PTC Therapeutics, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has maintained its negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD) following re-examination. The opinion will now be reviewed by the European Commission (EC) which is expected to decide on opinion adoption in approximately 67 days.
"CHMP again based its decision on the results of the primary analysis subpopulation of Study 041 instead of the totality of evidence for Translarna. That evidence includes data across three placebo-controlled trials and our STRIDE registry that demonstrates consistent short and long-term efficacy as well as safety supportive of continued authorization. In addition, the CHMP opinion is clearly against the expressed wishes of physicians, patients and families throughout Europe," said Matthew B. Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "As Translarna will remain authorized in Europe pending review by the European Commission, we will continue to ensure Translarna is available to boys and young men living with nonsense mutation Duchenne muscular dystrophy. In addition, PTC will ensure the EC has all possible evidence to support continued authorization."
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