FDA expands BMS CAMZYOS (mavacamten) to pediatric obstructive HCM (≥30 kg)

On 1 October 2026, Bristol Myers Squibb announced that the U.S. FDA approved an expanded indication for CAMZYOS® (mavacamten) to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) and improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more. The company said CAMZYOS is the only FDA-approved cardiac myosin inhibitor therapy for oHCM in a pediatric population under this labeling.

SCOUT-HCM evidence

Approval rests on the Phase 3 SCOUT-HCM trial (NCT06253221): a randomized, double-blind, placebo-controlled study in 44 adolescents aged 12 to <18 years with symptomatic NYHA Class II–III oHCM (CAMZYOS n=23, placebo n=21). CAMZYOS met the primary endpoint with a statistically significant reduction in Valsalva left-ventricular outflow-tract (LVOT) gradient at Week 28 versus placebo (least-squares mean difference about −48.0 mmHg, 95% CI −67.7 to −28.3, p<0.0001). No patient had LVEF below 50%; no adverse events led to discontinuation; serious adverse events occurred in 2 patients in each arm (9% vs 10%). No new adverse reactions beyond the adult safety profile were identified, the company said.

Safety context — read carefully

U.S. prescribing information includes a Boxed WARNING for risk of heart failure due to systolic dysfunction; echocardiogram monitoring of LVEF is required, and CAMZYOS is available only through a REMS program. Concomitant use with certain CYP inhibitors/inducers is contraindicated. This article summarises a company press release about a Phase 3 pediatric expansion; it is not personal medical advice. Treatment decisions belong with a qualified cardiologist familiar with REMS requirements.