PTC Therapeutics announced this morning that they have received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency, regarding the company’s application for a conditional marketing authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older. 

CureDuchenne was happy to play a role by being one of the initial funders of this research.

As the Duchenne community is aware, this is a stark reversal of the CHMP’s negative opinion that the company received in January 2014. It was brought about after PTC requested a re-examination of the data from their 48-week Phase IIb clinical trial. The decision won’t affect the ongoing Phase III confirmatory trial known as ACT DMD, which is due to complete its enrollment in mid-2014.

This CHMP decision is a major milestone for the company and the Duchenne community alike. It brings us one step closer to treatment options and the positive engagement of CHMP should benefit other potential DMD therapies that are in development within various companies.

For more information please see the following links.

Company press release: 

Company conference call: 


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