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Dyne Therapeutics Receives Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy
- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Data from ongoing DELIVER trial demonstrated sustained functional improvement through 18 months; results from Registrational Expansion Cohort expected late 2025 - WALTHAM, Mass., Sept. 29, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company, today announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 is being evaluated in the ongoing Phase 1/2 DELIVER global clinical trial. “In the DELIVER trial, we’ve seen unprecedented and sustained functional improvement, driven by significant dystrophin expression, underscoring the potential of DYNE-251 to transform the care of people living with Duchenne muscular dystrophy,” said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. “With orphan drug designation now granted in Japan, complementing existing designations in the U.S. and Europe, DYNE-251 continues to advance as a promising next-generation exon 51 skipping therapy. Individuals with mutations amenable to exon 51 skipping are among the most prevalent and functionally impacted in the DMD population. Potentially addressing this unmet need further fuels our commitment to develop a therapy that delivers functional improvement for the Duchenne community, and we look forward to collaborating with the Japanese regulators to determine the regulatory pathway for DYNE-251 in Japan.” In Japan, Orphan Drug designation is granted to drugs intended for the treatment of rare diseases affecting fewer than 50,000 patients in the country and for which there is a high medical need. Benefits include subsidies for development costs and potential market exclusivity for up to 10 years if approved. DYNE-251 has also been granted Breakthrough Therapy designation, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), and Orphan Drug designation from the FDA and European Medicines Agency (EMA) for the treatment of individuals with DMD, amenable to exon 51 skipping. About the DELIVER Trial DELIVER is a global, randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy (as measured by both biomarker and functio...