PDUFA date for FDA review of eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) extended to 26 May 2016- Sarepta

Sarepta Therapeutics, Inc. announced that the FDA will require additional time to complete its review of the New Drug Application (NDA) for eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. In a notice received from the FDA, the Prescription Drug User Fee Act (PDUFA) date for eteplirsen has been extended to May 26, 2016. The rescheduled date for the Peripheral and Central Nervous System Advisory Committee meeting has not yet been determined.

The FDA notified Sarepta that its January 8, 2016 submission of 4-year clinical effectiveness data, which included additional six minute walk test (6MWT) and loss of ambulation data compared to a historical control, has been designated as a major amendment to the NDA. The FDA stated that the PDUFA goal date has been extended by three months to allow for a full review of the submission. As described in the Sarepta Advisory Committee Briefing Document Addendum, the principal basis for establishing the effectiveness of eteplirsen is a comparison of patients in Study 201/202 to a historical control group.

Comment: The FDA has issued a similar response to BioMarin for its DMD candidate drisapersen, with a complete response letter calling for more evidence of the therapy’s benefits.