Givinostat is approved for marketing in Europe for certain patients with DMD

Givinostat, a histone deacetylase (HDAC) inhibitor that impacts muscle fibrosis and inflammationRéaction non spécifique suscitée par un agent pathogène. in Duchenne muscular dystrophy, is now conditionally approved in Europe under the name Duvysat™. It concerns:

DMD patients, aged 6 and over, ambulant, on corticosteroid therapy;

and requires further efficacy results to be produced on ambulant patients. In the EPIDYS trial, the group of patients taking givinostat performed significantly better in the 4-step test (in 1.25 seconds on average compared with 3.03 seconds on placeboLe placebo est un produit dont la présentation est identique à celle d’un médicament, mais qui ne contient pas de principe actif. Dans un essai clinique, un placebo est utilisé pour mesurer l’action réelle du médicament testé, en comparant les effets du médicament testé contenant le principe actif et ceux du placebo.), while the results for motor function and strength were more positive with givinostat, but there was no significant difference.

In France, givinostat has been available for prescription since February 2025 under a compassionate access scheme.

European Commission ores release, 6 June 2025

New treatment against Duchenne muscular dystrophy

European medecines agency (EMA, 25 April 2025)

 

Duvyzat : Overview

European medecines agency (EMA)