Bristol Myers Squibb (NYSE: BMY) won US FDA approval for an expanded indication for Camzyos (mavacamten), a selective cardiac myosin inhibitor, covering symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and pediatric patients weighing 30 kg or more. Bristol Myers Squibb said Camzyos is the first and only FDA-approved therapy for pediatric oHCM
The approval rests on the Phase III SCOUT-HCM trial, a randomized, double-blind, placebo-controlled study enrolling 44 adolescents aged 12 to under 18 years with symptomatic New York Heart Association (NYHA) Class II–III oHCM. Camzyos met the primary endpoint, with a least-squares mean difference in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 of −48.0 mmHg versus placebo (95% CI: −67.7 to −28.3; P<0.0001). No patients experienced left ventricular ejection fraction (LVEF) below 50%, no adverse events led to discontinuation, and no new adverse reactions were identified beyond the adult safety profile.
The label carries a boxed warning for heart failure risk due to systolic dysfunction, and the drug is available only through a restricted Risk Evaluation and Mitigation Strategy (REMS) program.
Mavacamten is an orally administered, selective, reversible allosteric cardiac myosin inhibitor that reduces excessive actin-myosin cross-bridge formation and the hypercontractility underlying oHCM.