Today, the U.S. Food and Drug Administration has approved Duvyzat (givinostat), an oral medication, for treating Duchenne Muscular Dystrophy (DMD) in patients aged six and above. Duvyzat is notable for being the first nonsteroidal drug authorized to tackle DMD across all genetic variations. It functions as a histone deacetylase (HDAC) inhibitor, targeting specific disease processes to alleviate inflammation and muscle loss.

Dr. Emily Freilich, Director of the Division of Neurology 1 at the FDA's Center for Drug Evaluation and Research, emphasized the agency's commitment to advancing DMD therapies and providing additional treatment options regardless of genetic mutation.

DMD predominantly affects males and is the most common form of childhood muscular dystrophy, characterized by progressive muscle weakening due to insufficient dystrophin protein. While life expectancy for DMD patients has improved, effective treatments remain essential.

Duvyzat's efficacy was evaluated through an 18-month phase 3 study using a randomized, double-blind, placebo-controlled approach. Patients treated with Duvyzat showed significantly less decline in muscle function compared to those on placebo, even while receiving standard steroid care throughout the study.

Common side effects of Duvyzat include diarrhea, abdominal pain, decreased platelet count, nausea/vomiting, increased triglycerides, and fever. Prescribing information emphasizes the necessity of evaluating platelet counts and triglyceride levels before initiation, with regular monitoring during treatment. Dosage adjustments may be necessary for patients experiencing moderate or severe diarrhea. Additionally, caution is advised for patients taking medications with QTc prolongation potential or those with specific heart conditions, as Duvyzat may increase the risk of irregular heartbeats.

Dosage of Duvyzat is weight-dependent, administered orally twice daily with food. The FDA granted priority review and fast track designation to this application, alongside orphan drug and rare pediatric disease designations. Italfarmaco S.p.A. received approval for Duvyzat.

 

Source: prnewswire.com

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