For its investigational therapeutic, DYNE-251, Dyne Therapeuticsa clinical-stage company focused on genetically driven neuromuscular diseases, announced it has been granted Orphan Drug designation by the Ministry of Health, Labour and Welfare (MHLW) in Japan. This designation is for the treatment of individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene amenable to exon 51 skipping. a clinical-stage company focused on genetically driven neuromuscular diseases, announced it has been granted Orphan Drug designation by the Ministry of Health, Labour and Welfare (MHLW) in Japan. This designation is for the treatment of individuals with Duchenne muscular dystrophy (DMD) who have mutations in the DMD gene amenable to exon 51 skipping.

This Japanese designation complements existing Orphan Drug status in the U.S. and Europe, as well as Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations from the U.S. FDA, underscoring the high medical need and potential of the therapy.

DYNE-251, which utilizes a Transferrin Receptor 1 (TfR1) targeting approach to enable the production of near full-length dystrophin, is currently being evaluated in the global Phase 1/2 DELIVER clinical trial.

Data from the ongoing trial have already demonstrated unprecedented and sustained functional improvement through 18 months, driven by significant dystrophin expression, according to Dyne's chief medical officer. The company has fully enrolled a registrational expansion cohort in the DELIVER trial to support a potential U.S. Accelerated Approval submission, with results from this cohort, which focuses on the primary endpoint of change in dystrophin protein levels at six months, expected in late 2025. The company is committed to collaborating with Japanese regulators to determine the local regulatory pathway, leveraging the benefits of the Orphan Drug designation in Japan, which include subsidies for development and potential market exclusivity for up to 10 years upon approval.