Hypertrophic Cardiomyopathy, Therapy Works Long-Term
Further confirmation of the efficacy and safety of mavacantem, the first (and so far only) drug that acts on the cause of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The long-term data just presented at the Congress of the European Society of Cardiology (ESC) in London attest to constant improvements for adult patients even after 3.5 years of continuous treatment. With the inclusion in the ESC and AHA/ACC guidelines as a recommended option when symptoms persist after first-line therapy, mavacamten is a standard of care for symptomatic oHCM.
The disease
Hypertrophic obstructive cardiomyopathy is a rare heart disease. It is thought to affect over 100,000 people in Italy alone and in 50-60% of cases it is hereditary. The cause, in fact, is a genetic mutation, responsible for both the abnormal arrangement and functioning of the heart muscle fibers and hypertrophy, that is, the progressive thickening of the heart muscle. The consequences are shortness of breath, loss of consciousness, dizziness and fatigue, up to heart failure, arrhythmias, stroke. The disease can also be a cause of sudden death among young athletes.
Efficacy and safety confirmed
Mavacamtem is the first and only drug approved in the United States and the European Union for the treatment of adult patients that targets the molecular cause of the disease. It has been in use for several years and continues to demonstrate safety and efficacy, so much so that at the latest update of long-term data, the majority of patients maintained consistent improvements in cardiac parameters measured by echocardiography and biomarkers. “The consistent and sustained improvements in cardiac parameters over three years with mavacamten demonstrate that this therapy fills an important treatment need for patients with symptomatic obstructive HCM,” said Pablo Garcia-PaviaHead of the Hereditary Heart Diseases and Heart Failure Unit at the Department of Cardiology, Puerta de Hierro University Hospital and Professor at the Spanish Institute of Cardiovascular Research in Madrid: “These positive long-term data, together with the inclusion of mavacamten in the ESC clinical guidelines for obstructive HCM, underscore the important role of this drug in the long-term care of this lifelong condition that requires ongoing management.” The analysis of the Explorer-Lte study revealed no new adverse effects in treatment with mavacamten, further strengthening the established safety and efficacy profile and redefining the treatment landscape for this patient population.
