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Italfarmaco

Italfarmaco: Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy

March 22, 2024

In response to Defeat Duchenne Canada’s request for a comment on how this impacts the Canadian Duchenne Community, the Italfarmaco Group issued the following statement: Italfarmaco is working hard to ensure that givinostat is made available to appropriate patients, around the world, as quickly as possible. Italfarmaco is present in Canada for their clinical trials and has selected four clinical sites (soon to be recruiting) for a trial in non-ambulant boys 9-<18 years old (NCT05933057). The recent approval in the US is a big achievement for the company and the approval would not have been possible without the bravery and dedication of the patient community. Italfarmaco assures us that they will not stop there. The company has a global presence and has initiated or plans to initiate discussions with regulatory agencies in other geographies. Italfarmaco knows the urgency of addressing the needs and the daily challenges of people living with DMD and their families and will work with these regulatory authorities as quickly as they can.


Highlights:

  • Orally administered treatment for DMD approved in patients 6 years and older
  • EPIDYS trial met primary endpoint demonstrating statistically and clinically meaningful treatment benefit in one of the largest DMD phase 3 trials to date
  • Median follow-up of over 3 additional years in open label long-term safety and tolerability study
  • Italfarmaco also announces establishment of new U.S.-based subsidiary ITF Therapeutics to expand company focus on rare diseases and lead commercial launch for Duvyzat™

Defeat Duchenne Canada is pleased to announce 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.”

“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.”

Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group

“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.”

Dr Francesco De Santis, President of Italfarmaco Holding and Chairman of Italfarmaco Group

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.

“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.”

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

“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.”

Pat Furlong, Founding President & CEO at Parent Project Muscular Dystrophy (PPMD)

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. Italfarmaco conducted clinical trials at sites in Canada and is working closely with healthcare providers and patient advocacy groups, including Defeat Duchenne Canada, to expand access.

About Duvyzat™

Duvyzat is an investigational drug discovered through Italfarmaco’s research and development efforts in collaboration with Telethon and Duchenne Parent Project (Italy). Duvyzat is a histone deacetylase (HDAC) inhibitor that modulates the deregulated activity of HDACs in the dystrophic muscle, which is a major consequence of the lack of dystrophin associated with DMD. Duvyzat’s mechanism of action has the potential to inhibit HDAC pathological overactivity in an effort to address the cascade of events leading to muscle damage, thereby counteracting the disease pathology and slowing down muscle deterioration.

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