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FDA approves nonsteroidal treatment for Duchenne muscular dystrophy

On March 21 2024, the US Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older. The FDA granted this applicationpriority reviewandfast trackdesignation. It also receivedorphan drugandrare paediatric diseasedesignations.

Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. It is a histone deacetylase (HDAC) inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.

Commenting on the approval, Debra Miller, CureDuchenne founder and CEO, said:

“We are pleased that the FDA approved Duvyzat (givinostat) 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.”

DMD is the most common childhood form of muscular dystrophy and typically affects males. It is a rare neurological disorder which causes progressive muscle weakness due to a lack of muscle protein called dystrophin. Over time, the muscles deteriorate causing problems with walking and muscle strength and ultimately problems with breathing leading to early death. Life expectancy for those with DMD has increased over the years, with some patients surviving beyond 30 years.

Emily Freilich, MD, director of the Division of Neurology 1, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, said: “DMD denies the opportunity for a healthy life to the children it affects. The FDA is committed to advancing the development of new therapies for DMD. 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.”

Duvyzat’s efficacy for the treatment of DMD was evaluated in a randomised, double-blind, placebo-controlled 18-month phase 3 study. The primary endpoint was the change from baseline to month 18 using a four stair climb to measure muscle function. All participants continued to receive a standard of care steroid regimen throughout the study and, after 18 months of treatment, patients treated with Duvyzat showed statistically significant less decline in the time it took to climb four stairs compared to placebo. The mean change from baseline to month 18 in time to climb four stairs was 1.25 seconds for patients receiving Duvyzat compared to 3.03 seconds for patients receiving placebo. 

A secondary efficacy endpoint was the change from baseline to month 18 in physical function as assessed by the North Star Ambulatory Assessment (NSAA)—a scale commonly used to rate the motor function in boys with DMD who are capable of walking. Compared to placebo, patients treated with Duvyzat saw less worsening in their NSAA score after 18 months.

The approval of Duvyzat was granted to Italfarmaco S.p.A.

Further information about DMD

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