The FDA has expanded the label for Bristol Myers Squibb’s Camzyos (mavacamten) to include adults and pediatric patients weighing at least 30 kg (66 lb) with symptomatic obstructive hypertrophic cardiomyopathy. The decision was supported by the Phase III SCOUT-HCM trial in 44 adolescents, in which Camzyos significantly reduced the Valsalva LVOT gradient at week 28. No LVEF values fell below 50%, no adverse-event discontinuations occurred, and serious adverse events were balanced between groups.
FDA Expands Camzyos Approval To Include Pediatric Patients With Obstructive HCM
Camzyos was initially approved by the FDA in 2022 for adult patients with symptomatic NYHA class II-III oHCM. Credit: NMK-Studio / Shutterstock.com.
The US Food and Drug Administration (FDA) has broadened the approved use of Bristol Myers Squibb’s Camzyos (mavacamten) to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and pediatric patients who weigh at least 30 kg (66 lb). The expanded indication notes the therapy is intended to improve functional capacity and relieve symptoms associated with oHCM.
The regulatory decision was supported by data from the double-blind, randomized Phase III SCOUT-HCM trial, which evaluated mavacamten versus placebo in 44 adolescent patients aged 12 to under 18 years. Camzyos met the trial’s primary endpoint, producing a statistically significant reduction in the Valsalva left ventricular outflow tract (LVOT) gradient at week 28 compared with placebo.
Safety findings in SCOUT-HCM were reassuring: no patients experienced a left ventricular ejection fraction (LVEF) below 50%, no adverse events led to treatment discontinuation, and serious adverse events were balanced between groups (two patients in the Camzyos arm [9%] and two in the placebo arm [10%]). Investigators reported no new safety signals beyond the established adult profile.
Al Reba, Senior Vice President for Immunology and Cardiovascular Commercialisation at Bristol Myers Squibb, said: "Today's approval of Camzyos for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy marks an important milestone for young patients and families facing a serious cardiovascular disease with substantial unmet need. For children and adolescents, disease symptoms can significantly affect daily life and development, and we are proud to offer an innovative new treatment option backed by robust clinical evidence."
Camzyos was first approved by the FDA in 2022 for adult patients with symptomatic New York Heart Association (NYHA) class II–III oHCM. Since that initial approval, more than 5,000 healthcare providers have prescribed Camzyos to over 25,000 patients in the United States. The medicine has also obtained regulatory clearances in more than 60 countries, supported by Phase III studies including EXPLORER-CN, HORIZON-HCM and SCOUT-HCM.
Bristol Myers Squibb said it is engaged in discussions with other international regulatory authorities regarding potential pediatric approvals. Separately, the company noted that in August 2026 the FDA granted accelerated approval for Zenbexus (iberdomide) in combination with daratumumab, hyaluronidase-fihj and dexamethasone (ZDd) for adults with multiple myeloma.
Originally published by Pharmaceutical Technology, a GlobalData-owned brand.
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