PTC Therapeutics today shared that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended not renewing the Marketing Authorization for Translarna™ (ataluren), a treatment for Duchenne patients with nonsense variants. The EMA also recommended not renewing the existing conditional authorization of Translarna.

Translarna received conditional marketing authorization in Europe in 2014, which was then renewed in 2017. PTC emphasizes that Translarna has shown a favorable safety profile, with over 3,000 patients treated to date. While the randomized clinical trials did not meet the primary endpoint of change in the Six-Minute Walk Distance test from baseline to week 48, data collected in the STRIDE Registry has indicated positive long-term effects of delaying loss of ambulation and slowing the decline of Forced Vital Capacity (FVC).

PPMD is disappointed to learn this news, and hopes the re-examination by the CHMP will be considered. 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.

PTC plans to submit a request for re-examination per EMA guidelines. Translarna will remain on the market and available to Duchenne patients in Europe with nonsense variants until the re-examination process is completed, which is expected to occur in January 2024. During a conference call this morning, PTC indicated that the company plans to continue working towards submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA). 

Some patients in the US have been receiving ataluren for as long as 14 years and have consequently not been able to participate in other clinical trials. The EMA’s decision does not directly affect patients in the US who are currently receiving ataluren. However, in the devastating case in which the FDA follows the EMA’s opinion, families of patients who have been on ataluren for an extended period of time may be put in a very difficult position. 

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 working with families of patients both currently receiving ataluren and those who would be eligible for the therapy, as well as other organizations and community partners, to submit additional letters to the FDA urging them to accept and review PTC’s NDA expeditiously.