The FDA has approved Duvyzat as the first nonsteroidal drug for patients with all genetic variants of Duchenne muscular dystrophy, the agency said March 21.
Duvyzat, or givinostat, targets pathogenic processes in the body to reduce inflammation and muscle loss. The twice-daily oral medication is indicated for children 6 and older with the rare neuromuscular condition.
In a phase 3 clinical trial, patients treated with Duvyzat demonstrated better physical function and took less time to climb four stairs compared to a placebo group, marking a statistically significant difference.
The drug's prescribing information includes a warning for clinicians, advising them to evaluate patients' platelet counts and triglycerides before prescribing.
The approval comes after the FDA granted the drug priority review, orphan drug and rare pediatric disease designations.
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