12. Revenue recognition

Net product sales

The Company views its operations and manages its business in one operating segment: life science.

During the years ended December 31, 2025, 2024, and 2023, net product revenues consisted of the following:

Years ended December 31,

2025

2024

2023

United States

International

Total

United States

International

Total

United States

International

Total

Translarna

$

235,299

235,299

$

321,071

321,071

$

332,753

332,753

Emflaza

146,361

146,361

207,215

207,215

255,087

255,087

Sephience

95,045

16,108

111,153

Upstaza/Kebilidi

10,779

45,847

56,626

16,913

16,913

18,841

18,841

All other products

37,264

37,264

36,946

36,946

31,496

31,496

Total net product revenue

$

252,185

$

334,518

$

586,703

$

207,215

$

374,930

$

582,145

$

255,087

$

383,090

$

638,177

Disaggregated net product revenues by country for the years ended December 31, 2025, 2024, and 2023 are as follows:

Years ended December 31,

2025

2024

2023

United States

$

252,185

$

207,215

$

255,087

Russia

100,445

105,377

85,974

Brazil

65,661

72,081

58,606

All other countries

168,412

197,472

238,510

Total net product revenue

$

586,703

$

582,145

$

638,177

Net product revenue for sales of Translarna in France are not depicted in the net product revenue tables above. Refer to Note 2 for further details.

For the year ended December 31, 2025, four of the Company’s distributors each accounted for over 10% of the Company’s net product sales. For the years ended December 31, 2024 and 2023, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales. 

As of December 31, 2025 and 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 certain sales events, and up to double digit royalties on worldwide annual net sales of a commercial product.

For the years ended December 31, 2025 and 2024 collaboration revenue related to the SMA License Agreement with Roche was immaterial. For the year ended December 31, 2023, the Company recognized collaboration revenue related to the SMA License Agreement with Roche of $100.0 million. The below summarizes the milestone achievements associated with the Company’s SMA program during the years ended December 31, 2025, 2024 and 2023.

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. For the year ended December 31, 2023, the Company recognized a sales milestone of $100.0 million for the achievement of $1.5 billion in worldwide annual net sales from Evrysdi. The remaining potential sales milestone as of December 31, 2025 is $150.0 million upon achievement of certain sales events. As of December 31, 2025, the Company does not have any remaining research and development milestones that can be received.

In addition to research and development and sales milestones, the Company is eligible to recognize royalties on worldwide annual net sales of a commercial product under the SMA License Agreement. For the years ended December 31, 2025, 2024 and 2023 the Company has recognized $244.2 million, $203.9 million, and $168.9 million of royalty revenue related to Evrysdi, respectively.

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 Phase 2A clinical trial and continue the ongoing open label extension (“OLE”) clinical trial of votoplam 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. As of December 31, 2025, these performance obligations were completed. 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 December 31, 2025, all performance obligations were completed.

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 year ended December 31, 2025, the Company recognized $1.0 billion in license revenues related to Novartis. For the years ended December 31, 2024 and 2023, the Company did not recognize license revenue related to Novartis. As all performance obligations are complete, there is no remaining deferred revenue related to the Novartis Agreement on the consolidated balance sheet as of December 31, 2025. Collaboration and license revenue during the year ended December 31, 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 year ended December 31, 2025, the Company did not recognize manufacturing revenue. For the years ended December 31, 2024 and 2023, the Company recognized $1.7 million and $7.7 million of manufacturing revenue, respectively, related to the production of 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 years ended 2025, 2024, and 2023.

As of December 31, 2025 and 2024, the Company did not have contract liabilities balances related to the production of plasmid DNA and AAV vectors for gene therapy applications for external customers. For the year ended December 31, 2024, the Company recognized $0.8 million related to the amounts included in the contract liability balance at the beginning of the period.

As of December 31, 2025 and 2024, the Company has no contract assets related to plasmid DNA and AAV production 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. There are no remaining performance obligations as of December 31, 2025 and 2024.

Historical Timeline

Fiscal YearFiled
2025Feb 19, 2026Showing above
2024Feb 27, 2025
2023Feb 29, 2024
2022Feb 21, 2023
2021Feb 22, 2022
2020Feb 25, 2021
2019Mar 2, 2020
2018Mar 1, 2019

About Revenue Disclosures

Revenue disclosures under ASC 606 explain how a company identifies performance obligations, allocates transaction prices, and determines when revenue is recognized. This section is essential for understanding whether reported revenue reflects genuine economic activity or aggressive accounting choices. Analysts examine the mix of point-in-time versus over-time recognition, which directly affects revenue timing and comparability.

Key signals: rising contract liabilities (deferred revenue) suggest strong future revenue visibility, while declining contract assets may indicate slowing project milestones. Watch for variable consideration estimates — rebates, returns, and performance bonuses that require management judgment. Significant changes in disaggregated revenue by geography or product line can reveal shifting business mix before it appears in headline numbers. Compare revenue growth against contract liability growth to assess sustainability, and scrutinize any changes in the timing of recognition that coincide with earnings pressure.