FDA accepts Dyne Therapeutics BLA for DMD drug z-rostudirsen

Dyne Therapeutics, Inc. (Nasdaq: DYN) announced that the U.S. Food and Drug Administration has accepted for review a Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.

The FDA granted Priority Review for the application and set a Prescription Drug User Fee Act (PDUFA) target action date of January 21, 2027. The BLA was submitted for Accelerated Approval based on dystrophin as a surrogate endpoint.

The application is based on data from the registrational expansion cohort of Dyne's DELIVER clinical trial, in which z-rostudirsen administered once every four weeks produced an increase in dystrophin production and functional improvements across multiple clinical endpoints.

Z-rostudirsen has received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations from the FDA, as well as Orphan Drug designation from the FDA, the European Medicines Agency, and Japan's Ministry of Health, Labour and Welfare.

John Cox, president and chief executive officer of Dyne, said: "With a potential approval in six months, we are continuing our launch preparations with the aim of making z-rostudirsen available as quickly as possible."

Dyne stated it continues to expect a potential U.S. launch of z-rostudirsen in Q1 2027, assuming approval is received on the anticipated timeline. The drug candidate continues to be evaluated in the long-term extension portion of the DELIVER trial and in the global confirmatory Phase 3 FORZETTO clinical trial.

DMD is a rare X-linked progressive neuromuscular disorder affecting approximately 12,000 individuals in the U.S. and 16,000 in the EU. Dyne is also advancing four additional development candidates targeting other DMD exon mutations.

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