Medical therapies for hypertrophic cardiomyopathy: Current state of the art

Hypertrophic cardiomyopathy (HCM) is predominantly an autosomal dominant genetic heart disease with an estimated prevalence of 1 in 200 to 1 in 500 in the general population. Clinical manifestations of HCM vary from asymptomatic state to mild functional intolerance to advanced heart failure, angina, and sudden cardiac death (SCD). Current management options for symptomatic HCM include lifestyle modifications, pharmacotherapy for symptom control and arrhythmia management, SCD risk stratification with or without defibrillator implantation, septal reduction therapy and, in some cases, heart transplantation. Until recently, none of the pharmacotherapies for management of HCM had been studied in multicenter randomized controlled trials. Mavacamten, a cardiac myosin inhibitor, is the first drug studied in this fashion and the first-in-class Food and Drug Administration approved medication that specifically targets the pathophysiology of HCM. We will review the currently available medical treatments for HCM and assess future directions.