FDA Approves Duvyzat Medicine to treat Duchenne Muscular Dystrophy

Duvyzat (givinostat) oral medication was approved today by the United States Food and Drug Administration for the treatment of Duchenne Muscular Dystrophy (DMD) in individuals aged six and up. Duvyzat is the first nonsteroidal medication approved to treat patients with all genetic variations of DMD. It functions as a histone deacetylase (HDAC) inhibitor, targeting pathogenic processes to minimize inflammation and muscle loss.

“DMD denies youngsters the opportunity to live healthy lives. Emily Freilich, M.D., head of the Division of Neurology 1, Office of Neuroscience in the FDA’s Center for Drug Evaluation and Research, stated that the agency is committed to advancing the discovery of new therapeutics for Duchenne Muscular Dystrophy. “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.”

DMD is the most common form of muscular dystrophy in childhood, affecting primarily males. Dystrophin deficiency is an uncommon neurological condition that causes increased muscular weakness. Over time, the muscles weaken, producing difficulty with walking and muscle strength, and eventually breathing problems, which lead to premature death. Life expectancy for people with DMD has grown over time, with some patients living past 30 years.

A randomized, double-blind, placebo-controlled 18-month phase 3 study was conducted to assess the efficacy of Duvyzat for the treatment of DMD. The primary outcome was muscular function change from baseline to month 18, as measured by a four-stair climb. All individuals received a standard of care steroid regimen throughout the study, and after 18 months of treatment, patients treated with Duvyzat had a statistically significant lower reduction in the time it took to ascend four stairs than placebo. The average change in time to ascend four steps from baseline to Month 18 was 1.25 seconds for patients using Duvyzat versus 3.03 seconds for placebo participants.

A secondary effectiveness goal was the change in physical function from baseline to month 18 as measured by the North Star Ambulatory Assessment (NSAA), which is a scale routinely used to score motor function in boys with DMD who can walk. After 18 months, patients who received Duvyzat experienced less NSAA score degradation than those who received a placebo.

Duvyzat’s most common side effects are diarrhea, abdominal pain, a drop in platelets (which can lead to increased bleeding), nausea/vomiting, a rise in triglycerides (a form of fat in the body), and fever.

Duvyzat’s prescribing material includes warnings stating that health care practitioners should check the patient’s platelet counts and triglycerides before prescribing the medication. Patients with a platelet count of less than 150 x 109/L should avoid taking Duvyzat. Platelet counts and triglycerides should be checked as directed during treatment to evaluate whether dosage changes are required. Dosage adjustments may also be required for moderate or severe diarrhea. Duvyzat may potentially cause QTc prolongation, increasing the risk of irregular heartbeats. Patients taking certain drugs that cause QTc prolongation or who have specific forms of heart problems should avoid Duvyzat.

The suggested dosage of Duvyzat is based on the individual’s weight. It should be given orally, twice a day, with food.

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