Dyne Therapeutics Receives Orphan Drug Designation in Japan
Dyne Therapeutics announced that the Ministry of Health, Labour and Welfare, MHLW, in Japan has granted Orphan Drug designation, ODD, for zeleciment basivarsen, for the treatment of myotonic muscular dystrophy type 1, DM1. Z-basivarsen is currently being evaluated in the Phase 1/2 ACHIEVE clinical trial in individuals with DM1. "By targeting the underlying biology of DM1, z-basivarsen has shown early and sustained improvements in myotonia, muscle strength and function, with a favorable safety profile," said Doug Kerr, M.D., Ph.D., chief medical officer of Dyne. "This designation in Japan, alongside those already granted in the U.S. and Europe, emphasizes the urgent need for new therapies and highlights the potential of z-basivarsen to deliver meaningful functional improvement for people living with DM1."
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Dyne Therapeutics Receives Orphan Drug Designation for z-basivarsen, ACHIEVE Trial Shows Sustained Improvement
- Clinical Trial Progress: Z-basivarsen demonstrates sustained functional improvement in the ACHIEVE trial, particularly in muscle strength and function, with enrollment for the registrational expansion cohort expected to complete in early Q2 2026, further advancing treatment options.
- Orphan Drug Designation: The MHLW in Japan has granted z-basivarsen orphan drug status for myotonic dystrophy type 1 (DM1), highlighting the urgent need for new therapies and potentially providing up to 10 years of market exclusivity if approved.
- Multiple Designations: Z-basivarsen has received Breakthrough Therapy, Fast Track, and Orphan Drug designations from the FDA, along with Orphan Drug designation from the EMA, indicating its development potential and market recognition on a global scale.
- Treatment Need: DM1 affects approximately 40,000 people in the U.S. and 55,000 in the EU, and the development of z-basivarsen aims to address the significant unmet medical need, carrying important social and economic implications.
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