transaction contemplated by the Novartis Agreement closed in January 2025. Under the Novartis Agreement, and upon the closing of the transaction, the Company received an upfront nonrefundable payment of $1.0 billion on the effective date and can receive up to $1.9 billion in development, regulatory and sales milestones, a 40% share of U.S. profits and losses, and tiered double-digit royalties on ex-U.S. sales. The Company evaluated the Novartis Agreement in order to determine the proper accounting treatment and concluded that the arrangement was not subject to ASC 730 or ASC 808, as the upfront payment was nonrefundable with no obligation for the Company to repay it and as the Company is not exposed to significant risks. The Company evaluated the arrangement under ASC 606, as a contract with a customer was determined to exist. Pursuant to the Novartis Agreement, the Company was required to transfer the applicable licenses and related manufacturing and other know how to Novartis and to complete the ongoing Phase 2A clinical trial and continue the ongoing open label extension (“OLE”) clinical trial pursuant to its existing development plan, with the goal of transitioning the ongoing OLE clinical trial to Novartis within 12 months after the effective date of the Novartis Agreement. Novartis will be responsible for all other development of licensed compounds and licensed products and the manufacture and commercialization of licensed compounds and licensed products worldwide. Therefore, the Company determined that there were three material and distinct performance obligations: the transfer of the licenses and know-how, completion of the Phase 2A clinical trial, and continuing the OLE clinical trial until transitioned to Novartis. As of September 30, 2025, all performance obligations were considered to be substantially complete. The Company determined that the transaction included the fixed consideration of the $1.0 billion and variable consideration in the form of milestones, profit share, and royalties. Management evaluated the variable consideration under ASC 606 and determined that it would be fully constrained until the contingencies were resolved or any applicable sales occurred. Management allocated the transaction price of $1.0 billion to the performance obligations based on the guidance in ASC 606. During the three and nine months ended September 30, 2025, the Company recognized $9.3 million and $1.0 billion in license revenues, respectively, related to Novartis. As all performance obligations are considered to be substantially complete, there is no remaining deferred revenue related to the Novartis Agreement on the consolidated balance sheet as of September 30, 2025. Collaboration and license revenue during the nine months ended September 30, 2025 was partially offset by $3.5 million related to a refund for a prior collaboration arrangement in relation to votoplam. Manufacturing Revenue ​ For the three and nine months ended September 30, 2025, the Company did not recognize any revenue related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the three months ended September 30, 2024, the Company did not recognize any revenue related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the nine months ended September 30, 2024, the Company recognized $1.7 million of manufacturing revenue related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. The Company has not made significant changes to the judgments made in applying ASC Topic 606 for the three and nine months ended September 30, 2025 and 2024. As of September 30, 2025 and December 31, 2024, the Company does not have a contract liabilities balance or contract assets related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. In June 2024, the Company sold its gene therapy manufacturing business in Hopewell Township, New Jersey. Accordingly, the Company does not expect to have manufacturing revenue going forward.

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- Definition The entire disclosure of revenue from contract with customer to transfer good or service and to transfer nonfinancial asset. Includes, but is not limited to, disaggregation of revenue, credit loss recognized from contract with customer, judgment and change in judgment related to contract with customer, and asset recognized from cost incurred to obtain or fulfill contract with customer. Excludes insurance and lease contracts. + References Reference 1: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 7 -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-7 Reference 2: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 9 -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-9 Reference 3: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 10 -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-10 Reference 4: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 15 -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-15 Reference 5: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 12 -Subparagraph (a) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-12 Reference 6: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 12 -Subparagraph (b) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-12 Reference 7: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 12 -Subparagraph (c) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-12 Reference 8: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 12 -Subparagraph (d) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-12 Reference 9: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 12 -Subparagraph (e) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-12 Reference 10: http://www.xbrl.org/2003/role/disclosureRef -Topic 606 -SubTopic 10 -Name Accounting Standards Codification -Section 50 -Paragraph 13 -Subparagraph (b)(2) -Publisher FASB -URI https://asc.fasb.org/1943274/2147479806/606-10-50-13 Reference 11: http://www.xbrl.org/2003/role/disclosureRef -Name Accounting Standards Codification -Topic 606 -Publisher FASB -URI https://asc.fasb.org/606/tableOfContent + Details
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