Adnkronos Rome, November 17 (Adnkronos Salute) – "Mavacamten was developed for the treatment of symptomatic hypertrophic obstructive cardiomyopathy, and in Italy it will be reimbursed for this indication. The drug acts at the molecular level in a highly targeted way: it targets the mechanisms that, in these genetic forms of cardiomyopathy, determine left ventricular outflow tract obstruction. By reducing this obstruction—which is the main cause of symptoms—the drug significantly improves patients' quality of life.
This data emerges from both clinical studies and our direct experience. Mavacamten increases exercise capacity, significantly reduces cardiac hypertrophy, and also improves the profile of biomarkers that measure the state of cardiac compensation." This is what Iacopo Olivotto, Full Professor of Cardiology and Director of Pediatric Cardiology at the Meyer Children's Hospital in Florence and Director of the Cardiomyopathy Service at the Careggi Children's Hospital in Florence, told Adnkronos Salute during the announcement – today in Rome – of AIFA's approval of the reimbursement of mavacamten for the treatment of symptomatic hypertrophic obstructive cardiomyopathy (HOC) (class II-III according to the NYHA classification) in adult patients in whom standard therapy is insufficient.
Hypertrophic obstructive cardiomyopathy is a genetic disease that can appear at any age: it is typically diagnosed between the ages of 30 and 50, but can also affect children and the elderly. In this condition, Olivotto explains, the heart is abnormally thickened, and in addition to the hypertrophy, there is an obstruction that impedes the flow of blood from the left ventricle. The result is that the heart must work at very high pressures, somewhat as if there were a narrowed valve, even though the obstruction is actually beneath the valve. This mechanism can promote even serious arrhythmias and, above all, causes significant functional limitations, which significantly impact daily, social, and work life, especially in younger and more active patients.
The overall prognosis is generally good, but the disease carries significant morbidity: episodes of atrial fibrillation, hospitalizations, the need for cardiac devices, and an often compromised quality of life. Until now, it was, in effect, an 'orphan' disease: the therapeutic tools available were limited and, in many cases, insufficient. Mavacamten finally appears to fill this gap. The clinical response, in most patients, is significantly superior to traditional treatments," he concludes.