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PTC Therapeutics Announces European Medicines Agency Validation of Marketing Authorization Application for Ataluren in Duchenne Muscular Dystrophy
Date:12/6/2012

SOUTH PLAINFIELD, N.J., Dec. 6, 2012 /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) today announced that the European Medicines Agency (EMA) has validated a Marketing Authorization Application (MAA) seeking conditional approval for ataluren, an investigational new drug for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Validation of the MAA confirms that the submission is complete and begins the EMA's Committee for Human Medicinal Products' (CHMP) review process. Ataluren is the only treatment currently in clinical development targeting the cause of disease in patients with a nonsense mutation.

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"Ataluren is a promising potential therapy for nonsense mutation Duchenne muscular dystrophy," stated Dr. Thomas Voit, Medical and Scientific Director, Institut de Myologie. "PTC has developed a standard for DMD clinical trials and now the DMD community can share in the achievement of the first MAA ever filed for DMD. We appreciate PTC's commitment to the clinical development of ataluren for this severe disorder for which only palliative treatment options currently exist." 

The Marketing Authorization Application's submission was accepted by EMA for review on the basis of a 48-week, 174-patient Phase 2b study showing that nmDMD patients treated with ataluren (10, 10, 20 mg/kg given daily) walked on average 31.3 meters farther than patients on placebo, as measured by the change in six-minute walk distance (6MWD) from baseline to Week 48. Patients receiving ataluren demonstrated a slower rate of decline in ambulation, based on an analysis of time to 10 percent worsening in 6MWD. While 44 percent of patients receiving placebo declined 10 percent or more in walking as measured by the 6-minute walk test (6MWT), only 26 percent of patients receiving ataluren declined 10 percent or more. S
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SOURCE PTC Therapeutics, Inc.
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