Posted by Friends of FSH Research on Dec 3, 2025
The Food and Drug Administration has granted Orphan Drug Designation to DX5057, the first and only oral small molecule DUX4 inhibitor in development for facioscapulohumeral muscular dystrophy (FSHD). This designation will help speed this first in-kind treatment for FSHD through the regulatory approval process.
DX5057 directly targets DUX4-driven pathology and has shown strong preclinical activity with a convenient oral route of administration.
Friends of FSH Research supported Altay with seed money in 2021 (The discovery of a novel small molecule inhibitor for DUX4) and 2022 (Determining in vitro and in vivo efficacy of novel DUX4 inhibitors), enabling it to develop this drug and attract further funding to develop it further and reach this significant milestone.
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