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PTC Therapeutics Provides Regulatory Update on Translarna™ (ataluren) in Europe
WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion
About this update from Ptc Therapeutics, Inc.
[{"type":"text","content":"WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna.\n\n\"We are of course disappointed that after this prolonged period of review the European Commission has decided to adopt the CHMP negative opinion on Translarna,\" said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. \"The EC's indication that European Union member states have a mechanism to maintain treatment speaks to the safety, benefit and lack of alternative therapies for boys and young men with nonsense mutation Duchenne muscular dystrophy. We look forward to working on a country-by-country basis to provide commercial drug where possible.\"\nAbout 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 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.\nAbout Duchenne Muscular Dystrophy (Duchenne)Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20's due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystroph...