Avidity Gets Orphan Drug Status In Japan For Delpacibart Etedesiran In Myotonic Dystrophy Type 1

(RTTNews) - Avidity Biosciences, Inc. (RNA), Tuesday said that the Japan Ministry of Health, Labour and Welfare (MHLW) has granted Orphan Drug designation to delpacibart etedesiran for the treatment of myotonic dystrophy type 1 (DM1).

Myotonic dystrophy type 1 (DM1) is a multisystem genetic disorder characterized by progressive muscle weakness, myotonia (difficulty relaxing muscles), and other symptoms affecting the heart, eyes, and other organs.

Del-desiran has also received Breakthrough Therapy, Orphan Drug and Fast Track designations by the U.S. Food and Drug Administration (FDA) and Orphan designation by the European Medicines Agency (EMA).

Avidity expects to complete participant enrollment in the Phase 3 HARBOR study in mid-2025 and submit marketing applications starting 2026 in the U.S., European Union and Japan.