Givinostat, a histone deacetylase (HDAC) inhibitor that impacts muscle fibrosis and inflammation 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 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)
European medecines agency (EMA)
