Dyne Therapeutics Secures Orphan Drug Designation in Japan for DYNE-251 in Duchenne Muscular Dystrophy
Dyne Therapeutics has received Orphan Drug designation in Japan for its investigational therapy DYNE-251, aimed at treating Duchenne muscular dystrophy (DMD) patients with specific mutations. This designation offers benefits such as development cost subsidies and potential market exclusivity for up to 10 years. DYNE-251 is currently in the Phase 1/2 DELIVER trial, showing sustained functional improvement. The therapy also holds multiple designations from the U.S. FDA and EMA. Results from the trial's expansion cohort are anticipated in late 2025.
Dyne Therapeutics Receives Orphan Drug Designation in Japan for Duchenne Muscular Dystrophy Therapy WALTHAM, Mass. (Sept. 29, 2025) - Dyne Therapeutics Inc. (Nasdaq: DYN) announced today that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation to DYNE-251, its investigational therapy for Duchenne muscular dystrophy (DMD) patients with mutations amenable to exon 51 skipping. The designation, which is reserved for treatments targeting rare diseases affecting fewer than 50,000 patients in Japan, provides benefits including development cost subsidies and potential market exclusivity for up to 10 years upon approval. DYNE-251 is currently being evaluated in the global Phase 1⁄2 DELIVER trial, where it has shown sustained functional improvement through 18 months of treatment. This designation in Japan adds to the therapy’s existing Orphan Drug, Breakthrough Therapy, Fast Track, and Rare Pediatric Disease statuses from the U.S. FDA and Orphan Drug designation from the European Medicines Agency (EMA). The grant was awarded exclusively to Dyne Therapeutics. Results from the registrational expansion cohort of the DELIVER trial are expected in late 2025. Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Dyne Therapeutics Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: GNW9536682-en) on September 29, 2025, and is solely responsible for the information contained therein. © Copyright 2025 - Public Technologies (PUBT)
