A patient from Rimini suffering from obstructive hypertrophic cardiomyopathy, due to a genetic mutation,
A patient from Rimini suffering from obstructive hypertrophic cardiomyopathy, due to a genetic mutation, was treated with a highly innovative drug for the first time in Italy in the Heart Failure and Cardiomyopathies Center of the Cardiology of Rimini, followed by doctors Silvia Amati, Samuela Carigi and Francesca Marzo.
The drug Mavacamten (Camzyos) is the first approved of a new class of drugs that reduce the activity of “cardiac myosin”, a protein fundamental for the contraction of heart cells, which plays a decisive role in creating obstruction of the left ventricular outflow in most patients with hypertrophic obstructive cardiomyopathy.
The positive efficacy and safety results of the Phase 3 EXPLORER-HCM (2020) and VALOR-HCM (2022) trials have allowed the approval of Mavacamten by the Federal Drug Administration (FDA), the European Medical Agency (EMA ) and subsequently by the Italian Medicines Agency (AIFA) for the treatment of adult patients with obstructive hypertrophic cardiomyopathy.
This treatment was possible in ASL Romagna thanks to the activation of the Compassionate Use Program by AIFA, for patients with an indication waiting for the drug to become reimbursable. In particular, the indication for Mavacamten is for adult patients (over 18 years of age) with hypertrophic obstructive cardiomyopathy, symptomatic (class II-III according to the New York Heart Association classification), who have exhausted or have not tolerated the alternatives available therapies.
Until now, the main treatment for these patients was represented by myectomy surgery, for which, however, the patient from Rimini had been excluded due to other pathologies.
Hypertrophic cardiomyopathy is not a rare disease, occurring in approximately 1 in 500 patients. Of these, almost 25% are obstructive.
The Heart Failure and Cardiomyopathy Center, which belongs to the Cardiology department directed by Dr. Filippo Ottani and belongs to the Emergency Department directed by Dr. Tiziana Perin, currently has hundreds of patients affected by hypertrophic cardiomyopathy in its care.
“In fact, the treatment of two other patients in the province of Rimini for whom the pharmacological therapies available so far have not been effective in controlling the disease is already planned – explain from the Heart Failure and Cardiomyopathies Center -. Probably the start of therapy will allow these patients to avoid surgery, considering the result achieved after a month in the patient under treatment and on the basis of the scientific data available so far”.
