Italfarmaco presented new cardiac data for givinostat, its histone deacetylase (HDAC) inhibitor, at the 2025 European Paediatric Neurology Society (EPNS) Congress. Post-hoc analysis from the Phase 3 EPIDYS trial in boys with Duchenne muscular dystrophy (DMD) indicated a slower decline in cardiac function with givinostat compared to placebo, alongside a clean cardiac safety profile. Eleven givinostat abstracts were accepted for presentation at the congress.
This news is crucial for the DMD community because cardiac complications are a major cause of morbidity and mortality in this patient population. Demonstrating a potential cardioprotective effect of a therapy already approved for DMD management is a significant step towards improving long-term outcomes and quality of life. This data may influence treatment guidelines and encourage earlier intervention with givinostat.
The EPIDYS trial data showed a positive trend in cardiac function preservation in the givinostat group, measured using electrocardiogram and echocardiogram parameters. A separate Phase 1 study confirmed the cardiac safety of givinostat at therapeutic doses, showing no risk of QTc interval prolongation—a critical concern with some cardiac medications. Givinostat received conditional marketing authorization in the EU in June 2025 and has previous approvals in the US and UK.
This positive cardiac data strengthens givinostat’s position as a DMD therapy. It may lead to increased adoption by clinicians and potentially expand its use in earlier stages of the disease. Further research exploring the long-term cardiac benefits of givinostat will be essential to confirm these findings and fully understand its potential impact on DMD-associated cardiomyopathy.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
