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Italfarmaco: Results from Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology

March 20, 2024
  • Trial met primary endpoint: givinostat demonstrated statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date
  • Givinostat was well tolerated with a well-documented safety profile consistent with previous givinostat trials
  • Italfarmaco anticipates U.S. Food and Drug Administration (FDA) PDUFA response scheduled for March 21, 2024

MILAN – The full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology. The EPIDYS clinical trial met its primary endpoint: change in four-stair climb assessment from baseline to 72 weeks, which demonstrated the potential of givinostat to delay disease progression when added to corticosteroid treatment. Givinostat-treated boys also showed favorable outcomes on key secondary endpoints assessed in the study.

“When managing DMD, a primary goal is to maintain motor function for as long as possible. The results from EPIDYS provide robust evidence that givinostat has the potential to support this goal.

“In addition to the primary endpoint, analysis of the North Star Ambulatory Assessment (NSAA), which measures motor function skills based on a 17-item rating scale, showed that treatment with givinostat resulted in 40% less decline compared to the control group. Taken together, these data suggest givinostat could be an effective new treatment for DMD management.”

Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health.

“The results of the phase 3 EPIDYS study are encouraging and highlight the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”

“We want to thank all the individuals and their families who participated in this international study and would also like to acknowledge the tireless efforts of the clinical investigators as well as the unwavering support of the DMD community.”

Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group.
Overview of Phase 3 EPIDYS Clinical Trial

EPIDYS was a multicentre, randomised, double-blind, placebo-controlled phase 3 trial which enrolled 179 ambulant boys 6 years of age or older with DMD across 3 clinical sites in Canada, as well as sites in the United States and Europe (NCT02851797). Participants were randomised 2:1 to receive oral givinostat or matching placebo twice daily along with their corticosteroid treatment regimen. Participants attended study site visits every 12 weeks for 72 weeks and were evaluated for all endpoints within the study protocol.

Givinostat received priority review, orphan drug and rare pediatric disease designations from the FDA and orphan drug designation from the European Commission. A New Drug Application (NDA) was filed with the U.S. FDA and the Prescription Drug User Free Act (PDUFA) date for givinostat is March 21, 2024. A Marketing Authorisation Application (MAA) for givinostat has been submitted to the European Medicines Agency (EMA) and is currently under review.

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