TL;DR
Get workout gear delivered free — and shop member deals
- Fast, free delivery on millions of items
- Access to Prime Big Deal Days deals on October 6–7
- Prime Video, Amazon Music and more included
The FDA expanded mavacamten (Camzyos) approval to children weighing at least 30 kg who have symptomatic obstructive hypertrophic cardiomyopathy. The decision follows the 44-adolescent SCOUT-HCM trial, but treatment remains subject to a restricted safety program because of the risk of heart failure from reduced heart-pumping function.
The FDA expanded approval of mavacamten, sold as Camzyos, to include children weighing at least 30 kg (about 66 pounds) who have symptomatic obstructive hypertrophic cardiomyopathy (HCM). The medicine is indicated to improve symptoms and functional capacity, adding a drug option specifically approved for pediatric patients with the condition.
The pediatric approval was based on SCOUT-HCM, a phase III randomized trial involving 44 adolescents. Participants had obstructive HCM and were receiving background treatment that could include a beta blocker, calcium channel blocker and/or disopyramide. The trial enrolled patients with a left ventricular ejection fraction of at least 60% and specified measures of obstruction.
At week 28, the mavacamten group had a 48.0 mm Hg reduction in the Valsalva left ventricular outflow tract gradient from baseline, compared with placebo; the reported difference was statistically significant (P<0.0001). The research team also reported improvements in obstruction at rest, measures of diastolic function and cardiac hypertrophy, and markers of cardiac stress and myocardial injury.
The report said no trial participant had a left ventricular ejection fraction below 50%, and investigators observed no cases of atrial fibrillation, symptomatic heart failure or death during the study. Those findings describe the trial group and do not remove the medicine’s known safety concerns or establish how it will perform in broader pediatric use.
A New Approved Option for Pediatric HCM
Children with symptomatic obstructive HCM have had limited pharmacological options specifically studied and approved for their age group. Extending mavacamten’s indication gives eligible families and clinicians an additional treatment choice aimed at reducing the obstruction that can contribute to symptoms and limit activity. The authorization does not mean every child with HCM is eligible: it applies to patients with the symptomatic obstructive form who meet the weight threshold.
Joseph Rossano, the SCOUT-HCM investigator at Children’s Hospital of Philadelphia, called the decision “a landmark moment for pediatric cardiology” in a statement issued by manufacturer Bristol Myers Squibb. The trial results support the approval, but the study’s size and duration mean longer-term outcomes and safety in wider patient populations remain important considerations.
mavacamten Camzyos pediatric heart medication
As an affiliate, we earn on qualifying purchases.
As an affiliate, we earn on qualifying purchases.
From Adult Approval to Pediatric Use
Mavacamten was initially approved for adults with obstructive HCM in 2022. The October 2026 action extends its use to qualifying pediatric patients. The source report described it as the first cardiac myosin inhibitor approved for pediatric symptomatic obstructive HCM; another medicine in the class, aficamten (Myqorzo), was reported as indicated for adults only.
HCM involves thickening of the heart’s left ventricle that is not explained by another cardiac, systemic or metabolic disease. Pediatric HCM is rare and is often inherited. In obstructive HCM, blood flow out of the left ventricle is impeded. The trial assessed the pressure gradient associated with that obstruction, including measurements made during a Valsalva maneuver.
“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
heart condition treatment for kids
As an affiliate, we earn on qualifying purchases.
As an affiliate, we earn on qualifying purchases.
Safety and Long-Term Outcomes
The supplied report does not give the full prescribing instructions for pediatric patients, the number of children expected to qualify, or longer-term follow-up results. The trial included 44 adolescents, so it cannot by itself answer all questions about uncommon adverse events or outcomes across a broader range of pediatric ages and patients.
Mavacamten can cause heart failure through systolic dysfunction. It is dispensed through a restricted Risk Evaluation and Mitigation Strategy (REMS) program, and its label warns against use in people with heart failure and lists certain medicines affecting CYP2C19 or CYP3A4 as contraindications. Families and clinicians will need to follow the product’s current labeling and monitoring requirements; the absence of specified safety events in this trial does not establish that risks are absent.
obstructive hypertrophic cardiomyopathy children medication
As an affiliate, we earn on qualifying purchases.
As an affiliate, we earn on qualifying purchases.
Prescribing Under REMS Safeguards
The immediate next step is clinical use under the medicine’s existing restricted safety program, with prescribing decisions based on the FDA-approved label and each patient’s condition and other medications. Clinicians will need to assess whether a child meets the indication and weight threshold and account for the warnings about reduced heart function and drug interactions.
Further follow-up and real-world experience may add information about longer-term benefits and risks in children. The source report did not specify a timeline for additional pediatric results or any planned change to the REMS program.
pediatric heart health monitoring devices
As an affiliate, we earn on qualifying purchases.
As an affiliate, we earn on qualifying purchases.
Key Questions
Which children are included in the expanded approval?
The approval covers children weighing at least 30 kg (about 66 pounds) who have symptomatic obstructive hypertrophic cardiomyopathy. It is not described as an approval for all forms of HCM.
What did the SCOUT-HCM trial find?
In the 44-adolescent phase III randomized trial, mavacamten reduced the Valsalva left ventricular outflow tract gradient by 48.0 mm Hg from baseline at week 28 compared with placebo, with a reported P value below 0.0001. Researchers also reported improvements in several other measures of obstruction and cardiac function.
Does the trial mean mavacamten has no serious risks for children?
No. The report said the trial recorded no ejection-fraction dips below 50%, atrial fibrillation, symptomatic heart failure or deaths among participants. Mavacamten still carries a risk of heart failure from systolic dysfunction and is subject to a restricted REMS program.
Is another cardiac myosin inhibitor approved for children?
The source report said aficamten (Myqorzo), another cardiac myosin inhibitor for symptomatic obstructive HCM, remained indicated for adults only at the time of the announcement.
Source: rss
Fall Picks
fall essentials
As an affiliate, we earn on qualifying purchases.
