Italfarmaco has announced that the European Commission (EC) has granted conditional marketing authorisation for Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor.
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It is approved for the treatment of Duchenne muscular dystrophy (DMD) in ambulant patients 6 years and older, regardless of the underlying genetic mutation, when taken together with corticosteroids. The EC decision follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) on the 25th of April, 2025. The approval applies to all 27 EU member states, as well as Iceland, Liechtenstein, and Norway. Italfarmaco is now working closely with national authorities and distribution partners to facilitate timely access to Duvyzat across the EU.
“People living with DMD in Europe have long awaited new therapeutic options that can alter the course of this devastating disease. Until now, there have been limited approved treatments that address the underlying pathology of DMD across the broad patient population. That changes with the approval of Duvyzat, which slows disease progression and preserves muscle function — regardless of the gene mutation — by targeting disease mechanisms,” said Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco Group. “We are committed to working closely with health authorities and the DMD community to ensure timely access to this important new treatment across Europe.”
The approval is based on the positive results of the EPIDYS Phase 3 multi-center, randomised, double-blind, placebo-controlled trial (NCT02851797). In the EPIDYS study, a total of 179 ambulant boys six years of age or older received either Duvyzat twice daily or placebo, in addition to corticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Duvyzat also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA) and fat infiltration evaluation by magnetic resonance imaging. Specifically, Duvyzat treatment was associated with 40% less decline in cumulative loss of NSAA items, indicating Duvyzat’s potential to delay disease progression in affected individuals. Most adverse effects observed with Duvyzat were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024. Long-term data from the ongoing EPIDYS extension study was compared to natural history cohorts using propensity score matching showing that the median age at loss of ambulation was 18.1 years in the Duvyzat group versus 15.2 years in controls.
The EC has granted Duvyzat conditional marketing authorisation in the EU. The approval makes Duvyzat available to ambulant DMD patients 6 years and older while Italfarmaco conducts additional clinical studies designed to further confirm and characterise its therapeutic benefit. Outside of the EU, Duvyzat was granted approval by the US Food and Drug Administration (FDA) in March 2024 for the treatment of DMD patients 6 years and older. In the UK, Duvyzat received approval by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients 6 years and older who are ambulatory and granted conditional marketing approval for non-ambulatory patients.