FDA approves mavacamten for children with obstructive HCM

The FDA approved Camzyos (mavacamten) for children with symptomatic obstructive hypertrophic cardiomyopathy who weigh at least 30 kg (approximately 66 lbs). The drug is now approved for adults and pediatric patients, and is the first treatment for children with the condition to improve functional capacity and symptoms, the FDA said.

Approval relied on the SCOUT-HCM trial, which included 44 patients and compared once-daily Camzyos with placebo for 28 weeks. The FDA said the drug reduced the pressure gradient in the left ventricular outflow tract while patients bore down, indicating less obstruction. No trial patients had a left ventricular ejection fraction drop below 50%, according to Healio. Camzyos carries a boxed warning for heart failure and is available only through a restricted Risk Evaluation and Mitigation Strategy program, the FDA said.