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A Systematic Review of Clinical Trials on Mavacamten in Hypertrophic Cardiomyopathy

Pavan Kumar Reddy Kalluru1, Sowmya Manjari Siddenthi2, Sai Sudha Valisekka3

  • 1Division of Cardiology, West Anaheim Medical Center, Anaheim, CA, USA.

Heart International
|July 25, 2025
PubMed

Insights

Mavacamten effectively treats symptomatic obstructive hypertrophic cardiomyopathy (oHCM) by reducing left ventricular outflow tract gradients and improving patient metrics. This cardiac myosin inhibitor offers a new non-invasive therapy for oHCM patients.

Area of Science:

  • Cardiology
  • Pharmacology
  • Medical Therapeutics

Background:

  • Hypertrophic cardiomyopathy (HCM) involves interventricular septum thickening, leading to outflow tract obstruction, mitral regurgitation, diastolic dysfunction, and arrhythmias.
  • Mavacamten is a novel, selective allosteric modulator of cardiac myosin, approved by the FDA for symptomatic obstructive HCM (oHCM).

Purpose of the Study:

  • To systematically review and synthesize clinical trial data on the efficacy and safety of mavacamten in treating obstructive hypertrophic cardiomyopathy (oHCM).
  • To assess mavacamten's impact on hemodynamic parameters, functional status, quality of life, and relevant biomarkers in oHCM patients.

Main Methods:

  • A systematic literature search was performed on Medline/PubMed and ClinicalTrials.gov.
  • Search terms included 'mavacamten/MYK-461' and 'hypertrophic cardiomyopathy/HCM'.
  • All published clinical trials to date were identified and included.

Main Results:

  • Mavacamten demonstrated consistent efficacy across multiple trials (PIONEER-HCM, EXPLORER-HCM, VALOR-HCM, etc.), significantly reducing left ventricular outflow tract gradients.
  • Improvements were observed in New York Heart Association functional class, oxygen uptake (pVO2), and quality-of-life metrics, with significant reductions in biomarkers like NT-proBNP.
  • The VALOR trial indicated a reduced need for septal reduction therapy; mavacamten was generally well-tolerated, though systolic dysfunction requires monitoring.

Conclusions:

  • Mavacamten represents a promising non-invasive pharmacological treatment for symptomatic oHCM.
  • It is particularly beneficial for patients unsuitable for or unresponsive to conventional therapies.
Abstract

Keywords:
Diastolic dysfunctionhypertrophic cardiomyopathyinterventionalleft ventricular outflow tract gradientsmavacamtenmyosin heavy chainsquality of lifetreatment outcome

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