CHMP Issues Negative Opinion on Translarna™ Following European Commission Request for Review

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[{"type":"text","content":"- PTC to submit a request for re-examination -\nWARREN, N.J., June 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD. This opinion follows the return of the previously issued negative opinion by the European Commission (EC) for re-review.\n\nPTC plans to request re-examination of the opinion. The marketing authorization for Translarna remains in effect, pending the outcome of the re-examination procedure and subsequent EC ratification. Based on the timeline of these procedures, PTC expects Translarna to remain on the market through the end of 2024 even if the negative opinion is maintained and ratified.\n\"We are disappointed that the CHMP has maintained its negative opinion on Translarna despite the request of the European Commission to consider the totality of evidence including the STRIDE real world evidence in a revised opinion,\" said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. \"The Scientific Advisory Group convened as part of this most recent review procedure concluded that the evidence of significant efficacy provided by STRIDE 'should not be ignored.' This view of the CHMP expert committee is consistent with the view of DMD key opinion leaders worldwide. We will request re-examination of the CHMP opinion to ensure that we make every effort to maintain availability of Translarna on the market for as long as possible for boys and young men with nmDMD.\"\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 State...

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