Adnkronos Rome, November 17 (Adnkronos Salute) – "The approval of mavacamten for the treatment of hypertrophic cardiomyopathy represents a substantial change in clinical practice. For the first time, we have a drug capable of directly targeting the mechanisms underlying this complex disease, which can cause obstruction to blood flow from the left ventricle, difficulty relaxing the heart, arrhythmias, and mitral regurgitation."
This was stated to Adnkronos Salute by Gianfranco Sinagra, director of the Cardiovascular Department and president-elect of the Italian Society of Cardiology, during the announcement – today in Rome – of the approval by AIFA for 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.
Mavacamten, a member of the new class of myosin inhibitors, can significantly reduce the obstruction gradient. It does so, explains Sinagra, by improving the cardiac muscle's energy and metabolic efficiency, promoting better pumping function and a reduction in heart size. All of this translates into concrete benefits for patients: fewer symptoms, increased exercise capacity, and—most likely—a positive impact on the long-term progression of the disease. It should be noted that approximately 50% of patients with hypertrophic cardiomyopathy experience significant obstruction. However, with traditional therapies, only less than a third achieved a satisfactory clinical benefit. Myosin inhibitors, especially when added to standard treatment (so-called on-top), allow for rapid improvements in the gradient and symptoms. Furthermore, they will likely make it possible to reduce the doses of other medications in the future, with a positive impact on quality of life.
In the long term, "a stable reduction in obstruction can impact the size and function of the heart, the risk of arrhythmias, and therefore the likelihood of serious events," observes the SIC president-elect. "It should be remembered that hypertrophic cardiomyopathy is one of the leading causes of sudden death in young people. For this reason, it is particularly encouraging to think that these drugs can profoundly alter the biology of the myocardium and perhaps prevent the most advanced and dangerous forms of the disease."
In Italy, "it is estimated that approximately 100.000 people are affected by hypertrophic cardiomyopathy. Of these," Sinagra points out, "about half have clinically significant forms, with symptoms impacting their quality of life, and only a fraction respond adequately to traditional therapies. It is a rare disease, but it carries a significant burden because it often affects individuals between the ages of 20 and 40, a stage in life when they are most active, productive, and engaged in physical and professional activities. In these individuals, the disease can cause significant disability, in addition to the more hidden risks associated with its very nature."
Diagnosis requires a "high level of clinical attention: suspicion is crucial, especially in the presence of symptoms such as fatigue, palpitations, or reduced exercise tolerance. Available clinical studies have shown that drugs like mavacamten improve precisely these aspects: symptom severity, exercise capacity on cardiopulmonary testing, and some biomarkers that reflect the biology of the disease," he concludes.