FDA Approves First Oral Nonsteroidal Drug for Duchenne

Milan-based biotech Italfarmaco has won FDA approval for Duvyzat (givinostat), the first oral nonsteroidal drug for treating Duchenne muscular dystrophy in children aged six and younger.

A histone deacetylase inhibitor, Duvyzat reduces inflammation and muscle loss.

Duvyzat was shown to reduce functional deterioration in an 18-month phase 3 trial when co-administered with standard steroidal therapy. While patients taking a placebo lost three seconds in the time needed to climb four stairs, those taking Duvyzat lost 1.25 seconds. Treated patients also experienced less worsening on a secondary functional endpoint, the North Star Ambulatory Assessment.

The drug was evaluated under priority review and holds orphan drug and rare pediatric disease drug status.

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