Avidity Biosciences, Inc., a biopharmaceutical company pioneering RNA therapeutics, has announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation to delpacibart etedesiran (del-desiran) for the treatment of myotonic dystrophy type 1 (DM1). Del-desiran, an investigational treatment targeting the root cause of DM1, is the first DM1 therapy to receive this designation in Japan. It also holds Breakthrough Therapy, Orphan Drug, and Fast Track designations from the U.S. FDA and Orphan designation from the European Medicines Agency (EMA).

Steve Hughes, M.D., CMO at Avidity, expressed excitement over the MHLW decision, highlighting del-desiran’s potential to address the unmet needs of DM1 patients globally. Data from the MARINA and MARINA-OLE studies show promising safety, tolerability, and clinical improvements. Avidity expects to complete enrollment in the Phase 3 HARBOR™ trial in mid-2025 and submit marketing applications in 2026.

Japan grants Orphan Drug designation to therapies for diseases affecting fewer than 50,000 patients, offering benefits such as prioritized clinical consultation, tax incentives, and priority application reviews. The Phase 3 HARBOR trial, set to enroll 150 DM1 patients, will assess del-desiran's effects on myotonia and other key symptoms.

For more information about the ongoing studies, visit the clinical trial websites for HARBOR and MARINA-OLE.

Source:https://www.aviditybiosciences.com/latest-news