Eteplirsen regulatory update

Sarepta said FDA requested additional data for eteplirsen from the open-label, confirmatory, U.S. Phase III PROMOVI trial to treat Duchenne muscular dystrophy (DMD) as part of the agency’s review of eteplirsen’s NDA. Sarepta said it will submit data “over the coming weeks” on dystrophin expression at baseline and 48 weeks from 13 PROMOVI patients. Last month, FDA delayed its decision on the NDA beyond a May 26 PDUFA date. Eteplirsen is under review to treat DMD amenable to exon 51 skipping. ...