PTC Therapeutics, Inc. announced today that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use’s (CHMP) January 2024 negative opinion on the annual renewal of the conditional marketing authorization of Translarna™ (ataluren), and has returned the opinion to the CHMP for re-evaluation.  As a result, Translarna, a dystrophin restoration therapy via stop-codon readthrough for the treatment of nonsense mutation Duchenne (nmDMD), remains on the market and available for patients in Europe consistent with its current marketing authorization.

According to PTC, the EC has asked the CHMP to further consider the totality of evidence, including data from patient registries and real-world evidence, in a revised opinion. 

Additionally, PTC has been informed by the European Medicines Agency (EMA) that the Scientific Advisory Group (SAG) meeting for Translarna held in September 2023, and all the procedural steps that followed, will be considered invalid. Furthermore, the input from the SAG meeting held in September 2023, as well as the meeting held in January 2024, will not be considered by the CHMP in any future evaluation of Translarna. PTC has indicated that the company “looks forward to working collaboratively with CHMP on next steps once they are defined.”

Translarna in the U.S.

While the EC’s decision does not directly affect regulatory activities in the U.S., PPMD is pleased to hear this news for Duchenne patients in Europe and we look forward to the CHMP’s re-evaluation. 

In the United States, PTC Therapeutics, Inc. announced in March that based on feedback from the FDA, the company plans to re-submit a New Drug Application (NDA) for Translarna™ (ataluren) by mid-2024. 

PPMD previously held a webinar with PTC to discuss the company’s activities to bring ataluren to U.S. patients and sent a request to the FDA urging them to accept PTC’s NDA and conduct a full review of ataluren. We believe that regardless of the outcome, the FDA’s review is critical for allowing families to make informed decisions about potential treatment options. We appreciate FDA’s willingness to review the resubmission and  remain optimistic about the possibility of an approved therapy that specially targets nmDMD.

Read PTC’s press release here.