FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment

In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S. Food and Drug Administration (FDA) approval of Duvyzat (givinostat), a novel histone deacetylase (HDAC) inhibitor, for patients aged 6 and older. This approval signifies a major milestone in the management of DMD, a rare and progressive neuromuscular disorder that affects individuals from early childhood.

Paolo Bettica, MD, PhD, Chief Medical Officer at Italfarmaco Group, highlighted the significance of this approval, stating, “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.” The development and approval of Duvyzat underscore Italfarmaco’s dedication to addressing the unmet needs of the DMD community and their families, who have been integral to reaching this landmark FDA approval.

Duvyzat’s approval is based on the results from the pivotal EPIDYS phase 3 trial, which demonstrated a statistically significant and clinically meaningful improvement in the time to complete the four-stair climb assessment among patients treated with Duvyzat, in addition to standard glucocorticosteroid therapy. The study also showed favorable outcomes in key secondary endpoints, including the North Star Ambulatory Assessment (NSAA) and fat infiltration evaluation by magnetic resonance imaging. These results, published in The Lancet Neurology in March 2024, indicate Duvyzat’s potential to delay disease progression and become a fundamental component of DMD treatment.

The FDA’s recognition of Duvyzat’s therapeutic potential is further evidenced by its priority review, orphan drug, and rare pediatric disease designations. Beyond the U.S., Italfarmaco has submitted a Marketing Authorisation Application for givinostat to the European Medicine Agency (EMA), with the application currently under review.

To support the commercialization of Duvyzat in the U.S., Italfarmaco has established ITF Therapeutics LLC, a fully owned subsidiary tasked with making Duvyzat accessible to the DMD patient population. The company is actively collaborating with healthcare providers, patient advocacy groups, and payers to ensure the broad availability of this innovative treatment.

This FDA approval not only represents a significant breakthrough in the treatment of Duchenne muscular dystrophy but also reinforces Italfarmaco’s role as a key player in the development of novel therapies for rare diseases. With Duvyzat now set to become a vital therapy for DMD management, the future holds new hope for affected individuals and their families, promising improved outcomes and quality of life.