Duvyzat approved for DMD

Italfarmaco S.p.A. announced  that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion recommending the granting of a conditional marketing authorisation for Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor. The recommended marketing authorisation is for the treatment of patients with Duchenne muscular dystrophy (DMD) aged six years and older who are able to walk (ambulant), when taken together with corticosteroids. Conditional approval would make Duvyzat available to patients while Italfarmaco continues to generate additional clinical evidence to further strengthen and expand upon the compelling results observed to date. The European Commission (EC) will review the CHMP’s recommendation and is expected to make a final decision in July 2025. Pending authorisation, Italfarmaco will work closely with local authorities to facilitate rapid access, ensuring Duvyzat is made available across the European Union (EU).

“The CHMP’s recommendation is a validation of Duvyzat’s therapeutic potential,” said Dr. Paolo Bettica,  Chief Medical Officer at Italfarmaco Group. “This milestone reflects our unwavering commitment to advancing innovative treatments that can provide life-changing benefits to individuals living with DMD. We are profoundly grateful to the families, caregivers, and patient communities whose engagement and advocacy have been instrumental in reaching this significant achievement.”

“The urgent need for disease-modifying therapies in Duchenne cannot be overstated, and this CHMP recommendation marks a critical step forward. Through years of rigorous research, Duvyzat has consistently demonstrated a favourable risk-benefit profile and the potential to significantly delay disease progression across a broad range of patients. Its unique mechanism of action represents an important addition to the treatment landscape, offering new hope that Duvyzat could become a foundational therapy for those living with Duchenne,” said Prof Eugenio Mercuri,   Professor of Paediatric Neurology at the Catholic University, Rome, Italy.

“Together with the DMD community, we welcome the CHMP’s opinion, which moves us closer to making Duvyzat available to eligible patients living with Duchenne in the EU,” said Dr. Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group at Italfarmaco. “The positive decision supports bringing this novel treatment to patients while we continue to generate additional clinical evidence. Our focus remains clear: to improve outcomes and quality of life for individuals affected by Duchenne.”

The positive CHMP decision is based on the results of the EPIDYS Phase III multicentre, 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 presented at the MDA conference, showing that givinostat may delay disease progression. Using propensity score matching, the median age at loss of ambulation was 18.1 years in the givinostat group versus 15.2 years in controls. 

Duvyzat will be available as an 8.86 mg/ml oral suspension.  Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official EU languages after the marketing authorisation has been granted by the European Commission.

 Citations- Mercuri E, Vilchez JJ, Boespflug-Tanguy O et al. !Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase III trial. Lancet Neurol. 2024, 23:393  DOI:https://doi.org/10.1016/S1474-4422(24)00036-X

Vandenborne, K., McDonald, C., Servais, L. et al. Givinostat in Duchenne muscular dystrophy: Effect on disease milestones [Poster presentation]. MDA Clinical & Scientific Conference, March 2025 Dallas, TX