FDA grants orphan drug designation for facioscapulohumeral muscular dystrophy treatment
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The FDA has granted orphan drug designation to Avidity Biosciences Inc. for AOC 1020, its novel RNA therapeutic to treat facioscapulohumeral muscular dystrophy.
According to a company press release, AOC 1020 aims to reduce expression of the disease-causing gene, double homeobox 4 (DUX4), in muscle cells of patients with facioscapulohumeral muscular dystrophy (FSHD). The investigational treatment consists of a proprietary monoclonal antibody that binds to transferrin receptor 1 combined with small interfering RNA that targets DUX4 mRNA.
The FDA had granted fast track designation to AOC 1020 in January.
“There are currently no treatment options to address this devastating and debilitating muscular dystrophy disorder,” Steve Hughes, MD, Avidity chief medical officer, said in the release. “We look forward to advancing AOC 1020 and bringing this much-needed therapy to patients.”
AOC 1020 is currently under evaluation in the phase 1/2 FORTITUDE clinical trial, a randomized, placebo-controlled, double-blind study that includes approximately 70 adults with FSHD.
Avidity plans to share data from a preliminary assessment of AOC 1020 in nearly half of study participants during the first half of 2024, the release stated.