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Updated: Jun 25, 2025
Investigating the Pathogenesis of MYH7 Mutation Gly823Glu in Familial Hypertrophic Cardiomyopathy using a Mouse Model
Published on: August 8, 2022
Promising therapies for adults with symptomatic obstructive hypertrophic cardiomyopathy: 2023 and beyond
Andrew Gaballa1, Shada Jadam1, Milind Y Desai1
1Hypertrophic Cardiomyopathy Center, Cleveland Clinic, Cleveland, OH, USA.
Insights
Hypertrophic cardiomyopathy (HCM) is a genetic heart condition. Cardiac myosin inhibitors like mavacamten offer a targeted therapy for HCM, improving symptom management in persistent cases.
Area of Science:
- Cardiology
- Genetics
- Pharmacology
Background:
- Hypertrophic cardiomyopathy (HCM) is a prevalent genetic heart disease affecting 0.2%–0.6% of the population.
- HCM presents a spectrum of symptoms, from asymptomatic to severe heart failure and sudden cardiac death.
- Current management includes lifestyle changes, screening, counseling, pharmacotherapy, and advanced interventions like transplantation.
Purpose of the Study:
- To review existing and emerging pharmacotherapies for hypertrophic cardiomyopathy (HCM).
- To discuss the role of cardiac myosin inhibitors (CMIs) in HCM management.
- To identify unmet needs and future directions in HCM pharmacotherapy.
Main Methods:
- Literature review of current HCM treatments including beta-blockers, calcium channel blockers, disopyramide, and CMIs.
- Analysis of recent clinical trial data on CMIs, specifically mavacamten.
- Discussion of clinical guidelines and future research needs.
Main Results:
- Mavacamten is the first FDA-approved CMI targeting HCM pathophysiology.
- Mavacamten is indicated for obstructive HCM (oHCM) with persistent symptoms despite maximal therapy.
- Further research is required for CMIs in patients with renal/hepatic impairment and in pregnant/breastfeeding populations.
Conclusions:
- Cardiac myosin inhibitors represent a significant advancement in targeted HCM therapy.
- Mavacamten should be integrated into standard care for symptomatic oHCM.
- Addressing drug interactions and specific patient populations is crucial for optimizing CMI use.
Introduction:
Hypertrophic cardiomyopathy (HCM) is a heterogeneous genetic heart disease with an estimated prevalence in the general population of 0.2% to 0.6%. Clinically, HCM can range from no symptoms to severe symptoms such as heart failure or sudden cardiac death. Currently, the management of HCM involves lifestyle modifications, familial screening, genetic counseling, pharmacotherapy to manage symptoms, sudden cardiac death risk assessment, septal reduction therapy, and heart transplantation for specific patients. Multicenter randomized controlled trials have only recently explored the potential of cardiac myosin inhibitors (CMIs) such as mavacamten as a directed pharmacological approach for managing HCM.
Areas Covered:
We will assess the existing medical treatments for HCM: beta-blockers, calcium channel blockers, disopyramide, and different CMIs. We will also discuss future HCM pharmacotherapy guidelines and underline this patient population's unfulfilled needs.
Expert Opinion:
Mavacamten is the first-in-class CMI approved by the FDA to target HCM pathophysiology specifically. Mavacamten should be incorporated into the standard therapy for oHCM in case of symptom persistence despite using maximally tolerated beta blockers and/or calcium channel blockers. Potential drug-drug interactions should be assessed before initiating this drug. More studies are needed on the use of CMIs in patients with kidney and/or liver failure and pregnant/breastfeeding patients.
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