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News
Mavacamten just got a pediatric upgrade. The FDA has expanded the approval of mavacamten (Camzyos) to include pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) weighing at least 30 kg — marking the first FDA-approved therapy for this condition in children. The decision was backed by the SCOUT-HCM trial, which showed significant reductions in left ventricular outflow tract obstruction in adolescents.
Mavacamten just got a pediatric upgrade. The FDA has expanded the approval of mavacamten (Camzyos) to cover pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who weigh at least 30 kg — making it the first FDA-approved therapy for this serious heart condition in children. Bristol Myers Squibb announced the decision, calling it a "landmark moment for pediatric cardiology."
The approval was based on the SCOUT-HCM trial, which enrolled 44 adolescents (ages 12–17) with NYHA class II or III obstructive HCM. Participants were randomized to mavacamten or placebo, and the drug delivered a striking reduction in left ventricular outflow tract (LVOT) gradient by week 28. Safety measures did not differ between the two study arms.
By the Numbers:
Why it matters: Until now, children with obstructive HCM had no FDA-approved medical therapy targeting the root cause of their condition. This approval opens a new treatment frontier for young patients who previously had limited options beyond watchful waiting or invasive procedures.
