Avidity's AOC 1020 Gets FDA Orphan Drug Designation For Muscular Dystrophy Treatment

(RTTNews) - Avidity Biosciences Inc. (RNA) said Tuesday that the U.S. Food and Drug Administration has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy or FSHD.

Facioscapulohumeral muscular dystrophy is 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 about half of study participants from the FORTITUDE trial in the first half of 2024.

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