Dyne announces MHLW in Japan granted ODD for zeleciment basivarsen

Dyne Therapeutics (DYN) 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.”