DUVYZAT (givinostat) is a histone deacetylase inhibitor approved for treating Duchenne muscular dystrophy (DMD) in patients aged 6 years and older. It modulates gene expression, potentially slowing disease progression by reducing muscle degeneration. The drug is administered orally as a weight-based suspension twice daily with food. Key safety considerations include monitoring for thrombocytopenia, hypertriglyceridemia, and QTc prolongation.
Clinical trials demonstrated efficacy in delaying functional decline, with a 1.78-second improvement in 4-stair climb time versus placebo over 18 months. Common adverse reactions include diarrhea, abdominal pain, and hematological changes. DUVYZAT requires rigorous adherence to dosing guidelines and regular laboratory monitoring to mitigate risks.
