USFDAJul 20, 2026, 07:32 AM
Dyne Therapeutics z-rostudirsen BLA Granted Priority Review by FDA
AI Summary
Dyne Therapeutics announced that the FDA has accepted for review its Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The FDA granted Priority Review and set a PDUFA target action date of January 21, 2027. The company anticipates a potential U.S. launch in Q1 2027, assuming approval, marking significant progress in its goal to provide functional improvement for DMD patients.
Key Highlights
- FDA granted Priority Review for z-rostudirsen BLA for Duchenne muscular dystrophy.
- PDUFA target action date for z-rostudirsen is set for January 21, 2027.
- Dyne Therapeutics expects a potential U.S. launch of z-rostudirsen in Q1 2027.
- BLA was submitted for Accelerated Approval based on dystrophin as a surrogate endpoint.
- Registrational expansion cohort of DELIVER trial showed robust and statistically significant dystrophin increase.
- Z-rostudirsen has Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations.
Price Impact
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