FDA approves mavacamten for pediatric obstructive HCM patients

The U.S. Food and Drug Administration (FDA) has approved an expanded indication for mavacamten (Camzyos) to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in pediatric patients. The approval covers adolescents aged 12 to 18 years and children weighing at least 30 kg.

This decision marks the introduction of the first cardiac myosin inhibitor for this specific patient population. The approval is based on results from the phase 3 SCOUT-HCM trial, which demonstrated that patients treated with mavacamten experienced a significant reduction in the Valsalva-provoked left ventricular outflow tract (LVOT) gradient compared to a placebo.

Clinical data indicated that the safety profile in adolescents was consistent with previous adult studies, with no patients experiencing a reduction in left ventricular ejection fraction below 50%. Bristol Myers Squibb is currently discussing the pediatric data with various global regulatory authorities.