ITF Therapeutics affiliate of Italfarmaco, today announced publication of positive long-term safety and efficacy data for Duvyzat (Givinostat) as a treatment for Duchenne muscular dystrophy (DMD) from the company’s open-label extensions of its Phase 2 and Phase 3 (EPIDYS) trials.
The results, published in Annals of Clinical and Translational Neurology, show that long-term treatment with givinostat, a novel histone deacetylase (HDAC) inhibitor, in combination with corticosteroids, delayed disease progression in ambulant patients aged six years and older with DMD.
Learn More: What is Duvyzat (Givinostat)?
Importantly, clinical benefit was observed across treatment groups receiving givinostat, with positive impact on key mobility functions such as rising from the floor, climbing stairs, and the ability to walk (ambulation). Givinostat has received marketing authorization in the U.S., E.U. and U.K.
- New data published in Annals of Clinical and Translational Neurology show that long-term treatment with givinostat can significantly delay loss of key mobility functions in patients with Duchenne muscular dystrophy.
- Meaningful clinical benefit was observed across treatment groups, regardless of disease stage at initiation.
- Givinostat remained well-tolerated during extended use, consistent with previous clinical trials.
- Average follow-up in the extension study exceeded 36 treatment months, with some patients receiving treatment for over eight years since initiating givinostat treatment.
The study included multiple cohorts of boys with DMD all of whom received givinostat over various durations. Some had previously taken part in the Phase 2 or Phase 3 EPIDYS studies – either on active treatment or placebo – before entering the extension study. Others enrolled directly into the extension study after meeting eligibility criteria while not participating in the main trial. The total treatment exposure for some patients exceeded eight years.
