PTC Therapeutics: CHMP issues negative opinion for Translarna renewal

PTC Therapeutics announced that the Committee for Medicinal Products for Human Use of the European Medicines Agency issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna. “We are disappointed that the CHMP has maintained its negative opinion on the Translarna authorization which will result in the withdrawal of a therapy for patients in Europe with nonsense mutation Duchenne Muscular Dystrophy that the data support is safe and effective,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “The CHMP’s decision, which is against the stated wishes of the patient community and expert physicians, will be devastating for children and young men in Europe for whom no other disease-modifying therapies are available.” Per European regulations, the European Commission has 67 days to adopt the opinion.