FDA approves mavacamten for adolescents with obstructive HCM

The U.S. Food and Drug Administration (FDA) has approved mavacamten (Camzyos) for use in adolescents aged 12 to younger than 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). This expansion of the drug's indication follows its previous approval for adults with NYHA class II or III oHCM.

Mavacamten is the first cardiac myosin inhibitor approved for this adolescent population. The decision was supported by results from the phase 3 SCOUT-HCM trial, which evaluated the efficacy and safety of the treatment in adolescents. The trial demonstrated that mavacamten significantly reduced the Valsalva-provoked left ventricular outflow tract (LVOT) gradient compared to a placebo at 28 weeks.

Obstructive hypertrophic cardiomyopathy is a cardiac disorder involving abnormal thickening of the heart muscle that can obstruct blood flow from the left ventricle. In adolescents, the condition often leads to symptoms such as exercise intolerance, dyspnea, chest discomfort, and fatigue.