Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
Dyne Therapeutics submitted a Biologics License Application (BLA) to the FDA for z-rostudirsen, targeting Duchenne muscular dystrophy patients amenable to exon 51 skipping. The application seeks Accelerated Approval based on dystrophin production as a surrogate endpoint, supported by data from the registrational expansion cohort of the Phase 1/2 DELIVER trial. Dyne has requested Priority Review, which would shorten the FDA review period to six months, supporting a potential U.S.
Key figures
- Drug Name
- z-rostudirsen (DYNE-251)
- Phase Of Trial
- Phase 1/2
- Dosing regimen
- 20 mg/kg Q4W
- Expected launch
- Q1 2027
- Target population
- Exon 51 DMD
- Priority review duration
- 6 months
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