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Targeted Therapies in Hypertrophic Cardiomyopathy: A Practical Review of Evidence, Implementation, and Innovation
Madeline P Smoot1, James P MacNamara1, Michael P Ayers1,2
1Department of Medicine, University of Virginia School of Medicine, Charlottesville, VA, 22904, USA.
Journal of Cardiovascular Pharmacology
|July 6, 2026
Summary
Cardiac myosin inhibitors (CMIs) offer a targeted therapy for hypertrophic cardiomyopathy (HCM), improving outcomes in obstructive HCM. Further research is needed for non-obstructive HCM and broader disease modification.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Hypertrophic cardiomyopathy (HCM) is a common inherited cardiac disorder caused by sarcomere dysfunction.
- Current treatments for HCM manage symptoms but do not address the underlying molecular pathology.
- Hyperactive actin-myosin cross-bridge cycling is a key mechanism in HCM pathogenesis.
Purpose of the Study:
- To review the pharmacology, clinical evidence, and safety of cardiac myosin inhibitors (CMIs) for HCM.
- To explore the current role and limitations of CMIs in obstructive and non-obstructive HCM.
- To discuss emerging therapies for broader HCM phenotype modification.
Main Methods:
- Systematic review of pharmacology and clinical trial data for CMIs.
- Analysis of dosing strategies and safety profiles of CMIs.
- Evaluation of emerging therapeutic approaches for HCM.
Main Results:
- CMIs represent a novel class of targeted therapeutics for HCM.
- CMIs have shown efficacy in transforming the management of obstructive HCM.
- The efficacy of CMIs in non-obstructive HCM remains uncertain, indicating unmet needs.
Conclusions:
- CMIs are a significant advancement in treating obstructive HCM.
- Further investigation is required to establish the role of CMIs in non-obstructive HCM.
- Next-generation therapies hold promise for comprehensive HCM management across all phenotypes.
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