FDA Delays Sarepta’s PDUFA Date of DMD Candidate

The FDA has delayed its decision on Sarepta Therapeutics’ Duchenne muscular dystrophy candidate eteplirsen, citing the company’s submission of four-year efficacy data as a major amendment to the NDA.

Sarepta included additional six minute walk test and loss of ambulation data, the company said. The candidate’s PDUFA date has been pushed to May 26.

Eteplirsen previously was scheduled to be reviewed Jan. 22 by the Peripheral and Central Nervous System Advisory Committee, but the meeting was cancelled due to a severe snowstorm. A makeup date has not yet been set.

In the meeting’s briefing documents, the FDA said it wanted more evidence of the candidate’s efficacy, as the sponsor’s data were potentially skewed. The FDA 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.