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The FDA expanded approval of mavacamten (Camzyos) to children weighing at least 30 kg who have symptomatic obstructive hypertrophic cardiomyopathy. The decision was based on the 44-adolescent SCOUT-HCM trial, which found reduced outflow obstruction over 28 weeks; the drug carries a restricted safety program because of heart-failure risk.
The U.S. Food and Drug Administration has expanded approval of mavacamten (Camzyos) to children weighing at least 30 kilograms who have symptomatic obstructive hypertrophic cardiomyopathy, extending a treatment option previously approved for adults. The drug is intended to improve functional capacity and symptoms, but it is subject to restricted access and monitoring because of the risk of heart failure caused by reduced heart pumping function.
The pediatric approval was supported by SCOUT-HCM, a phase III randomized trial involving 44 adolescents. Participants had symptomatic obstructive HCM, a left ventricular ejection fraction of at least 60%, and specified levels of obstruction measured by the left ventricular outflow tract (LVOT) gradient. They were also receiving background treatment with a beta blocker, calcium channel blocker and/or disopyramide.
After 28 weeks, adolescents assigned mavacamten had a mean improvement in Valsalva LVOT gradient of 48.0 mm Hg from baseline compared with placebo, a statistically significant result (P<0.0001), according to the trial findings reported by the investigators. The report also described improvements in measures including obstruction at rest, diastolic function, cardiac hypertrophy and markers of cardiac stress and myocardial injury.
The trial report recorded no ejection-fraction readings below 50% and no cases of atrial fibrillation, symptomatic heart failure or death among participants taking mavacamten. Those findings describe the trial population and period; they do not remove the safety warning. The medicine remains available through a restricted Risk Evaluation and Mitigation Strategy (REMS) program for adults and children.
A New Option for Young Patients
The FDA decision gives eligible children a treatment specifically approved for symptomatic obstructive HCM, a condition in which thickened heart muscle can obstruct blood flow out of the left ventricle. The original report describes pediatric HCM drug options as historically limited and not well established, making an approved therapy relevant to families and clinicians weighing symptom management.
The approval does not mean the medicine is suitable for every child with HCM. It applies to patients who meet the weight and condition criteria, and the safety restrictions require clinicians to weigh potential benefit against the risk of systolic dysfunction and heart failure. The trial was small and involved adolescents, so the results should not be read as establishing effects across every age group or clinical situation covered by the pediatric indication.
For the field, the decision also marks a difference between available drugs: mavacamten is now indicated for eligible pediatric patients, while the other cardiac myosin inhibitor named in the report, aficamten (Myqorzo), remains indicated only for adults with symptomatic obstructive HCM.
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From Adult Approval to Pediatric Use
Mavacamten was first approved for adults with obstructive HCM in 2022. The October 1, 2026, FDA expansion follows evidence from SCOUT-HCM, which evaluated the medicine in adolescents against placebo. The new indication is for pediatric patients weighing at least 30 kg, or about 66 pounds, with symptomatic obstructive HCM.
HCM is described in the source report as a rare, often genetically inherited cardiovascular condition involving thickening of the left ventricle that is not explained by another cardiac, systemic or metabolic disease. In obstructive HCM, the thickened muscle can impede blood leaving the heart. Trial participants were selected using heart-function and gradient criteria, so the study’s results relate to a defined group rather than all children diagnosed with HCM.
Joseph Rossano, a SCOUT-HCM investigator at Children’s Hospital of Philadelphia, said in a statement issued by manufacturer Bristol Myers Squibb that the approval gives children an FDA-approved therapy to reduce LVOT obstruction. Lisa Salberg, founder and CEO of the Hypertrophic Cardiomyopathy Association, said the approval was welcome for younger patients. These are attributed responses to the decision, not independent conclusions about long-term outcomes.
“The FDA approval of Camzyos for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy represents a landmark moment for pediatric cardiology.”
— Joseph Rossano, MD, SCOUT-HCM investigator at Children’s Hospital of Philadelphia, in a Bristol Myers Squibb press release
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Limits of the Available Evidence
The source report does not provide the full FDA label, detailed dosing instructions, or the complete safety data from SCOUT-HCM. It also does not specify how long-term outcomes will be monitored after approval or whether the trial results will be confirmed in larger or longer studies. The trial enrolled 44 adolescents and followed its primary gradient outcome through 28 weeks, so it cannot by itself establish long-term effects or answer every question about use in younger children.
Although no specified serious safety events were reported in the adolescent trial, mavacamten can reduce systolic function. The medicine’s label warns against use in people with heart failure and lists concurrent use with certain CYP2C19 or CYP3A4 medications as contraindicated. The precise patient-level decisions and monitoring schedule should be determined under the approved prescribing information and REMS requirements.
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Prescribing Under REMS Safeguards
Eligible pediatric patients may be considered for mavacamten under the restricted REMS program and the drug’s prescribing information. Clinicians and families will need to review eligibility, potential interactions, and the required safeguards before treatment. The original report does not give a specific launch date, access timeline or details of any planned follow-up study.
Further information from the FDA label, real-world use and future research may clarify how the treatment performs beyond the 28-week trial period, including its longer-term safety and effects in a broader pediatric population. Until then, the approval establishes a new option for eligible patients, while its benefits and risks must be assessed for each child.
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Key Questions
Which children are covered by the expanded approval?
The indication covers pediatric patients weighing at least 30 kg (about 66 pounds) who have symptomatic obstructive hypertrophic cardiomyopathy. The trial participants also met specific heart-function and outflow-gradient criteria; families should consult clinicians about eligibility under the FDA-approved label.
What did the SCOUT-HCM trial find?
In a randomized trial of 44 adolescents, mavacamten improved the Valsalva LVOT gradient compared with placebo over 28 weeks. The report also describes changes in several other heart-function measures, but the study does not establish long-term outcomes.
What safety precautions apply to mavacamten?
Mavacamten is available through a restricted REMS program because of the risk of heart failure caused by systolic dysfunction. Its label warns against use in people with heart failure and lists certain CYP2C19 and CYP3A4 medication combinations as contraindicated.
Is aficamten also approved for children?
No pediatric indication for aficamten (Myqorzo) is described in the source report. It says the medicine remains approved for adults only with symptomatic obstructive HCM.
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