FDA Approves Camzyos for Children With Obstructive HCM

The FDA has approved Camzyos (mavacamten) capsules for children weighing at least 30 kg with symptomatic obstructive hypertrophic cardiomyopathy, or oHCM, according to an FDA announcement. Camzyos was first approved for adults in 2022 and is now cleared for both adults and pediatric patients who meet the weight threshold.

oHCM is an inherited condition that thickens the heart muscle and can narrow the left ventricular outflow tract, the pathway blood takes leaving the heart. That narrowing can cause shortness of breath, fatigue, and dangerous heart rhythms.

The pediatric approval rests on SCOUT HCM, a phase 3 randomized, double blind, placebo controlled trial in 44 patients aged 12 to 18 with symptomatic oHCM. Over 28 weeks, patients received Camzyos or placebo daily, and the study measured change in the Valsalva LVOT gradient, a pressure reading taken during a breath holding maneuver. Camzyos significantly reduced this gradient compared to placebo. Because similar reductions in adults have tracked with improved functional capacity and symptoms, the FDA expects the same benefit in children.

Camzyos carries a boxed warning for heart failure risk and is available only through a restricted REMS program. Dizziness and fainting were the most common side effects reported above placebo rates. It also received orphan drug designation for this indication.

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