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Trial Shows Mavacamten Benefits Adolescents with Obstructive Heart Condition

Camzyos significantly reduced heart obstruction in 44 adolescents with obstructive hypertrophic cardiomyopathy, showing improvements in cardiac structure and symptoms with a safety profile similar to placebo.

  • On Sunday, March 29, 2026, Bristol Myers Squibb announced positive Phase 3 SCOUT-HCM trial data for Camzyos , marking the first study of a cardiac myosin inhibitor in adolescents aged 12 to 17 with symptomatic obstructive hypertrophic cardiomyopathy.
  • Pediatric-Onset obstructive hypertrophic cardiomyopathy carries a "far worse prognosis" than adult cases, yet current guidelines rely on extrapolated adult data because no approved pediatric therapies existed previously, according to Joseph Rossano, Chief of Cardiology at Children's Hospital of Philadelphia.
  • Mavacamten significantly reduced obstruction at 28 weeks, with patients experiencing a mean drop of about 48.5 mm Hg in the Valsalva LVOT gradient compared with almost no change in the placebo group.
  • Data indicated a safety profile similar to adults with no new safety signals, while Rossano noted, "There's a signal that this may be favorably remodeling the heart," potentially improving the disease's natural history.
  • Researchers plan to track outcomes to at least 50 weeks in the ongoing SCOUT-HCM long-term extension study, with future investigations potentially exploring efficacy in children younger than 12 years old.
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Precision Medicine Breakthrough: Mavacamten Markedly Reduces Heart Obstruction in Teens with Hypertrophic Cardiomyopathy

Children's Hospital of Philadelphia findings offer hope for pediatric cardiac patients and their families

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Healio broke the news in on Sunday, March 29, 2026.
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