Defeat Duchenne Canada is happy to share that Duvyzat® (givinostat) has received positive results from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) review based on the results of the Phase 3 study.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended conditional authorization for givinostat in ambulant patients aged 6 years and older who are also taking corticosteroids to treat Duchenne muscular dystrophy.
What is Duvyzat® (givinostat)?
Duvyzat is an orally administered histone deacetylase (HDAC) inhibitor. HDACs are enzymes that play an important role in many cellular processes, including muscle regeneration and repair. For people with Duchenne muscular dystrophy (DMD), HDACs are overactive, which breaks muscle down and prevents muscle repair. An HDAC inhibitor such as Duvyzat can be used to reduce the HDAC activity, restoring gene expression and muscle repair.
In the Phase 3 EPIDYS study, 179 ambulant males six years of age or older received either Duvyzat or placebo (a pill that looks like the treatment but contains no real medicine) twice daily, in addition to corticosteroid treatment. The EPIDYS study met its primary endpoint (or goal of treatment), demonstrating a significant difference in time to complete the four-stair climb assessment for those children who received treatment compared to those who received the placebo. Duvyzat also showed 40% less decline in North Star Ambulatory Assessment (NSAA; this is a 17-item assessment that is used to measure motor abilities often used to measure DMD disease progression or the impact of treatment) and fat infiltration into the muscle, which was evaluated by magnetic resonance imaging (MRI; a non-invasive procedure that creates an image of most internal body parts). Most adverse effects or side effects were mild to moderate.
Duvyzat was approved by the US Food and Drug Administration (FDA) in March 2024 and the UK Medicines and Healthcare Products Regulatory Agency (MHRA) in December 2024. Full approval will still need to be confirmed by the European Commission (EC). Clinical trials in Canada are ongoing at several sites.