Posted by Michael Wonder on 15 Feb 2019
Sarepta announces FDA acceptance of golodirsen (SRP-4053) new drug application for patients with Duchenne muscular dystrophy amenable to skipping exon 53
14 February 2019 - FDA grants priority review status.
Sarepta Therapeutics announced the FDA has accepted its new drug application seeking accelerated approval for golodirsen (SRP-4053) and provided a regulatory action date of 19 August 2019.
Golodirsen is a phosphordiamidate morpholino oligomer engineered to treat those individuals with Duchenne muscular dystrophy (Duchenne) who have genetic mutations subject to skipping exon 53 of the dystrophin gene.
Golodirsen is also being studied in Sarepta’s ongoing ESSENCE study (4045-301), a global, randomized double-blind, placebo-controlled study assessing the safety and efficacy of golodirsen and casimersen, our exon 45 skipping agent. The Division has previously confirmed that the ESSENCE study could possibly serve as a post-marketing confirmatory study.
