Dyne Therapeutics (NASDAQ:DYN) secured a regulatory win in Asia as Japanese authorities granted orphan drug status to its lead candidate for myotonic dystrophy, providing the biotech with a clear path toward market exclusivity in the world’s third-largest pharmaceutical market.
The Japan Ministry of Health, Labour and Welfare bestowed the Orphan Drug designation on zeleciment basivarsen (also known as z-basivarsen or DYNE-101) on Tuesday.
The therapy is designed to treat myotonic dystrophy type 1 (DM1), a rare and progressive genetic disease that currently has no approved disease-modifying treatments.
The designation provides Dyne with development subsidies, tax credits, and up to 10 years of market exclusivity in Japan upon approval.
The regulatory boost comes as Dyne pushes its Phase 1/2 ACHIEVE trial toward critical milestones.
The company announced it remains on track to complete enrollment in the trial's registrational expansion cohort in early Q2 2026.
Early data from the study has already demonstrated sustained functional improvements in muscle strength and myotonia—the inability to relax muscles after use—with a favorable safety profile.
The Japanese designation follows similar "Breakthrough" and "Orphan" statuses in the U.S. and Europe, highlighting global interest in Dyne’s FORCE platform, which aims to deliver genetic medicine directly to muscle tissue.