Givinostat, a drug developed by Italfarmaco for Duchenne and Becker muscular dystrophies, is a histone deacetylase (HDAC) inhibitor.
On 21 March 2024, it was granted marketing authorisation in the United States by the FDA on the basis of the results of the EPIDYS phase III double-blind, placebo-controlled trial, which took place in France.
The EPIDYS trial involved 179 boys with DMD, aged between 6 and 17 (mean age 9.8), who were walking and being treated with corticosteroids. They received either givinostat or a placebo oral suspension twice daily for one and a half years. Analysis of the data from 81 participants treated with givinostat and 39 with placebo, with between 5 and 30% fat fraction (VLFF) in the thigh muscle showed :
- a slower decline in the speed required to climb 4 steps in the treated group than in the placebo group. At the end of the treatment, the former took on average 1.78 seconds less, a significant difference ;
- a significant difference between the NSAA scores of the two groups, in favour of the treated group, as in the 6-minute walk test;
- muscle composition less rich in fat cells in the treated group than in the untreated group;
- relatively good tolerance of givinostat, despite frequent side effects (vomiting, diarrhoea, headaches, nasopharyngitis, abdominal pain, etc.).
The evaluation of givinostat is continuing in an open trial, which is taking place in France. A marketing authorisation application has also been submitted to the EMA.
Italfarmaco Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy. Italfarmaco press release, 21 March 2024.
