Mavacamten represents a paradigm shift toward mechanism-based precision therapeutics for symptomatic hypertrophic obstructive cardiomyopathy, though its use requires strict LVEF monitoring.
Mavacamten may benefit refractory HOCM; hypothesis-generating and should not yet change practice.
Mavacamten is a first-in-class, selective allosteric inhibitor of cardiac myosin adenosine triphosphatase (ATPase) that has emerged as a novel therapeutic option for patients with symptomatic hypertrophic obstructive cardiomyopathy (HOCM) who remain refractory to conventional therapy, such as beta-blockers and calcium channel blockers. Clinical trials have demonstrated that mavacamten reduces left ventricular outflow tract (LVOT) obstruction, improves diastolic function, and enhances exercise capacity, thereby addressing the key pathophysiological mechanisms underlying HOCM. However, its use requires careful dose titration due to the potential for reversible reductions in left ventricular ejection fraction (LVEF). While mavacamten represents a meaningful therapeutic advancement in HOCM management, its role in other conditions such as non-obstructive hypertrophic cardiomyopathy (HCM) and heart failure with preserved ejection fraction (HFpEF) remains investigational. Preliminary data suggest favorable effects on diastolic parameters and cardiac biomarkers in HFpEF, but larger studies are needed to establish efficacy. While these challenges persist, ongoing research is exploring broader cardiomyopathy populations, optimization of combination therapies, and development of novel formulations. As a targeted modulator of myocardial contractility, mavacamten exemplifies the shift toward mechanism-based, precision therapeutics in cardiovascular medicine.
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Ramadan et al. (2025) studied this question.