PTC Therapeutics reports CHMP opinion against Translarna marketing authorization

PTC Therapeutics announced that the Committee for Medicinal Products for Human Use, or CHMP, of the European Medicines Agency, or EMA, has given a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD. The negative opinion also applies to the renewal of the existing conditional authorization. PTC plans to submit a request for re-examination per EMA guidelines. Translarna will remain on the market and available to patients with nmDMD until the re-examination process is completed. Based on CHMP procedural guidance, the opinion following the re-examination process would be expected to occur in January 2024, with EC ratification of the opinion within the following 67 days. “We are surprised and extremely disappointed by the CHMP decision, given the well-established and favorable safety and efficacy profile of Translarna,” said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics. “Of course, this decision is most devastating for the hundreds of boys and young men in Europe with nonsense mutation DMD, for whom no other approved therapies are available. We will be submitting a request for re-examination to the CHMP to reverse this opinion, as we have done previously in the regulatory history of Translarna in Europe.”