The FDA has approved Duvyzat for the treatment of Duchenne muscular dystrophy among patients aged 6 years and older, according to a press release.

Duvyzat (givinostat, Italfarmaco) is a histone deacetylase inhibitor that regulates the activity of histone deacetylase in dystrophic muscle to slow down muscle damage. This approval marks the first nonsteroidal drug available to treat patients with all genetic variants of Duchenne muscular dystrophy, according to the FDA.

The approval was based, in part, on data from the randomized, double-blind, placebo-controlled EPIDYS study of 179 boys aged 6 years and older who received givinostat twice daily or placebo alongside glucocorticosteroids. As Healio previously reported, results from the trial showed the drug reduced the decline of muscle function by 40%, as measured by a four stair climb test, after 18 months of treatment, meeting the study’s primary endpoint.

Diarrhea, abdominal pain, a reduction in platelets, nausea/vomiting, increased triglycerides and fever were the most commonly observed adverse events associated with givinostat.

“The FDA’s approval of Duvyzat for [Duchenne muscular dystrophy], 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 [Duchenne muscular dystrophy],” Paolo Bettica, MD, PhD, chief medical officer at Italfarmaco Group, said in a company press release.“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 [Duchenne muscular dystrophy] management in the U.S. as quickly as possible.”

The company said ITF Therapeutics, a new subsidiary based in the United States, will oversee the marketing and distribution of givinostat.