The FDA approved Duvyzat (givinostat), a novel histone deacetylase (HDAC), for the treatment of patients six years of age and older with Duchenne muscular dystrophy (DMD). This approval marks an important addition to the portfolio of therapeutic options available to patients with DMD, which is the most common type of muscular dystrophy globally.

The announcement was made by ITF Therapeutics, a new U.S. subsidiary of Italfarmaco, an Italian company founded in 1938. ITF Therapeutics will be responsible for the commercialization of Duvyzat in the United States and is working closely with healthcare providers, patient advocacy groups, and payors to make Duvyzat available to patients.

“We are pleased that the FDA approved Duvyzat for individuals with Duchenne age six and older. This adds to list of approved treatments for families facing this devastating disease and is an important step forward in accelerating transformative treatments for everyone independent of their genetic mutation,” noted Debra Miller, CureDuchenne founder and CEO.

DMD is a rare and severe neuromuscular genetic disease characterized by progressive muscle weakness and degeneration. The condition, which occurs in approximately one in every 3500–6000 male births worldwide, is caused by mutations in the DMD gene that result in the absence of a functional dystrophin protein. Without dystrophin, muscle fibers are highly susceptible to continuous injury, leading to chronic inflammation, impairment of muscle regeneration and muscle replacement by fibrotic and fat tissue. DMD symptoms are usually first seen between two and five years of age and worsen over time affecting the ability to walk. Eventually, the heart and respiratory muscles are affected, leading to premature death.

Duvyzat is an orally administered 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 been shown 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.

The approval is based on the results of the pivotal multicenter, randomized, double-blind, placebo-controlled Phase III EPIDYS trial (NCT02851797). Results from this study were published in The Lancet Neurology, in the paper, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicenter, randomized, double-blind, placebo-controlled, Phase III trial.

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.

“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.”

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