Duvyzat (Givinostat) in Duchenne Muscular Dystrophy: Mechanisms, Clinical Impact, and Future Directions

Abstract

Duchenne Muscular Dystrophy (DMD) is a chronic, progressive neuromuscular disorder characterized by muscle degeneration and weakness due to mutations in the dystrophin gene. Although there is no cure, emerging therapies such as histone deacetylase (HDAC) inhibitors offer promising avenues to slow disease progression. Duvyzat (givinostat), an orally active HDAC inhibitor, has recently received FDA approval following results from the Phase 3 EPIDYS trial, which demonstrated a statistically significant improvement in motor function. Patients treated with Duvyzat showed a 1.25-second faster performance on the four-stair climb (4SC) test compared to placebo, and a 1.91-point higher North Star Ambulatory Assessment (NSAA) score over 72 weeks. Duvyzat was also associated with reduced muscle fat infiltration on MRI. This review discusses the mechanism of HDAC inhibition, clinical evidence supporting Duvyzat's efficacy, its safety profile, and implications for the future of DMD treatment. Continued research is essential to explore long-term outcomes and synergistic potential with gene-targeted therapies.