Exploring New and Old Therapies for Obstructive Hypertrophic Cardiomyopathy

he application of contemporary treatment strategies to hypertrophic cardiomyopathy (HCM) has transformed this common genetic heart disease from one of grim progression to one now compatible with normal longevity and excellent quality of life. Mavacamten, a myosin inhibitor, represents the first pharmacological strategy advanced for HCM in nearly 35 years, culminating recently with publication of a phase 3 randomized clinical trial (EXPLORER-HCM Mavacamten for Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy). 1 Given the enthusiasm surrounding this novel drug, even before Food and Drug Administration approval, it is timely to provide perspective on the potential role for mavacamten, particularly compared with the current established therapies for patients with obstructive HCM.