FX.co ★ Avidity Biosciences: FDA Removes Partial Clinical Hold On Del-desiran - Quick Facts

Avidity Biosciences (RNA) has announced that the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on delpacibart etedesiran. This investigational drug targets the underlying cause of myotonic dystrophy type 1, and is currently under evaluation in the Phase 3 HARBOR trial involving patients with this condition. Known as AOC 1001, del-desiran represents Avidity's leading product candidate developed through its AOC platform. It is engineered to reduce the levels of the disease-associated mRNA, DMPK, which is linked to dystrophy type 1. Del-desiran has been granted Breakthrough Therapy, Orphan Drug, and Fast Track designations by the FDA, along with Orphan designation from the European Medicines Agency.