FDA asks Sarepta panel for efficacy vote

FDA will ask an advisory committee to vote on five questions at an April 25 meeting to discuss an NDA from Sarepta Therapeutics Inc. (NASDAQ:SRPT) for eteplirsen ( AVI-4658). On Friday, FDA added the questions to discussion topics previously proposed to its Peripheral and Central Nervous System Drugs Advisory Committee in briefing documents released ahead of the meeting (see BioCentury Extra, April 21).

Eteplirsen is under review to treat Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Sarepta is building its case primarily on efficacy data from 12 patients treated in a placebo-controlled Phase IIb trial (Study 201) and its open-label extension phase (Study 202) compared to a historical control group of 13 untreated patients. ...