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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

afety results showed that ataluren was generally well tolerated and adverse events were similar to placebo. Serious adverse events were infrequent and none were considered to be related to ataluren.

"We applaud PTC for its continued commitment to the DMD patient community and its comprehensive clinical development of ataluren," said Dr. Elizabeth Vroom, Chair, United Parent Project Muscular Dystrophy. "There is an urgent need for new therapeutic options for the patients and families and the path to approval of a new therapy can be arduous. We are extremely grateful to PTC not only for the robust work on developing ataluren but for helping set standards in our community which will benefit future drug development."

PTC also announced today the design of a confirmatory Phase 3 clinical trial of ataluren in patients with nmDMD that would support conditional approval and is expected to be initiated in the first quarter of 2013. The primary objective of the multicenter, randomized, double-blind, placebo-controlled Phase 3 study is to confirm the ability of ataluren to slow disease progression as assessed by ambulatory ability based on the 6MWD in patients with nmDMD. Secondary endpoints related to physical function and quality of life will also be assessed. Approximately 220 patients are expected to be enrolled in this global study. Further details concerning the study, including enrollment criteria, will be made available on www.clinicaltrials.gov.

"The EMA's validation of our MAA for ataluren for review is a significant milestone for PTC and for the patients, families and professionals who have contributed to this achievement," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "There is a clear and urgent need for new options for patients living with nmDMD, and we are committed to continuing our efforts to deliver ataluren to all patients who may benefit from this approach."'/>"/>

SOURCE PTC Therapeutics, Inc.
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