Givinostat, a histone deacetylase (HDAC) inhibitor that acts on muscle fibrosis and inflammation in Duchenne muscular dystrophy, should be authorised in Europe very soon, under the name Duvysat™ (it has been authorised in the United States since March 2024).
The application submitted to the EMA by the Italfarmaco laboratory was examined by the Committee for Medicinal Products for Human Use (CHMP), which came out on 25 April:
- in favour of a conditional marketing authorisation ;
- for patients with DMD, aged 6 and over, ambulant, on corticosteroid therapy;
- with the need to produce further efficacy results on ambulant patients. In the EPIDYS trial, the group of patients taking givinostat performed significantly better in the 4-step test (in an average of 1.25 seconds 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.
The conditional authorisation decision still has to be approved by the European Commission before it becomes effective, but givinostat has already been available for prescription in France since February 2025 under a compassionate access scheme.
The Filnemus neuromuscular rare diseases health network has posted details of this derogation on its website, along with details of the need to collate follow-up data on the BaMaRa platform (in French).
New treatment against Duchenne muscular dystrophy. European medecines agency (EMA) 25 avril 2025
Duvyzat : Overview. European medecines agency (EMA)
