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Current Cardiology Reports

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

  • Open Access
  • 01-12-2025
  • Cardiomyopathy
  • Myocardial Disease (A Abbate and M Merlo, Section Editors)
Published in:
Authors
Giorgia Panichella
Manuel Garofalo
Maddalena Ragagnin
Angela Ilaria Fanizzi
Mattia Zampieri
Annamaria Del Franco
Francesco Cappelli
Maurizio Pieroni
Iacopo Olivotto
Published in
Current Cardiology Reports | Issue 1/2025

Abstract

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.

Summary

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.
Title
Mavacamten in the Treatment of Obstructive Hypertrophic Cardiomyopathy: from Pathophysiology To Real World Data
Authors
Giorgia Panichella
Manuel Garofalo
Maddalena Ragagnin
Angela Ilaria Fanizzi
Mattia Zampieri
Annamaria Del Franco
Francesco Cappelli
Maurizio Pieroni
Iacopo Olivotto
Publication date
01-12-2025
Publisher
Springer US
Published in
Current Cardiology Reports / Issue 1/2025
Print ISSN: 1523-3782
Electronic ISSN: 1534-3170
DOI
https://doi.org/10.1007/s11886-025-02316-6
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This content is intended for healthcare professionals outside of the UK.

Independent Medical Education Grant:
  • Bayer HealthCare Pharmaceuticals Inc.
Learn more Link opens in a new window
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