PTC Therapeutics Receives Positive Opinion from CHMP for Translarna™ (ataluren)

- The first treatment for the underlying cause of Duchenne muscular dystrophy -

SOUTH PLAINFIELD, New Jersey, May 23, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that following its 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 company's application for a conditional marketing authorization of Translarna^TM (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

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"We are very pleased with the outcome of the CHMP review of Translarna's marketing authorization application (MAA) and the level of engagement we experienced with CHMP members throughout the review process," said Robert J. Spiegel, M.D., Chief Medical Officer of PTC Therapeutics, Inc. "We are grateful to the patients, families, advocacy groups and physicians who have supported PTC Therapeutics through many years of research and development of Translarna. It is important to note that this journey continues through the completion of our Phase 3 Translarna confirmatory trial in nmDMD (ACT DMD) which is a high priority for PTC and the DMD community."

Dr. Craig McDonald, Professor of Physical Medicine and Rehabilitation at the University of California, Davis, who developed and validated the 6-minute walk test as a primary clinical endpoint in Duchenne muscular dystrophy (DMD) stated, "This is a historic day for the DMD community. Translarna is the first treatment for the underlying cause of nonsense mutation DMD to receive a positive opinion from the CHMP. The Phase 2b 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. In addition, in the prespecified group of patients with less than a 350 meter baseline 6-minute walk distance, a 68 meter benefit was observed in patients treated with 40 mg/kg Translarna given daily, relative to placebo. A conditional approval by the European Commission based on this positive opinion would allow children with nmDMD in the European Union to gain access to Translarna while PTC Therapeutics completes its ongoing confirmatory trial."