Givinostat hydrochloride monohydrateProduct ingredient for Givinostat

Givinostat is a small molecule histone deacetylase (HDAC) inhibitor. It has been investigated as a treatment for a variety of inflammatory diseases, like Crohn's disease and juvenile idiopathic arthritis, cancers like leukemia and lymphoma, as well as several muscular dystrophies. In the context of muscular dystrophy, inhibitors of HDAC appear to exert their therapeutic effects by targeting pathogenic processes that cause inflammation and muscle loss.^4,1

Givinostat was granted FDA approval in March 2024 for the treatment of patients ≥6 years of age with Duchenne muscular dystrophy (DMD).^3,4 It is the first non-steroidal drug approved to treat patients with all genetic variants of DMD.⁴