Promising therapies for adults with symptomatic obstructive hypertrophic cardiomyopathy: 2023 and beyond

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.