Bristol Myers Squibb secures expanded US approval for Camzyos
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The FDA has expanded the indication for Camzyos (mavacamten) to include pediatric patients weighing at least 30kg with symptomatic obstructive hypertrophic cardiomyopathy.
The US Food and Drug Administration (FDA) has expanded the approved indication for Bristol Myers Squibb’s (BMS) Camzyos (mavacamten) to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and paediatric patients weighing at least 30kg (66lb).
The therapy is indicated to improve functional capacity and symptoms.
The regulatory decision was supported by findings from the double-blind, randomised Phase III SCOUT-HCM trial, which assessed the cardiac myosin inhibitor (CMI) against a placebo in 44 adolescent patients aged 12 to under 18 years.
Camzyos achieved the primary endpoint by producing a statistically significant reduction in the Valsalva left ventricular outflow tract (LVOT) gradient at week 28 compared with placebo.
During the study, no patients recorded a left ventricular ejection fraction (LVEF) below 50%, and no adverse events led to treatment discontinuation.
Serious adverse events occurred in two patients in the Camzyos cohort (9%) and two in the placebo group (10%), with no new safety concerns identified beyond the known adult profile.
Camzyos was initially approved by the FDA in 2022 for adult patients with symptomatic NYHA class II-III oHCM, with more than 5,000 healthcare providers prescribing it to over 25,000 US patients to date. The therapy has gained regulatory clearances across more than 60 countries, supported by Phase III trials conducted in China, Japan, and other international regions, including EXPLORER-CN, HORIZON-HCM, and SCOUT-HCM.
The company is in discussions with other international regulatory authorities regarding potential paediatric approvals. In August 2026, the FDA granted accelerated approval to BMS’ Zenbexus (iberdomide), along with daratumumab and hyaluronidase-fihj and dexamethasone (ZDd), to treat adult multiple myeloma patients.
