DUVYZAT (givinostat) is a histone deacetylase inhibitor approved for treating Duchenne muscular dystrophy in patients 6 years and older, demonstrating efficacy by mitigating fat infiltration progression within skeletal muscles compared to placebo.

Therapeutic Efficacy of DUVYZAT in DMD Management

DUVYZAT functions as a histone deacetylase inhibitor, though the precise mechanism underlying its therapeutic effect in DMD remains incompletely characterized. Clinical efficacy was demonstrated in Study 1, where magnetic resonance spectroscopy revealed DUVYZAT's capacity to attenuate fat infiltration progression in vastus lateralis muscles. Specifically, patients with baseline fat fractions of >5% to ≤30% exhibited a mean increase of 7.48% over 18 months compared to 10.89% increase in placebo recipients. This reduction in muscle fat infiltration rate represents a critical marker of disease progression modulation in DMD.