Sarepta falls after FDA data request
Sarepta Therapeutics Inc. (NASDAQ:SRPT) fell $5.25 (13%) to $33.99 on Tuesday after FDA requested additional information from the company about the existing data set for eteplirsen to "inform a decision" on whether the data could support accelerated approval for Duchenne muscular dystrophy (DMD). Sarepta said FDA made the request at a March end-of-Phase II meeting to discuss a potential NDA for the phosphorodiamidate morpholino oligomer (PMO) that induces exon 51 skipping on the dystrophin gene.
According to Sarepta, FDA requested a "comprehensive summary to support dystrophin as a surrogate" endpoint that would reasonably predict clinical benefit in DMD patients, as well as "a detailed discussion of all clinical outcomes in the eteplirsen study." Sarepta said the agency has not made a final decision on whether it will accept an NDA for accelerated approval of eteplirsen. Sarepta added that it still plans to start a confirmatory trial for the compound in the indication in 1Q14. ...