FDA approves Duvyzat (givinostat) to treat patients 6 years or older with Duchenne muscular dystrophy-- Italfarmaco S.p.A.

The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase III EPIDYS trial (NCT02851797). In the EPIDYS study, a total of 179 ambulant boys six years of age or older received either Duvyzat twice daily or placebo, in addition to glucocorticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating that patients on Duvyzat showed a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Duvyzat also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), and fat infiltration evaluation by magnetic resonance imaging. The majority of adverse effects observed with Duvyzat were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024.

Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC. ITF Therapeutics will be responsible for the commercialisation of Duvyzat in the U.S. and the company is working closely with healthcare providers, patient advocacy groups and payors to make Duvyzat available to patients.

See- VOLUME 23, ISSUE 4, P393-403, APRIL 2024. "Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase III trial."- Prof Eugenio Mercuri, MD Prof Juan J Vilchez, MD, Prof Odile Boespflug-Tanguy, MD, Craig M Zaidman, MD, Prof Jean K Mah, MD, Nathalie Goemans, MD et al. Published: April,2024DOI:https://doi.org/10.1016/S1474-4422(24)00036-X. The Lancet Neurology.