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Novel Drug Approved for All Duchenne Muscular Dystrophy Variants

— Givinostat is the first nonsteroidal drug approved for DMD regardless of genetic mutation

MedpageToday
FDA APPROVED oral givinostat (Duvyzat) over an anatomical model of the proximal muscles of the male body

The FDA approved oral givinostat (Duvyzat) to treat Duchenne muscular dystrophy (DMD) in patients ages 6 years and older, the Thursday.

Givinostat is a histone deacetylase (HDAC) inhibitor that targets pathogenic processes to reduce inflammation and loss of muscle. It's the first nonsteroidal drug approved for patients with all genetic variants of DMD, the agency said.

"DMD denies the opportunity for a healthy life to the children it affects," said Emily Freilich, MD, of the neuroscience office in the FDA's Center for Drug Evaluation and Research. "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 childhood form of muscular dystrophy, and is characterized by a mutation that leads to a lack of dystrophin and muscle loss. It affects about one in 3,300 boys and has no known cure. Last year, the FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys), the first gene therapy for DMD.

Givinostat was assessed in the phase III study, a randomized, double-blind, placebo-controlled trial of 179 ambulant boys ages 6 years and older with DMD. The primary endpoint was the change from baseline to month 18 using a four-stair climb assessment to measure muscle function.

All participants received a standard-of-care steroid regimen throughout the study. After 18 months of treatment, boys treated with givinostat showed statistically in the time it took to climb the stairs compared with those in the placebo group.

A secondary endpoint was the change in scores on the North Star Ambulatory Assessment (NSAA), a scale that assesses aspects of functional domains associated with daily activities. Compared with placebo, patients treated with givinostat saw less worsening in their NSAA score after 18 months.

The most common adverse events in the givinostat group were diarrhea and vomiting. Thrombocytopenia, fever, and increased triglyceride levels also occurred. No treatment-related deaths were reported in the trial.

includes warnings that healthcare providers should evaluate a patient's platelet counts and triglycerides before prescribing givinostat, the FDA noted. Patients with a platelet count less than 150 x 10^9/L should not take the drug. Platelet counts and triglycerides should be monitored during treatment to determine whether dose changes are needed.

Dosages may also need to be modified for moderate or severe diarrhea or for QTc prolongation. "Patients taking certain medications that also cause QTc prolongation or have certain types of heart disease should avoid taking Duvyzat," the agency said.

  • Judy George covers neurology and neuroscience news for 鶹ý, writing about brain aging, Alzheimer’s, dementia, MS, rare diseases, epilepsy, autism, headache, stroke, Parkinson’s, ALS, concussion, CTE, sleep, pain, and more.