Update for the European Duchenne Community

We have received several questions regarding the status of the re-examination process for the recent European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) opinion on the conditional authorization for Translarna™ (ataluren). We want to confirm the re-examination procedure is ongoing and our PTC team is fully dedicated and working incredibly hard to ensure that we address concerns raised by the CHMP in its previous review of the Translarna data. As a reminder, despite the CHMP opinion published in September, Translarna remains available for current patients in Europe and physicians can continue to prescribe Translarna for new patients at this time.

We have elected to pursue re-examination of the negative opinion on the renewal of the conditional marketing authorization only. We plan to demonstrate the evidence of clinical benefit recorded in the Translarna clinical trials, across a number of disease endpoints. In addition, we will discuss how the data collected in the real-world STRIDE registry uniquely confirms the long-term benefit of Translarna in delaying loss of ambulation. This evidence of short and long-term clinical benefit, along with the safety data collected in approximately 3,000 patients support that the previously established benefit-risk profile of Translarna remains unchanged.

According to EMA guidelines, the re-examination period is likely to continue through late January 2024 when the CHMP will provide an opinion on the continued conditional marketing authorization of Translarna. The CHMP’s opinion will then be submitted to the European Commission (EC) for adoption, which is likely to occur 67 days later.       

We understand that this may be a difficult and confusing time for patients, their families and treating physicians. We are incredibly grateful for your continued support, and we are fully committed to making every effort in the re-examination process to reverse the negative opinion to enable the continued availability of Translarna to boys with nonsense mutation Duchenne muscular dystrophy in Europe.