The Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for golodirsen (SRP-4053; Sarepta), a treatment designed for patients with Duchenne muscular dystrophy (DMD) who have genetic mutations that are amenable to exon 53 skipping.

Using Sarepta’s proprietary exon-skipping technology, golodirsen, a phosphorodiamidate morpholino oligomer (PMO), works by binding to exon 53 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing. Exon skipping allows for production of an internally truncated but functional dystrophin protein. 

The NDA submission includes data from the Phase 1/2 4053-101 clinical trial. A total of 25 boys, with confirmed deletions of the dystrophin gene amenable to exon 53 skipping, were enrolled in the trial. The primary outcomes were incidence of adverse events (AEs) and serious AEs; change from baseline in total distance walked in the 6-Minute Walk Test (6MWT); and change from baseline in dystrophin protein levels. In a press release, the Company stated that results from the study were statistically significant in favor of golodirsen on all endpoints.

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The FDA previously granted Orphan Drug designation to golodirsen and has set a Prescription Drug User Fee Act date of August 19, 2019 for the application. “If approved, golodirsen will serve up to another 8% of the Duchenne community, bringing us closer to helping as many Duchenne patients as possible,” said Doug Ingram, president and chief executive officer, Sarepta. 

Golodirsen is also being evaluated in the ESSENCE study (4045-301), a global, randomized double-blind, placebo-controlled trial assessing the safety and efficacy of golodirsen and casimersen (an exon 45 skipping agent). 

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