The FDA has approved Camzyos (mavacamten) for pediatric patients weighing at least 30 kilograms, or about 66 pounds, who have symptomatic obstructive hypertrophic cardiomyopathy. The agency described it as the first approved drug intended to improve functional capacity and symptoms in children with the inherited heart condition. Camzyos was first approved for adults in 2022.
Obstructive hypertrophic cardiomyopathy causes the heart muscle to thicken, potentially narrowing the passage through which blood leaves the left side of the heart. Symptoms can include shortness of breath and fatigue; patients may also develop abnormal heart rhythms.
The pediatric decision was based on SCOUT-HCM, a Phase 3 trial involving 44 patients aged 12 to 18. Participants received Camzyos or a placebo once daily for 28 weeks. The main measure was the change in pressure across the narrowed passage when patients performed a bearing-down maneuver.
Camzyos reduced that pressure measure significantly more than placebo, indicating less obstruction to blood flow. In adult studies, reduced obstruction was accompanied by improvements in functional capacity and symptoms. On the basis of the pediatric trial and adult findings, the FDA expects Camzyos to improve those outcomes in eligible pediatric patients.
Camzyos carries a boxed warning for heart failure. It is available only through a restricted distribution program, the Camzyos Risk Evaluation and Mitigation Strategy, intended to manage that risk.
The expanded approval gives clinicians a drug treatment for eligible children with symptomatic obstruction. The pediatric trial measured the drug’s effect on obstruction over 28 weeks, while the approval addresses expected improvements in patients’ ability to function and their symptoms.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox!
Sign up now!