PTC Therapeutics, Inc. has shared an update on regulatory activities in Europe and the United States for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants. 

Europe: European Medicines Agency (EMA) Update

Following the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna in Europe in September 2023, PTC has submitted the briefing document as part of the re-examination procedure.

PTC has indicated that the re-examination procedure will focus only on the renewal of the existing conditional marketing authorization. The procedure is expected to last until late January 2024, when an opinion will be provided by CHMP. This opinion will then be sent for adoption by the European Commission within the following 67 days.

PPMD is pleased to hear this news and is hopeful that CHMP will consider the re-examination. Pulling marketing authorization for Translarna would leave Duchenne patients in Europe without variant-specific treatment options, further limiting therapeutic options at clinicians’ disposal for treating Duchenne.

United States: Food and Drug Administration (FDA) Update

In the United States, PTC recently met with the U.S. Food and Drug Administration (FDA) to discuss a potential path to a resubmission of a New Drug Application (NDA) for Translarna. The discussion focused on the totality of evidence collected to date from the Translarna clinical trials and the STRIDE registry. Based on the discussion, FDA suggested that PTC request a follow-up meeting to align on the specific contents that could support a potential NDA filing. PTC expects this meeting will occur in the first quarter of 2024.

In January 2023, PPMD held a webinar with PTC to discuss the company’s activities to bring ataluren to US patients and sent a request to the FDA urging them to accept PTC’s NDA and conduct a full review of ataluren. PPMD believes that regardless of the outcome, the FDA’s review is critical for allowing families to make informed decisions about potential treatment options. 

PPMD is pleased to learn that PTC expects this follow-up meeting early next year  and hopes  FDA will  accept and review PTC’s NDA expeditiously.

Read PTC’s community letter here.