FDA Approves Duchenne Muscular Dystrophy Treatment for Children

Duvyzat receives approval under fast-track designation.

The U.S. Food and Drug Administration (FDA) has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged six years and older. Duvyzat, the first nonsteroidal drug approved for all genetic variants of the disease, functions as a histone deacetylase (HDAC) inhibitor targeting pathogenic processes to mitigate inflammation and muscle loss.

“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,” said Director Emily Freilich of the Division of Neurology 1, Office of Neuroscience 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.”

Duchenne Muscular Dystrophy is the most prevalent childhood form of muscular dystrophy and mainly affects males. It leads to gradual muscle weakness due to the absence of dystrophin muscle protein. The efficacy of Duvyzat was demonstrated in an 18-month phase 3 study. The FDA granted the drug priority review under its fast-track designation.

As the Lord Leads, Pray with Us…

  • For Director Freilich as she oversees the Division of Neurology 1 in the FDA’s Office of Neuroscience.
  • For Director Patrizia Cavazzoni to be discerning as she heads the Center for Drug Evaluation and Research within the FDA.

Sources: Food and Drug Administration


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