CHMP recommends Translarna for Duchenne Muscular Dystrophy-PTC Therapeutics

Following a request for re-examination, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion regarding the application for a conditional marketing authorization of Translarna (ataluren), from PTC Therapeutics, for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD) in ambulatory patients aged five years and older. The CHMP made a negative recommendation in January 2014.

The Phase IIb clinical trial provided strong evidence that Translarna slows disease progression as measured by the 6-minute walk test. A clinically meaningful 31.3 meter benefit in 6-minute walk distance, relative to placebo, was achieved in 48 weeks of treatment in patients five years and older and this was supported by positive trends in multiple secondary efficacy endpoints.