Editorial: Hypertrophic Cardiomyopathy and Precision Medicine in Cardiovascular Disease

Molecular targets in diseased cells and tissues are the basis for precision medicine that stratifies patients into specific subgroups to develop targeted therapies. Myosin heavy chain (MHC) gene isoforms encode the proteins that form the thick filaments in muscle that enable muscle contraction. Management of familial hypertrophic cardiomyopathy has relied on surgical and symptomatic medical (pharmacological) treatment. However, in April 2022, the US Food and Drug Administration (FDA) approved a small molecule that selectively but reversibly inhibits sarcomeric myosin, mavacamten (MYK-461, Camzyos) for adults with symptomatic hypertrophic cardiomyopathy, New York Heart Association (NYHA) class II-III. Mavacamten is a first-in-class, precision medicine for patients with symptomatic hypertrophic obstructive cardiomyopathy that inhibits cardiac myosin and reduces actin-myosin interactions to reduce left ventricular outflow tract obstruction, improve exercise capacity, and reduce symptoms. This editorial aims to introduce how understanding the genetic and molecular basis of hypertrophic cardiomyopathy has resulted in a novel approach to precision medicine in cardiovascular disease.