CHMP negative opinion on PTC 124 for Duchenne MD-PTC Therapeutics
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion on the MAA for conditional approval of PTC 124 (ataluren), from PTC Therapeutics, for the treatment of nonsense mutation Duchenne Muscular Dystrophy (nmDMD). The response is consistent with the company's previous guidance concerning the substantial risks regarding conditional EMA approval and underscores the importance of PTC's work in completing the ongoing confirmatory Phase II clinical trial.
In April 2013, PTC initiated a 48-week, 220-patient confirmatory Phase III clinical trial of ataluren for the treatment of nmDMD. The trial is on track to complete enrollment in mid-2014 with top-line data expected in mid-2015. PTC intends to request a re-examination of the CHMP opinion with a final outcome expected in the second quarter of 2014, when the confirmatory study is expected to be more fully enrolled.
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