Novel Myosin-Based Therapies in Hypertrophic Cardiomyopathy

AbstractPurpose of reviewHypertrophic cardiomyopathy is the most common genetic disease of myocardium and is recognized to have substantial clinical impact including sudden cardiac death, arrhythmias, and heart failure. Historically medical therapies have been used in hypertrophic cardiomyopathy (HCM) with limited prospective randomized data to support their effectiveness. In this review, we discuss a novel class of drug that inhibits myosin (myo-blocker) which was developed to precisely target the mechanism of HCM in detail.Recent findingsWe discuss and contextualize the clinical trial results for mavacamten, positioning it in the therapeutic landscape as well as presenting future questions and further developments to be made in this area.SummaryMany sarcomeric mutations driving hypertrophic cardiomyopathy seem to have a primary effect of causing hypercontractility which may drive many secondary problems such as structural remodeling, arrhythmias, and further contractile deficits. Myo-blockers such as the  first-in-class mavacamten are small molecules that specifically target myosin to decrease contractility and have been developed to precisely ameliorate the hypercontractile mechanism of HCM. Mavacamten has been shown in large, randomized, double-blind clinical trials to have benefit generally in i mproving symptoms, decreasing LV outflow tract obstruction, and promoting beneficial remodeling in HCM with obstructive physiology.
Source: Current Treatment Options in Cardiovascular Medicine - Category: Cardiology Source Type: research