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Dyne Therapeutics Announces U.S. FDA Acceptance of Biologics License Application (BLA) for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)

DYN
  • FDA accepted the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
  • Priority Review granted with a Prescription Drug User Fee Act (PDUFA) target action date set for January 21, 2027.
  • Submission for Accelerated Approval is based on dystrophin as a surrogate endpoint.
  • Registrational expansion cohort data from the DELIVER trial showed statistically significant increases in dystrophin production & functional improvement with a favorable safety profile.
  • Dyne Therapeutics expects a potential U.S. launch in Q1 2027 if approval is received on schedule.
  • Z-rostudirsen is a PMO conjugated to an antigen-binding fragment targeting the transferrin receptor 1 to produce near-full length dystrophin in muscle & central nervous system tissues.
  • The therapy holds Breakthrough Therapy, Fast Track, Rare Pediatric Disease, & Orphan Drug designations from U.S., European, & Japanese regulators.
  • Dyne is advancing four additional development candidates (DYNE-253, DYNE-245, DYNE-244, DYNE-255) for other DMD exons.
  • Z-rostudirsen continues evaluation in the long-term extension of the DELIVER trial & the global Phase 3 FORZETTO clinical trial.