PTC Updates on Regulatory Activities for Translarna in Duchenne

 As planned, PTC has submitted the briefing document as part of the re-examination of the initial Committee for Medicinal Products for Human Use (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna in Europe. 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.

In the United States, PTC recently participated in a Type C meeting 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.

"PTC continues to work to secure global registrations that will enable access to Translarna for patients with nmDMD worldwide," said Matthew B. Klein, M.D., Chief Executive Officer of PTC. "The data collected to date demonstrate the short-and long-term benefits of Translarna on key functional aspects of disease, including ambulatory and neuromuscular function. In Europe, we believe we can address the concerns raised by the CHMP in its initial negative opinion on the renewal of Translarna conditional authorization. In the U.S., we look forward to continuing to work collaboratively with FDA on the contents of a potential NDA resubmission."

About Translarna™ (ataluren)
Translarna (ataluren), discovered and developed by PTC Therapeutics, is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne. Translarna, the tradename of ataluren, is licensed in multiple countries including Great Britain, Northern Ireland and the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged 2 years and older. Ataluren is an investigational new drug in the United States.

https://www.prnewswire.com/news-releases/ptc-therapeutics-provides-updates-on-translarna-regulatory-activities-302006665.html