The U.S. Food and Drug Administration approved Italfarmaco S.p.A’s Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy or DMD in patients six years of age and older.
Duvyzat is the first nonsteroidal drug approved by the agency to treat patients with all genetic variants of DMD. It is a histone deacetylase or HDAC inhibitor that works by targeting pathogenic processes to reduce inflammation and loss of muscle.
Duvyzat should be administered orally twice daily with food, and the recommended dosage of the drug is determined by the individual’s body weight.
DMD is the most common childhood form of muscular dystrophy and typically affects males. The rare neurological disorder causes progressive muscle weakness due to a lack of muscle protein called dystrophin. Over time, the patients face problems with walking and muscle strength and ultimately problems with breathing leading to early death.
The approval of Duvyzat was based on a randomized, 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, and patients treated with Duvyzat showed statistically significant less decline in the time it took to climb four stairs compared to placebo.
Emily Freilich, 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. … 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.”
The FDA granted priority review and fast track designation tothis application. It also received orphan drug and rare pediatric disease designations.
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