Avidity Biosciences’ AOC 1020 granted Orphan Drug designation in FSHD

Avidity Biosciences announced that the FDA has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy – FSHD -, a serious, rare, hereditary muscle-weakening condition marked by life-long, progressive loss of muscle function that causes significant pain, fatigue, and disability. AOC 1020 is being evaluated in the Phase 1/2 FORTITUDE clinical trial in adults with FSHD and is the company’s second muscle-targeting small interfering RNA AOC in clinical development. Avidity plans to share data from a preliminary assessment of AOC 1020 in approximately half of study participants from the FORTITUDE trial in the first half of 2024.