PTC Therapeutics announced on October 18, 2024, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion. This decision concerns the renewal of the conditional marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD) following re-examination.
The CHMP's opinion will now be reviewed by the European Commission (EC), which is expected to make a final decision on opinion adoption in approximately 67 days. PTC Therapeutics expressed disappointment, stating that the CHMP based its decision on a subpopulation analysis of Study 041 rather than the totality of evidence.
PTC highlighted that the evidence includes data from three placebo-controlled trials and its STRIDE registry, which demonstrates consistent short and long-term efficacy and safety. Translarna will remain authorized in Europe pending the EC's review, and PTC plans to ensure its continued availability to patients.
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