Avidity Biosciences, Inc. (RNA) Wednesday said the Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead drug candidate for the treatment of myotonic dystrophy type 1 (DM1).
AOC 1001 is designed to treat the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies available now.
The company had reported positive long-term data from the Phase 2 MARINA-OLE study of AOC 1001, showing reversal of disease progression in people with DM1. Avidity plans to initiate Phase 3 HARBOR study for AOC 1001 this quarter.
AOC 1001 has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency for the treatment of DM1.
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