Advances in the management of obstructive hypertrophic cardiomyopathy: The role of mavacamten.

Abstract

Introduction: Hypertrophic cardiomyopathy (HCM) is a common inherited cardiovascular disorder characterized by asymmetric left ventricular hypertrophy and increased risk of sudden cardiac death. While current treatments like beta-blockers, calcium channel blockers, and invasive procedures provide symptom relief, they do not prevent disease progression. Mavacamten, a first-in-class cardiac myosin inhibitor, offers a promising alternative by directly targeting the underlying cause of left ventricular outflow tract (LVOT) obstruction in obstructive HCM. This drug reduces myosin ATPase activity, decreasing myocardial contractility and alleviating symptoms such as shortness of breath, fatigue, and chest pain. Clinical trials, including EXPLORER-HCM and VALOR-HCM, have demonstrated significant improvements in LVOT obstruction, exercise capacity, and overall quality of life while reducing the need for invasive procedures.

Areas covered: This review discusses mavacamten's mechanism of action, pharmacokinetics, clinical efficacy, safety, and integration into clinical practice, highlighting its role as a key treatment for patients who do not respond to conventional therapies.

Expert opinion: Mavacamten is generally well tolerated, although it requires careful monitoring of left ventricular ejection fraction (LVEF) and genetic factors such as CYP2C19 variations. The drug represents a significant advancement in the management of symptomatic obstructive HCM, offering a noninvasive, disease-modifying option.