FDA raises doubts on eteplirsen (Sarepta)for Duchenne Muscular Dystrophy

Sarepta Therapeutics,provided an update on its discussions with the FDA regarding its planned New Drug Application (NDA) submission and confirmatory clinical study with eteplirsen for the treatment of Duchenne Muscular Dystrophy (DMD). Citing recent developments since Sarepta's last meeting with the agency, including a failed study with a competitive product (drisapersen) and recent natural history data in DMD, the FDA indicated the new data raise "considerable doubt" about both the dystrophin biomarker and the supportive clinical efficacy assessed on the 6-minute walk test (6MWT) in the Phase IIb clinical study of eteplirsen. As a result of these recent data, the FDA stated that they "currently consider an NDA filing for eteplirsen as premature".