Mavacamten—A Targeted Therapy for Hypertrophic Cardiomyopathy

Abstract

The pathophysiology of hypertrophic cardiomyopathy is primarily comprised of dynamic left ventricular outflow tract obstruction, mitral regurgitation, and diastolic dysfunction. Symptoms such as dyspnea, angina, or syncope can occur because of left ventricular (LV) hypertrophy and reduced LV cavity size. Currently, focus on symptom relief through optimizing LV preload and reducing inotropy is the mainstay of therapy through the use of β-blockers, nondihydropyridine calcium channel blockers, and disopyramide. Mavacamten is a novel cardiac myosin inhibitor recently approved by the Food and Drug Administration for the treatment of obstructive hypertrophic cardiomyopathy. Mavacamten normalizes myosin and actin cross-bridging to decrease contractility and ultimately reduce LV outflow tract gradients to maximize cardiac output. In this review, we report on the mechanism of action of mavacamten, safety profile, and phase 2 and 3 clinical trial data. Because of the risk of heart failure resulting from systolic dysfunction, careful patient selection and close monitoring are key for implementing this therapy into cardiovascular practice.