Mavacamten: A Novel Disease-Specific Treatment for Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy (HCM) is characterized by an abnormal thickening of the myocardium, leading to left ventricular outflow tract obstruction. Current treatments for HCM include non-disease-specific medications such as beta blockers or invasive interventions. Mavacamten has been studied for its effects on adenosine triphosphatase, myocardial-specific sarcomeric proteins, and myocardial tissue calcium sensitivity. Given these properties, mavacamten could be used as a disease-specific treatment for HCM. Clinical trials of mavacamten have shown improvements in left ventricular outflow tract obstruction among other favorable improvements in biochemical markers and the clinical symptoms of the disease. While trials to date have been relatively small, mavacamten shows promise as a future disease-specific treatment for HCM.