FDA Sets January 2027 Target Action Date for Z-Rostudirsen in Duchenne Muscular Dystrophy
KEY TAKEAWAYS
- The FDA accepted the Biologics License Application for zeleciment rostudirsen in Duchenne muscular dystrophy amenable to exon 51 skipping.
- The application received Priority Review, with a PDUFA target action date of January 21, 2027.
- The submission seeks Accelerated Approval based on dystrophin production as a surrogate endpoint.
The Food and Drug Administration accepted the biologics license application (BLA) for zeleciment rostudirsen (z-rostudirsen; Dyne Therapeutics, Waltham, MA), an investigational therapy for individuals with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The Agency granted Priority Review and assigned a Prescription Drug User Fee Act target action date of January 21, 2027.
According to a statement released by Dyne Therapeutics, the company is seeking Accelerated Approval for z-rostudirsen based on measures of dystrophin production as a surrogate end point intended to predict clinical benefit. The application is supported by data from the registrational expansion cohort of the global phase 1/2 DELIVER trial (NCT05524883), in which z-rostudirsen, administered once every 4 weeks, met the primary end point. According to the company, treatment was associated with a statistically significant increase in dystrophin production, functional improvement across multiple clinical measures, and a favorable safety profile.
Regulatory and Development Highlights
- Z-rostudirsen is designed for people with DMD mutations amenable to exon 51 skipping.
- The therapy consists of a phosphorodiamidate morpholino oligomer conjugated to an antigen-binding fragment targeting transferrin receptor 1.
- Z-rostudirsen has received FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations.
- The therapy is under investigation in the long-term extension portion of DELIVER and the global confirmatory phase 3 FORZETTO trial (NCT07608432).
Source
Dyne Therapeutics. Dyne Therapeutics announces US FDA acceptance of Biologics License Application (BLA) for z-rostudirsen in exon 51 Duchenne muscular dystrophy (DMD). Dyne Therapeutics. Published July 20, 2026. Accessed August 3, 2026. https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-us-fda-acceptance-biologics-license