Camzyos (mavacamten) approved for adolescents with obstructive hypertrophic cardiomyopathy
FDA expanded the Camzyos label to include patients aged 12 to under 18 with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) — the first cardiac myosin inhibitor approved for this age group. The decision was supported by the phase 3 SCOUT-HCM trial, in which mavacamten significantly reduced outflow-tract gradient vs placebo at 28 weeks.
Camzyos (mavacamten) ↗ is a first-in-class cardiac myosin inhibitor originally approved in 2022 for adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). On 30 September 2026, FDA expanded the label ↗ to include adolescents aged 12 to under 18 years — making it the first disease-targeted therapy for this age group.
About obstructive HCM
Hypertrophic cardiomyopathy (HCM) ↗ is the most common inherited cardiac condition, affecting approximately 1 in 500 people. The obstructive form occurs when the thickened septal wall narrows the left ventricular outflow tract, causing exertional breathlessness, chest pain, pre-syncope, and — in a minority — sudden cardiac death. Until mavacamten's approval, adolescent patients relied on beta-blockers, calcium channel blockers, or, for severe obstruction, invasive septal reduction therapy (American Heart Association overview ↗).
Evidence: SCOUT-HCM trial
The approval was supported by the phase 3 SCOUT-HCM trial. Adolescents with symptomatic oHCM were randomised to mavacamten or placebo. At week 28, mavacamten significantly reduced Valsalva-provoked left ventricular outflow tract (LVOT) gradient — the primary endpoint — compared with placebo. Safety was consistent with the adult programme; REMS programme enrollment remains required due to the risk of systolic dysfunction (monitored by echocardiography). Full trial data are available via ClinicalTrials.gov ↗.
What this means for prescribers
Mavacamten is a REMS-restricted drug ↗. Prescribers must enrol patients and arrange regular echocardiography before initiation and during treatment to monitor for reduced ejection fraction. The Bristol Myers Squibb Camzyos HCP site ↗ provides dosing guidance and REMS enrolment information.
FAQ
What does FDA approval mean?
FDA approval means the agency has determined, based on scientific evidence submitted by the manufacturer, that a drug's benefits outweigh its known risks for a specific indication and patient population. The review process includes evaluation of clinical trial data, manufacturing quality, and proposed labelling. See the FDA drug approval process overview ↗.
Does approval mean the drug is available immediately?
Not always. Commercial launch timelines depend on manufacturing scale-up, pricing, and distribution. REMS-restricted drugs ↗ require prescriber or pharmacy enrolment before the product can be dispensed. Rare disease drugs may also have limited initial supply. Check the manufacturer's website or RxNav ↗ for availability status.