Sarepta Discontinues Development of SRP-5051 for Duchenne Muscular Dystrophy Due to Safety Concerns

Discontinuation of SRP-5051 Development:
Sarepta Therapeutics has discontinued the development of SRP-5051 (vesleteplirsen), a next-generation treatment for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping, due to safety concerns.

Safety Concerns:
The primary safety concern was hypomagnesemia (low blood levels of magnesium), which was seen early in the study and was considered manageable but persisted in a subset of patients even after discontinuation of the drug. Additionally, a decline in kidney function was observed in some patients.

FDA Feedback:
The decision to discontinue the development was influenced by feedback from the FDA and the evolving therapeutic landscape for DMD.

Clinical Trial Impact:
The global, Phase 2, multi-arm, ascending dose MOMENTUM study has been discontinued as part of this decision.

Community Response:
The Parent Project Muscular Dystrophy (PPMD) expressed disappointment but remains hopeful that the data from this trial will inform other potential treatments for DMD.