PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™ (ataluren), which will result in the withdrawal of the therapy for Duchenne patients with nonsense mutations  in Europe. The decision to withdraw Translarna is disheartening and weighs heavily on the hopes of families seeking effective treatments.

In the United States, PTC Therapeutics, Inc. plans to continue to move forward with the U.S. Food and Drug Administration (FDA) in discussing a potential path to submit a New Drug Application (NDA) for Translarna. PTC has indicated that a meeting is scheduled for the first quarter of 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. 

Read PTC’s press release here.