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Avidity granted FDA Fast Track designation for AOC 1020
The Fly

Avidity granted FDA Fast Track designation for AOC 1020

Avidity Biosciences announced that the FDA has granted Fast Track designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy, or FSHD. FSHD 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 studied in the Phase 1/2 FORTITUDE clinical trial in adults with FSHD and is the company’s second muscle-targeting small interfering RNA, or siRNA, 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. Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Currently, there are no FDA-approved treatments for people living with FSHD. Avidity has three distinct rare disease programs in the clinic. In addition to AOC 1020, the company is also evaluating AOC 1001 in the Phase 1/2 MARINA and MARINA open-label extension clinical trials for the treatment of myotonic dystrophy type 1 and AOC 1044 in the Phase 1/2 EXPLORE44 trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping.

Published first on TheFly

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