Dyne Therapeutics 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.
Significant Dystrophin Expression
“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.”
Orphan Drug Designation to 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. Learn More: Breakthrough Therapy Designation for DYNE-251
Is my son amenable for exon 51 skipping therapies?: Mutations and Deletions Amenable to Exon 51 Skipping Therapies
About DYNE-251
DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for individuals with DMD who have mutations in the DMD gene that are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to an antigen-binding fragment (Fab) that binds to the transferrin receptor 1 (TfR1). It is designed to enable the production of near full-length dystrophin in muscle and the central nervous system (CNS) to provide functional improvement. DYNE-251 has received Breakthrough Therapy designation, Fast Track and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA), as well as Orphan Drug designation from the FDA and European Medicines Agency (EMA) for the treatment of individuals with DMD, amenable to exon 51 skipping.
In addition to DYNE-251, Dyne is building a DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44.
Learn More: Upcoming Exon 51 Skipping Therapies for the Treatment of Duchenne
