FDA approves Italfarmaco’s Duvyzat as first nonsteroidal Duchenne muscular dystrophy drug
The US Food and Drug Administration (FDA) has approved Italfarmaco’s Duvyzat (givinostat) as the first nonsteroidal drug to treat patients with all genetic variants of Duchenne muscular dystrophy (DMD).
Estimated to affect one in every 3,500 to 6,000 male births worldwide, DMD is a severe neuromuscular genetic disease caused by a change or mutation in the gene that encodes instructions for dystrophin, which is required to strengthen and protect muscles. Symptoms of the disorder are usually first seen in early childhood and worsen over time, affecting patients’ ability to walk as well as their heart and respiratory muscles.
Italfarmaco’s Duvyzat is an oral histone deacetylase inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle. The FDA’s decision on the drug applies to DMD patients aged six years and older and is supported by the late-stage EPIDYS trial, which randomised 179 ambulant boys to receive either Duvyzat twice daily or placebo, in addition to glucocorticosteroid treatment.
The study met its primary endpoint, with Duvyzat-treated patients demonstrating a statistically significant and clinically meaningful difference in time to complete the four-stair climb assessment. Treatment with Duvyzat was also associated with favourable results on key secondary endpoints, including North Star Ambulatory Assessment, a scale commonly used to rate the motor function in boys with DMD who are capable of walking.
“This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation,” said Emily Freilich, director of the division of neurology, office of neuroscience in the FDA’s Center for Drug Evaluation and Research.
Also commenting on the authorisation, Paolo Bettica, chief medical officer at Italfarmaco Group, said: “The FDA’s approval of Duvyzat for DMD, based on our robust and successful clinical development programme, reflects Italfarmaco’s commitment to providing a safe and proven-effective therapy that can have a meaningful impact for people living with DMD.”
Bettica added that the company is now focusing on making Duvyzat available as a treatment for DMD management in the US “as quickly as possible”.
March 25, 2024
