A breakthrough in the treatment of hypertrophic cardiomyopathy in Poland – reimbursement of the first causal therapy
Since July 1, 2019, mavacamten, the first causal drug in the treatment of hypertrophic cardiomyopathy, has been included on the list of reimbursed drugs. Representatives of patient organizations and doctors expressed their satisfaction that this innovative and groundbreaking therapy will be available to Polish patients.
– The reimbursement of mavacamten is great news for patients with hypertrophic cardiomyopathy and their families. This is the first drug that directly affects the cause of the disease, and not only alleviates its symptoms. Thanks to this therapy, patients can count on a significant improvement in the quality of life and avoid invasive surgical procedures, as well as return to work or physical activity. I am pleased and grateful to the Ministry of Health for its support and positive response to the voices of patient organizations. and experts for access to this breakthrough therapy for Polish patients – said Agnieszka Wołczenko, president of the Polish Association of Patients with Heart and Vascular Diseases “EcoSerce”.
Mavakamten was registered by the European Commission in June 2023. The basis for the registration of the drug were the results of the Explorer-HCM and VALOR-HCM studies, in which Polish patients also participated. The studies showed that in patients with symptoms of hypertrophic cardiomyopathy, such as: shortness of breath, chest tightness, shortness of breath, fatigue, low heart function, who were treated with mavakamten, a significant reduction in these symptoms, improvement in physical capacity and improvement in the quality of life were observed. EXPLORER-HCM clinical trials and VALOR-HCM also showed that mavacamten lowers NYHA(i) classes in patients with oHCM, both as add-on therapy to standard treatment and in patients eligible for surgery.
– Mawakamten is a representative of a completely new class of drugs – cardiac myosin inhibitors. Its action is based on inhibiting pathological contraction of the heart muscle and increasing the number of muscle fibers in a low-energy relaxed state. This improves the energetics of muscle fibers. Explorer-HCM and VALOR-HCM studies have shown that the drug leads to a reduction of the gradient in the outflow tract, improves physical performance, reduces symptoms, improves quality of life and causes a reduction in NT-proBNP concentration, which is an objective indicator of heart overload. There is also a reduction in the mass of the left ventricular muscle. In addition, the condition of some patients who would require surgery improves to such an extent that they no longer need these procedures – said prof. dr hab. n. med. Wojciech Wojakowski, head of the 3rd Cardiology Clinic of the Upper Silesian Medical Center Hospital in Ochojec, coordinator of the Explorer HCM study in Poland(ii).
It is worth emphasizing that mawakamten was included in the European Society of Cardiology (ESC) 2023 guidelines as a recommended therapy for patients with hypertrophic cardiomyopathy. The drug’s reimbursement was among priorities of the “Decalogue of Polish cardiology for 2023–2025” developed by the Polish Cardiac Society.
What is hypertrophic obstructive cardiomyopathy?
Obstructive hypertrophic cardiomyopathy (oHCM) is characterized by thickening of the heart muscle, most often the left ventricle. It leads to impaired diastolic function of the thickened ventricle and, consequently, heart failureand even death of the patient. The most common symptoms of the disease are: chronic fatigue, exertional dyspnea (exertion intolerance), heart palpitations.
Currently, in the treatment of hypertrophic obstructive cardiomyopathy symptomatic drugs are used (beta-blockers and calcium channel blockers), and in a small group of patients with higher NYHA classes III-IV, surgical treatment or alcohol ablation of the interventricular septum is used.
In Poland, approximately 1,300 patients suffer from hypertrophic obstructive cardiomyopathy.(iii), however, this population is probably underestimated. Polish data indicate that over 30% of patients with hypertrophic cardiomyopathy die within 5 years of diagnosis.
(I) NYHA scale – proposed by the New York Heart Association (NYHA) 4-level scale used to classify the severity of heart failure symptoms in a patient. The higher the class, the more advanced the disease
(II) https://zdrowie.wprost.pl/choroby/sercowo-vascular/11412178/prof-wojakowski-skutecznosc-nowego-leku-w-kardiomiopatii-zostala-udokumentowana.html
(III) Data from mz.gov.pl
