A Breakthrough in the Treatment of Hypertrophic Cardiomyopathy

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Maka Gegenava, MD, PhD Assistant Professor, Department of Internal Medicine #2, Tbilisi State Medical University

At the 2025 Congress of the European Society of Cardiology (ESC), I presented a report on the effect of mavacamten (a myosin inhibitor) on diastolic dysfunction and various Doppler-echocardiography parameters in patients with obstructive hypertrophic cardiomyopathy (oHCM). I performed the research in the Cardiomyopathy Department at the University of California, San Francisco (UCSF), under the supervision of the President of the American Society of Echocardiography.

Hypertrophic cardiomyopathy is a genetic, autosomal dominant heart disease characterized by left ventricular hypertrophy. The main pathophysiological features of the disease are hypercontractility, diastolic dysfunction, and dynamic obstruction of the left ventricular outflow tract. The main clinical manifestations are atrial fibrillation, heart failure, and malignant ventricular arrhythmias.

The diagnosis and management of the disease require the involvement of a highly qualified, multidisciplinary team. The diagnostic process includes a patient examination, genetic testing, and a wide range of laboratory and instrumental studies.

Currently, the main treatments are beta-blockers, calcium channel blockers (mainly verapamil or diltiazem), and disopyramide. Invasive interventions include septal reduction therapies—alcohol septal ablation, myectomy, and, if necessary, an ICD (implantable cardioverter-defibrillator) is also used.

At the ESC 2025 Congress, several promising methods of gene therapy for hypertrophic cardiomyopathy were presented as a novelty, which are currently in the research phase.

As for mavacamten, it is a new-generation, first-class myosin inhibitor recommended by both American and European guidelines for patients with obstructive hypertrophic cardiomyopathy (oHCM). The drug is used in patients who have symptoms of heart failure (NYHA Class II or III) and aims to improve cardiac functional capacity and alleviate symptoms.

The use of mavacamten in non-obstructive HCM is not currently recommended.

As far as I know, mavacamten is not yet available in our country. Unfortunately, the cost of the drug is quite high, and its administration requires constant patient monitoring to prevent and manage side effects, which necessitates expensive laboratory and instrumental studies.

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