FDA approves Bristol Myers Squibb's Camzyos for pediatric oHCM

The U.S. Food and Drug Administration approved an expanded indication for Bristol Myers Squibb's Camzyos (mavacamten) on September 30, 2026. The cardiac myosin inhibitor is now approved to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more who have symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

The regulatory decision makes Camzyos the only FDA-approved therapy for oHCM in a pediatric population. It was based on data from the Phase 3 SCOUT-HCM trial, which evaluated 44 adolescent patients aged 12 to younger than 18 years. In the study, 23 patients received Camzyos and 21 received a placebo.

The trial met its primary endpoint, showing a statistically significant reduction in the Valsalva left ventricular outflow tract gradient at Week 28 compared with placebo. No patients experienced a left ventricular ejection fraction below 50%, and no adverse events led to treatment discontinuation. Serious adverse events occurred in two patients in the Camzyos group (9%) and two in the placebo group (10%).

Camzyos carries a boxed warning for the risk of heart failure due to systolic dysfunction and is available only through the Camzyos REMS restricted distribution program. First cleared by the FDA in 2022 for adults, the medicine has been prescribed by more than 5,000 healthcare providers to over 25,000 patients in the United States, and Bristol Myers Squibb is currently discussing the pediatric data with regulators internationally.