Hypertrophic cardiomyopathy: investigational drugs inhibiting myosin and upcoming agents

INTRODUCTION: Hypertrophic cardiomyopathy (HCM), a phenotypically variable disorder with a genetic basis, was first described in the late 1800s. Since the discovery of the disease, various medical and surgical treatments have been proposed with surgical treatments proving to be of more benefit than medical in patients with severe symptoms. Although beta blockers, calcium channel blockers, and disopyramide have been used for their negative inotropic effect, the data behind utilization of these medications is weak at best. AREAS COVERED: Herein, we describe a first-in-man class of medications called cardiac myosin inhibitors (CMI), which have been recently approved by the Food and Drug Administration (FDA) for the treatment of symptomatic patients with obstructive HCM. PubMed was the primary database searched. EXPERT OPINION: Whether these medications will stand the test of time remains to be seen. They do appear to provide significant benefit and disease modification in early randomized trials with the drawback of decreasing contractility and ejection fraction. In our opinion, this new class of medications is an option for patients with NYHA class II-III symptoms from obstructive HCM who have EF ≥ 55%.