The FDA has approved Bristol Myers Squibb's Camzyos (mavacamten) for pediatric patients weighing at least 30 kg (66 lbs) with symptomatic obstructive hypertrophic cardiomyopathy, making it the first approved cardiac myosin inhibitor for children. The approval is based on the Phase 3 SCOUT-HCM trial of 44 adolescents, where Camzyos significantly reduced Valsalva left ventricular outflow tract obstruction by 48.0 mm Hg compared to placebo. The drug retains a boxed warning for heart failure risks.
FDA approval of Camzyos for pediatric patients
- ▪The pediatric approval of Camzyos (mavacamten) makes it the first and only FDA-approved cardiac myosin inhibitor therapy for symptomatic obstructive hypertrophic cardiomyopathy in a pediatric population.
- ▪The FDA pediatric approval of Camzyos (mavacamten) was based on efficacy and safety data from the Phase 3 SCOUT-HCM clinical trial.
- ▪The U.S. Food and Drug Administration approved Bristol Myers Squibb's Camzyos (mavacamten) on September 30, 2026, to treat symptomatic obstructive hypertrophic cardiomyopathy in pediatric patients weighing at least 30 kg (66 lbs).
Design of the SCOUT-HCM trial
- ▪The Phase 3 SCOUT-HCM trial evaluated 44 adolescent patients aged 12 to under 18 years with symptomatic NYHA class II-III obstructive hypertrophic cardiomyopathy over a 28-week period.
- ▪In the SCOUT-HCM trial, 23 patients received once-daily Camzyos (mavacamten) starting at 2.5 mg or 5 mg based on weight, while 21 patients received a placebo.
Efficacy results of the SCOUT-HCM trial
- ▪In the SCOUT-HCM trial, Camzyos (mavacamten) achieved its primary endpoint, reducing the least-squares mean Valsalva left ventricular outflow tract gradient by 48.0 mm Hg compared to placebo at week 28.
- ▪Secondary endpoints in the SCOUT-HCM trial favored Camzyos (mavacamten), showing reductions in resting left ventricular outflow tract gradient, postexercise gradient, and maximal left ventricular wall thickness.
Safety results of the SCOUT-HCM trial
- ▪In the SCOUT-HCM trial, no pediatric patients experienced a left ventricular ejection fraction decline below 50%, and no deaths, cases of atrial fibrillation, or symptomatic heart failure occurred.
- ▪Serious adverse events in the SCOUT-HCM trial occurred in two patients in the Camzyos (mavacamten) group and two patients in the placebo group, with no events leading to treatment discontinuation.
Camzyos safety requirements
- ▪Camzyos (mavacamten) carries a boxed warning for heart failure due to systolic dysfunction and is available only through a restricted Risk Evaluation and Mitigation Strategy program.
- ▪The FDA label for Camzyos (mavacamten) requires echocardiographic left ventricular ejection fraction assessment before and during treatment.
Cardiac myosin inhibitors
- ▪The pediatric approval of Camzyos (mavacamten) gives the medication the broadest indication of any cardiac myosin inhibitor.
- ▪Another cardiac myosin inhibitor for symptomatic obstructive hypertrophic cardiomyopathy, aficamten (Myqorzo), remains indicated only for adult patients.
Previous approval and use in adults
- ▪As of July 2026, Camzyos (mavacamten) had been prescribed by more than 5,000 healthcare providers to over 25,000 patients in the United States.
- ▪Camzyos (mavacamten) was first approved by the FDA in 2022 for adults with symptomatic NYHA class II-III obstructive hypertrophic cardiomyopathy.
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