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Revenue recognition
 3 Months Ended
Mar. 31, 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 months ended March 31, 2025 and 2024, net product sales outside of the United States were $105.6 million and $120.1 million, respectively, consisting of sales of Translarna, Tegsedi, Waylivra, and Upstaza. Translarna net revenues made up $86.2 million and $103.6 million of the net product sales outside of the United States for the three months ended March 31, 2025 and 2024, respectively. During the three months ended March 31, 2025 and 2024, net product sales in the United States were $47.8 million and $57.5 million, respectively, consisting solely of sales of Emflaza. During the three months ended March 31, 2025, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $47.8 million and $38.5 million of the net product sales, respectively. During the three months ended March 31, 2024, two countries, the United States and Russia, accounted for at least 10% of the Company’s net product sales, representing $57.5 million and $52.6 million of the net product sales, respectively. For the three months ended March 31, 2025 and 2024, two of the Company’s distributors each accounted for over 10% of the Company’s net product sales.

As of March 31, 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 March 31, 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 months ended March 31, 2025 and 2024, the Company did not recognize collaboration revenue related to the SMA License Agreement with Roche.

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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 months ended March 31, 2025 and 2024, the Company has recognized $36.4 million and $31.2 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 PTC518 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 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 March 31, 2025, the first two performance obligations were considered to be substantially complete. The OLE Clinical Trial is still ongoing and is expected to be fully transitioned to Novartis by the end of 2025.

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 months ended March 31, 2025, the Company recognized $989.8 million in license revenues related to performance obligations for Novartis already completed. The remaining $10.2 million was recorded as deferred revenue in the consolidated balance sheet and will be recognized over time as the work related to the OLE Clinical Trial is performed. Collaboration and license revenue during the three months ended March 31, 2025, was partially offset by $3.6 million related to a refund for a prior collaboration arrangement in relation to PTC518.

Manufacturing Revenue

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For the three months ended March 31, 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 March 31, 2024, the Company recognized $1.4 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 months ended March 31, 2025 and 2024.

As of March 31, 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.