ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy

WEDNESDAY, April 1, 2026 (HealthDay News) -- Mavacamten, the first-in-class cardiac myosin inhibitor approved for treatment of symptomatic adults with obstructive hypertrophic cardiomyopathy (HCM), is efficacious for adolescents with obstructive HCM, according to a study published online March 29 in the New England Journal of Medicine to coincide with the annual meeting of the American College of Cardiology, held from March 28 to 30 in New Orleans.Joseph W. Rossano, M.D., from the Children's Hospital of Philadelphia, and colleagues conducted a randomized international study assessing the efficacy and safety of mavacamten in adolescents (aged 12 to <18 years) with obstructive HCM. After five weeks of screening, 44 participants were randomly assigned to either mavacamten (2.5 or 5 mg/day based on body weight) or placebo for 28 weeks.The researchers found that participants receiving mavacamten had a substantial improvement in terms of the change in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 28, with average decreases of 48.5 and 0.5 mmHg in the mavacamten and placebo groups, respectively. Significant improvements in favor of mavacamten were also seen in secondary end points, including change in resting LVOT gradient, maximal left ventricular wall thickness, peak oxygen consumption, and symptom measures such as fatigue and shortness of breath. Troponin and peptide levels decreased in those taking mavacamten and increased in those taking placebo."Beyond symptom relief, there's a signal that this may be favorably remodeling the heart, which could improve the natural history of the disease," Rossano said in a statement. "This suggests that it could be important to start children on this therapy when they’re young, before they've had many decades of ongoing injury to the heart from the obstruction."The study was funded by Bristol Myers Squibb, manufacturer of mavacamten.Abstract/Full Text (subscription or payment may be required)More Information.Sign up for our weekly HealthDay newsletter

ACC: Mavacamten Efficacious for Adolescents With Obstructive Hypertrophic Cardiomyopathy

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