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.
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June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.