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FDA Approves Duvyzat

FDA Approves Duvyzat (givinostat) for Duchenne Muscular Dystrophy

MILAN--(BUSINESS WIRE)-- Mar 22, 2024 -- Italfarmaco S.p.A. announced today that the U.S. Food and Drug Administration (FDA) has approved Duvyzat™ (givinostat), a novel histone deacetylase (HDAC) inhibitor, for the treatment of patients 6 years or older with Duchenne muscular dystrophy (DMD), a rare X-linked progressive and life-limiting neuromuscular condition with symptoms from early childhood.

“The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development program, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD,” said Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group. “We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval. Our focus now is to make Duvyzat available as a treatment for DMD management in the U.S. as quickly as possible.”

Dr Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group added, “Duchenne muscular dystrophy is a disease with significant unmet medical need and Duvyzat has the potential to benefit a broad DMD patient population independent of the underlying gene mutation that causes the disease. The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”

The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase 3 EPIDYS 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 glucocorticosteroid treatment. The EPIDYS study met its primary endpoint demonstrating that patients on Duvyzat showed 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. The majority of adverse effects observed with Duvyzat were mild to moderate in severity. Results from this study were published in The Lancet Neurology in March 2024.

“There is a tremendous unmet need for novel therapies in DMD that can achieve meaningful benefits for a broad range of patients. Duvyzat’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,” added Craig M. McDonald, MD, Professor at the Department of Pediatrics and Physical Medicine Rehabilitation at the University of California Davis Health and investigator for the EPIDYS trial. “I would like to thank all patients and their families for participating in the clinical trials and for making this approval possible.”

“We are thrilled with the FDA’s approval of Duvyzat, a new therapy for DMD. It is an oral medication that will be available to every person 6 years and older with DMD. This brings great hope for the Duchenne community, and we believe this will be a key therapy to prevent disease progression in Duchenne,” said Pat Furlong, Founding President & CEO at Parent Project Muscular Dystropy (PPMD).

Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC. ITF Therapeutics will be responsible for the commercialisation of Duvyzat in the U.S. and the company is working closely with healthcare providers, patient advocacy groups and payors to make Duvyzat available to patients.

Duvyzat received priority review, orphan drug and rare pediatric disease designations from the FDA. A Marketing Authorisation Application (MAA) for givinostat as a potential treatment for DMD has been submitted to the European Medicine Agency (EMA) and is currently under review. Italfarmaco has a global presence and is also working with other regulatory agencies.

About Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe neuromuscular genetic disease characterised by progressive muscle weakness and degeneration and is the most common type of muscular dystrophy globally. DMD is caused by mutations in the dystrophin gene that result in the absence of a functional dystrophin protein. Without dystrophin, muscle fibres are highly susceptible to injury and this continuous muscle injury leads to chronic inflammation, impairment of muscle regeneration and muscle replacement by fibrotic and fat tissue. The disease primarily affects boys, with symptoms usually first seen between two and five years of age. Symptoms worsen over time affecting the ability to walk. Eventually, heart and respiratory muscles are affected, which are the two main causes of premature death. DMD incidence is approximately one in every 3500 - 6000 male births worldwide.

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