FDA expands CAMZYOS approval to paediatric patients with obstructive hypertrophic cardiomyopathy
Posted on October 4, 2026
The U.S. Food and Drug Administration has expanded the approval of Bristol Myers Squibb’s CAMZYOS, also known as mavacamten, to include certain paediatric patients with symptomatic obstructive hypertrophic cardiomyopathy.
CAMZYOS can now be used to improve functional capacity and symptoms in adults and paediatric patients weighing at least 30 kg, or 66 pounds, with symptomatic obstructive hypertrophic cardiomyopathy.
Bristol Myers Squibb said CAMZYOS is the first and only FDA approved treatment for obstructive hypertrophic cardiomyopathy in a paediatric population and now has the broadest indication of any cardiac myosin inhibitor.
The expanded indication is supported by positive findings from the Phase III SCOUT HCM study, which assessed the efficacy and safety of CAMZYOS in patients aged 12 to under 18 years.
The trial included 23 patients who received CAMZYOS and 21 who were given placebo. Participants had symptomatic New York Heart Association Class II or III obstructive hypertrophic cardiomyopathy.
Patients entering the study had a left ventricular ejection fraction of at least 60%, a Valsalva left ventricular outflow tract gradient of at least 30 mmHg and a maximum gradient of at least 50 mmHg either at rest or following provocation.
Background treatment with beta blockers, calcium channel blockers, disopyramide or combinations of these therapies was permitted.
SCOUT HCM met its primary endpoint at week 28.
Patients treated with CAMZYOS experienced a mean reduction of 49.4 mmHg from baseline in provoked Valsalva left ventricular outflow tract gradient, compared with a reduction of 1.8 mmHg among patients receiving placebo.
The least squares mean difference versus placebo was minus 48.0 mmHg, with a 95% confidence interval ranging from minus 67.7 to minus 28.3 and a p value below 0.0001.
CAMZYOS also demonstrated improvements across selected secondary measures.
Resting left ventricular outflow tract gradient decreased by a mean of 41 mmHg with CAMZYOS, while the placebo group experienced an increase of 5.3 mmHg.
The least squares mean difference between the groups was minus 47.0 mmHg.
Among evaluable patients, post exercise left ventricular outflow tract gradient also improved with CAMZYOS. The least squares mean difference compared with placebo was minus 41.7 mmHg.
Maximum left ventricular wall thickness decreased during treatment as well. The least squares mean difference compared with placebo was minus 1.8 mm.
No patients receiving CAMZYOS experienced a reduction in left ventricular ejection fraction below 50%, and no adverse events resulted in treatment discontinuation.
No new adverse reactions were identified beyond those already observed in adults.
Serious adverse events occurred in two patients receiving CAMZYOS, representing 9% of the treatment group, and two patients receiving placebo, representing 10%.
Al Reba, Senior Vice President of Immunology and Cardiovascular Commercialization at Bristol Myers Squibb, said the expanded approval represents an important development for younger patients and families affected by symptomatic obstructive hypertrophic cardiomyopathy.
He said the disease can create a substantial burden during an important period of development and highlighted the growing clinical evidence supporting CAMZYOS.
Joseph Rossano, MD, Principal Investigator of SCOUT HCM and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia, described the decision as an important milestone in paediatric cardiology.
He said children with the condition now have an FDA approved therapy specifically shown to reduce left ventricular outflow tract obstruction.
CAMZYOS carries a boxed warning for the risk of heart failure caused by systolic dysfunction.
The treatment can reduce left ventricular ejection fraction, meaning patients require echocardiogram assessments before and during therapy.
Starting CAMZYOS is not recommended in patients with a left ventricular ejection fraction below 55%, and treatment should be interrupted if this falls below 50% or if heart failure symptoms or worsening clinical status develop.
CAMZYOS is available only through a restricted Risk Evaluation and Mitigation Strategy programme because of the risk of heart failure.
The FDA first approved CAMZYOS in 2022 for adults with symptomatic New York Heart Association Class II or III obstructive hypertrophic cardiomyopathy.
Bristol Myers Squibb said the medicine has since been prescribed by more than 5,000 healthcare professionals to over 25,000 patients in the U.S.
The company is also discussing the paediatric SCOUT HCM findings with regulatory authorities in other countries as it seeks additional approvals.
Related Topics and Keywords
BMS, Bristol-Myers Squibb (BMS), camzyos, Cardiac myosinhibitor, cardiology, Cardiovascular Disease, clinical trials, hcm, Hypertrophic Cardiomyopathy, mavacamten, obstructive HCM, pediatric cardiology, pharma news
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