Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
- Priority Review granted; PDUFA target action date set for January 21, 2027 - - Submission for Accelerated Approval based on dystrophin as a surrogate endpoint - - In the registrational expansion cohort of the DELIVER trial, treatment with z-rostudirsen once every 4 weeks resulted in a robust and statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favorable safety profile(1) - WALTHAM, Mass., July 20, 2026
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