FDA panel doubts Sarepta has shown eteplirsen's efficacy

Members of FDA's Peripheral and Central Nervous System Drugs Advisory Committee voted 7-3 with three abstentions that clinical results provided by Sarepta Therapeutics Inc. (NASDAQ:SRPT) did not provide substantial evidence that eteplirsen ( AVI-4658) is effective to treat Duchenne muscular dystrophy (DMD).

Eteplirsen is under review to treat DMD amenable to exon 51 skipping. Sarepta provided data from 12 patients treated in a placebo-controlled Phase IIb trial (Study 201) and its open-label extension phase (Study 202) that was compared to a historical control group of 13 untreated patients. In the last of five voting questions, panelists were asked whether those data showed eteplirsen's efficacy. ...