Dyne Therapeutics receives orphan drug designation in Japan for DYNE-251 in DMD

Dyne Therapeutics (DYN) announced that the Ministry of Health, Labour and Welfare in Japan has granted Orphan Drug designation for DYNE-251 in individuals with Duchenne muscular dystrophy 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.” DYNE-251 has also been granted Breakthrough Therapy designation, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, and Orphan Drug designation from the FDA and European Medicines Agency for the treatment of individuals with DMD, amenable to exon 51 skipping.