FDA approves first drug to improve functional capacity and symptoms in children with rare inherited heart condition

The FDA approved Camzyos (mavacamten) as the first treatment to improve functional capacity and symptoms in children with symptomatic obstructive hypertrophic cardiomyopathy, extending approval to pediatric patients weighing at least 30 kg, after adult approval in 2022. Efficacy was supported by the phase 3 SCOUT-HCM trial in 44 patients aged 12-18, where once-daily dosing for 28 weeks significantly reduced the Valsalva LVOT gradient versus placebo. The drug carries a boxed warning for heart failure risk and is available only through the Camzyos REMS program.