FDA Approves Givinostat as First Nonsteroidal Drug for Duchenne Muscular Dystrophy


The FDA recommends the dosage of givinostat (Duvyzat; Italfarmaco, ITF Therapeutics) should be based on the individual’s body weight and administered twice daily with food.

The FDA has approved givinostat (Duvyzat; Italfarmaco, ITF Therapeutics), an oral medication, for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 6 years and older, according to a press release form the agency. This marks the first approval of a nonsteroidal drug for patients with all genetic variants of DMD.1

U.S. Food and Drug Administration FDA approved vector stamp | Image Credit: Calin - stock.adobe.com

Image Credit: Calin - stock.adobe.com

Givinostat is a histone deacetylase inhibitor targeting pathogenic processes that reduce inflammation and muscle loss, according to the press release. The FDA recommends the dosage of givinostat based on the individual’s body weight and should be administered twice daily with food.1

“DMD denies the opportunity for a healthy life to the children it affects. The FDA is committed to advancing the development of new therapies for DMD,” Emily Freilich, MD, director of the Division of Neurology 1 in the Office of Neuroscience at the FDA’s Center for Drug Evaluation and Research, said in the press release. “This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation.”1

The approval was based on the multicenter, randomized, double-blinded phase 3 EPIDYS (NCT02851797) trial, which included 179 boys aged 6 years and older. Individuals received either givinostat twice daily or the placebo, in addition to glucocorticosteroid treatment, according to a press release from the company.2 The primary endpoint included the change from baseline to month 18, measured by a 4 stair climb for muscle function. The secondary efficacy endpoint was the change from baseline to moth 18 in physical function, assessed by the North Star Ambulatory Assessment, which is commonly used to rate motor functions in boys with DMD who are capable of walking.1

About The EPIDYS Study

Trial Name: Clinical Study to Evaluate the Efficacy and Safety of Givinostat in Ambulant Patients With Duchenne Muscular Dystrophy
ClinicalTrials.gov ID: NCT02851797

Sponsor: Italfarmaco

Completion Date: February 2022

After 18 months of treatment, those taking givinostat demonstrated statistically significant less decline in the time it took to climb 4 stairs compared to the placebo, with the median change from baseline to 18 months being 1.25 seconds compared to 3.03 seconds with the placebo. Further, the FDA reported that patients taking givinostat saw less worsening on the secondary endpoint than those taking the placebo.1

The majority of adverse events were mild to moderate in severity, according to the press release from the company. The most common AEs were diarrhea, abdominal pain, a decrease in platelets, nausea and vomiting, an increase in triglycerides, and a fever.1,2

“There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. [Givinostat’s] unique mechanism of action has shown a positive risk/benefit profile and the ability to delay disease progression, supporting its potential to become a key component of the standard of care for people living with DMD,” Craig M McDonald, MD, professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health, said in the press release.2

Previously, the FDA granted the drug priority review, fast track designation, orphan drug designation, and rare pediatric disease designation, according to the press release.1 The full results of the study were published in The Lancet Neurology.2,3

  1. FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy. News release. FDA. March 21, 2024. Accessed March 22, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-nonsteroidal-treatment-duchenne-muscular-dystrophy
  2. Italfarmaco Receives FDA Approval for Duvyzat (givinostat) in Duchenne Muscular Dystrophy. News release. Italfarmaco. March 21, 2024. Accessed March 22, 2024. https://www.businesswire.com/news/home/20240321735508/en/Italfarmaco-Receives-FDA-Approval-for-Duvyzat%E2%84%A2-givinostat-in-Duchenne-Muscular-Dystrophy
  3. 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 3 trial. Lancet Neurol. 2024;23(4):393-403. doi:10.1016/S1474-4422(24)00036-X
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