Mavacamten in the Treatment of Obstructive Hypertrophic Cardiomyopathy: from Pathophysiology To Real World Data.

PURPOSE OF REVIEW: This review aims to provide a comprehensive overview of mavacamten in the management of obstructive hypertrophic cardiomyopathy (oHCM), from its molecular mechanism of action to clinical trial outcomes and real-world application. It explores the efficacy, safety, and practical considerations of mavacamten use, while highlighting evolving indications and future directions.

RECENT FINDINGS: Randomized trials have shown that mavacamten significantly reduces left ventricular outflow tract gradients, improves symptoms and exercise capacity, and induces structural reverse remodeling with a favorable safety profile. Real-world data confirm these benefits in broader patient populations. Pharmacogenetic variability, titration protocols, and cost-effectiveness analyses have further refined its clinical use. Mavacamten represents a paradigm shift in oHCM treatment by targeting disease at its sarcomeric origin. Its integration into routine care is expanding, supported by real-world evidence. Ongoing studies will clarify its role in non-obstructive HCM, heart failure with preserved ejection fraction, pediatric populations, and early-stage disease.