Mavacamten: Hope on the Horizon

Banya Aung Myint
21/01/2025

Take home messages

  • Mavacamten was recommended by National Institute for Health and Care Excellence (NICE) in September 2023 as a treatment option for NYHA class II-III symptomatic obstructive hypertrophic cardiomyopathy despite optimal medical therapy.
  • Mavacamten is well tolerated and provides sustained improvement of symptoms and LVOT gradient reduction in obstructive HCM.
  • Mavacamten is not recommended for patients with a left ventricular ejection fraction (LVEF) <55%, as it may reduce LVEF and result in heart failure due to systolic dysfunction.

Introduction

Mavacamten is the first-in-class, cardiac-specific myosin inhibitor that targets myosin, a protein central to the pathophysiology of obstructive hypertrophic cardiomyopathy (HCM). By reversibly binding to myosin molecules (myosin ATPase), Mavacamten reduces the hypercontractile state of the cardiac muscle by lowering the probability of myosin being in an active state and thereby mitigating associated adverse metabolic effects1,2 .  The United State Food and Drug Administration (FDA) approved Mavacamten in April 2022, following results from the pivotal EXPLORER-HCM trial.10