Altay Therapeutics is pleased to announce that the FDA has granted Orphan Drug Designation to DX5057, the first and only oral small molecule DUX4 inhibitor in development for facioscapulohumeral muscular dystrophy (FSHD).

FSHD affects ~40,000 people in the U.S. and leads to progressive muscle weakness, disability and loss of independence, yet no therapies are currently approved.

DX5057 directly targets DUX4-driven pathology and has shown strong preclinical activity with a convenient oral route of administration. We’re excited to continue advancing this important therapy toward the clinic.

https://www.linkedin.com/posts/fshd-fshdcommunity-musculardystrophy-ugcPost-7397110130716291072-cush