Sarepta's golodirsen under FDA Priority Review

Sarepta Therapeutics Inc. (NASDAQ:SRPT) said Feb. 14 that FDA accepted and granted Priority Review to its NDA for golodirsen (SRP-4053) to treat Duchenne muscular dystrophy amenable to skipping exon 53. its PDUFA date is Aug. 19.

Sarepta is seeking golodirsen's accelerated approval based on data from the Phase I/II 4053-101 study, in which the therapy significantly improved exon skipping, dystrophin expression and dystrophin intensity levels. The company expects that the Phase III ESSENCE study of golodirsen and casimersen (SRP-4045), which began in 2016, may serve as a confirmatory trial; data are due in 2022. Golodirsen has Orphan Drug designation from FDA to treat DMD...