Mavacamten is the first available cardiac myosin inhibitor that specifically addresses the pathophysiological mechanisms of obstructive hypertrophic cardiomyopathy (HCM). This innovative and personalized therapy offers a new therapeutic target that was previously unattainable, with the potential to prevent invasive procedures in the treatment of this condition.
Hypertrophic cardiomyopathy is the most common congenital cardiomyopathy, with a prevalence of 1 case per 500 individuals. It is characterized by abnormal thickening of the left ventricle, which can hinder pumping function and cause severe symptoms, including arrhythmias and sudden cardiac death.
Mavacamten, a cardiac myosin inhibitor, improves disease symptoms and provides a non-invasive alternative to traditional treatments like surgical myectomy or alcohol septal ablation. It targets the specific mechanisms of the disease, reducing the risk of complications.
Based on clinical evidence from various trials, CatSalut authorized the Bellvitge University Hospital (HUB) last November to prescribe Mavacamten as a provisional treatment for obstructive cardiomyopathy in a patient with symptoms refractory to first-line treatment. This patient became the first in Catalonia to be treated with a myosin inhibitor.
The drug acts on the fundamental mechanism of the disease: excessive cardiac contractile force, which triggers the cascade of symptoms and complications that can be severe in one-third of patients. According to follow-up evaluations, the patient has tolerated the drug well, and symptoms have improved favourably.
The opportunity to initiate the first treatment with Mavacamten has enabled the establishment of a specialized multidisciplinary and territorial reference clinic dedicated to managing this high-complexity condition. This initiative is spearheaded by the Hereditary Cardiovascular Diseases Unit of the Cardiology Service, in collaboration with the hospital's Pharmacy Service. The goal is to provide a clinical framework for patients eligible for these new molecules, offering a comprehensive assessment to ensure safe and effective disease management.
According to Dr. Carles Díez, head of the Hereditary Cardiovascular Diseases Unit: “Thanks to the collaboration with Nursing, Pharmacy, and the Molecular Genetics team of the Biochemistry Service at HUB, we have been able to develop this project with myosin inhibitors, addressing a critical medical need and providing an innovative and comprehensive approach to patients with this condition.”
Currently, three additional patients are receiving this treatment. The HUB plans to progressively expand the therapy to new patients with hypertrophic cardiomyopathy, establishing itself as a reference centre for managing high-complexity conditions with specificity and therapeutic innovation.
The personalization of treatment involves adjusting medication doses according to each patient's specific characteristics, such as age, sex, weight, medical history, genetic factors, and concomitant medication. This approach places the patient at the centre of care, considering their individual response to the drug and potential interactions with other treatments or medical conditions.
Rigorous monitoring is essential to ensure the treatment's efficacy and safety. This includes periodic evaluations with clinical tests, laboratory analyses, and advanced imaging studies, enabling proactive treatment adjustments, early detection of adverse effects, and maximization of clinical benefits.