Italian pharmaceutical firm Italfarmaco has received the US Food and Drug Administration (FDA) approval for Duvyzat (givinostat) to treat Duchenne muscular dystrophy (DMD).

Duvyzat is an investigational drug discovered by Italfarmaco’s research and development team in collaboration with Telethon and the Duchenne Parent Project (Italy).

The histone deacetylase (HDAC) inhibitor modulates the deregulated activity of HDACs in the dystrophic muscle, which drives the lack of dystrophin, associated with DMD.

Its mechanism of action inhibits HDAC pathological overactivity, thereby counteracting the disease pathology and slowing down muscle deterioration.

The drug is indicated for the treatment of patients aged 6 years and above with DMD, a rare X-linked progressive and life-threatening neuromuscular condition.

Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD, said the US regulator.

Italfarmaco Holding president and Italfarmaco Group chairman Francesco De Santis said: “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.”

Italfarmaco Group chief medical officer Paolo Bettica said: “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, who 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.”

The FDA approval is supported by the results from EPIDYS, a randomized, double-blind, placebo-controlled 18-month Phase 3 clinical trial.

In the Phase 3 study, a total of 179 ambulant boys aged six years and above received either Duvyzat twice daily or a placebo, in addition to gluco-corticosteroid treatment.

The study met its primary endpoint of Duvyzat showing a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment.

The drug also showed favourable results on key secondary endpoints including North Star Ambulatory Assessment (NSAA), and fat infiltration evaluation by magnetic resonance imaging.

EPIDYS trial investigator Craig McDonald said: “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.”

Parent Project Muscular Dystrophy (PPMD) founding president and CEO Pat Furlong said: “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.”

Italfarmaco has significantly expanded its footprint in the US by forming a new subsidiary, ITF Therapeutics, which will be responsible for the commercialisation of Duvyzat in the US.

The Italian drugmaker is working closely with healthcare providers, patient advocacy groups and payors to make Duvyzat available to patients.