Avidity Biosciences: Japan’s MHLW grants ODD to delpacibart etedesiran

Avidity Biosciences (RNA) announced that the Japan Ministry of Health, Labour and Welfare has granted Orphan Drug designation to delpacibart etedesiran for the treatment of myotonic dystrophy type 1, an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies. Del-desiran is the first investigational treatment for DM1 to receive Orphan Drug designation in Japan. Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. FDA and Orphan designation by the European Medicines Agency.