The US Food and Drug Administration (FDA) has approved Camzyos (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in patients weighing 30 kg (66 lbs) or more.
oHCM is a rare genetic heart condition characterized by excessive growth of heart muscle cells. The condition involves significant obstruction in the left ventricular outflow tract, leading to symptoms like dyspnea, fatigue, and potentially life-threatening cardiac events. Patients may also experience left ventricular hypertrophy, microvascular ischemia, myocardial fibrosis, and diastolic dysfunction. oHCM is often associated with genetic mutations in the MYH7 and MYBPC3 genes.
Mavacamten is a selective, reversible, allosteric cardiac myosin inhibitor designed to target hypercontractility, the source of oHCM.
The approval is based on positive results from the phase 3 SCOUT-HCM clinical trial (NCT06253221) evaluating the efficacy and safety of mavacamten (n=23) versus placebo (n=21) in patients ages 12 to 17 years with symptomatic NYHA (New York Heart Association) Class II-III obstructive hypertrophic cardiomyopathy.
Mavacamten met its primary endpoint, demonstrating a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at week 28 compared with placebo.
Additionally, no patients experienced left ventricular ejection fraction (LVEF) below 50% and no adverse events led to treatment discontinuation. No new adverse reactions were identified beyond the safety profile observed in adults. Serious adverse events occurred in 2 patients each in the mavacamten (9%) and placebo (10%) groups. Full results were published in The New England Journal of Medicine.
The full US Prescribing Information for Camzyos (mavacamten) includes a Boxed WARNING for the risk of heart failure.
To learn more about oHCM and other rare heart conditions, visit https://checkrare.com/diseases/heart-diseases/