MILAN (ITALPRESS) – With the publication of the AIFA in the Official Journal, marstacimab (Hympavzi trade name) is officially available in Italy under repayment. The drug, administered by subcutaneous means, once a week, through pre-filled pen, with fixed dosage, is indicated for the routine prophylaxis of bleeding episodes in patients with hemophilia A and B severe without inhibitors of age equal to or greater than 12 years and of weight equal to or greater than 35 kg. The most common forms are hemophilia A, which affects more than 4,000 patients, and hemophilia B, with about 900 cases. Both are caused by the lack of specific coagulation factors: factor VIII (FVIII) and factor IX (FIX), fundamental proteins for the proper functioning of the coagulation process, the natural mechanism that allows haemorrhages to be stopped. The standard therapeutic options for hemophilia A and B are based on the replacement of coagulation factors, which provides for intravenous administration of the lacking factors (FVIII and FIX) in order to prevent or control hemorrhagic episodes. In this context, marstacimab, developed by Pfizer, represents a new option in the therapeutic panorama for patients with severe A and B hemophilia without inhibitors; the first non-substitutional approved treatment with rebalancing mechanism of hemostasis, acting on the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI), a protein that normally limits the coagulation process. Through the inhibition of this mechanism of regulation, the drug helps to restore the balance of the coagulation system, reducing bleeding without directly replacing the lacking factors. Despite the progress achieved in the last decades in the management of hemophilia, therapeutic needs are still unsatisfactory. “In the clinical practice of hemophilia one of the main challenges remains the control of bleeding – explains Maria Elisa Mancuso, Referent Center for Hemorrhagic Diseases at IRCCS Humanitas Research Hospital and Professores at Contract at Humanitas University – which represent a concrete risk for many patients and can determine, over time, joint complications and a significant impact on quality of life. This adds the weight of intravenous infusions, necessary for many therapies. In this context, marstacimab is inserted, which, through its non-substitutional anti-TFPI mechanism, contributes to restoring the hemostatic balance and reducing hemorrhagic episodes. Moreover, subcutaneous administration and fixed dosage simplify prophylaxis, favoring autonomy and adherence to therapy”. The indication of the new therapy is supported by the results of Phase 3 BASIS study: marstacimab reduced the annualized rate of bleeding (ABR) treated by 36% compared to routine prophylaxis in patients with hemophilia A or B without inhibitors, demonstrating non-lowerity and statistical superiority compared to prophylactic factors based on a favorable safety profile. In the long-term extension study (OLE), currently in progress, interim data show that effectiveness has been consistent over time, with a further maintenance of low blood rates treated. “Pfizer’s commitment to haemophilia research, which began over forty years ago, led to the introduction of treatments that in the 1990s revolutionized the life of patients,” says Barbara Capaccetti, Medical Director of Pfizer in Italy. «Today, with marstacimab we make a further step forward, offering an innovative therapy based on a solid scientific rationale that simplifies the management of the disease and improves concretelythe quality of life of people with hemophilia A and B. In addition to scientific progress, it remains fundamental for us to collaborate with the scientific community, with patient associations and institutions so that innovation is translated into concrete opportunities of care, capable of responding also to the daily needs of people with hemophilia». In addition to the clinical aspects of pathology, hemophilia deeply affects the quality of life of the patient, highlighting the need to ensure not only adequate clinical support, but also overall taking paths. “The management of the disease can still lead to limitations in the daily lives of people, capable of affecting autonomy, social relations and the possibility to look with full serenity to their future”, says Cristina Cassone, President of the Federation of Emophilic Associations (FedEmo) and spokesperson of the patient community. “In order that no patient is without support or disadvantage, the Federation undertakes every day to promote greater awareness of the disease and to collaborate with institutions and professionals to ensure increasingly adequate treatment paths and fair access to therapies. At the same time, investing in the continuous training of specialists and health professionals is fundamental to guarantee an increasingly adequate and attentive grip to the real needs of people”. Living with hemophilia often means confronting oneself with continuous attention in the management of everyday life, thus impacting also the psychological condition and emotional balance of the patient and, sometimes, of the whole family: “Hemophilia affects every aspect of the patient’s life and his family, from school and working activities to sports practice and social life,” says Andrea Buzzi, President of Fondazione Paracelso. “As anyone who lives with a chronic disease, the person with hemophilia needs, in addition to medical care and essential life-saving drugs, constant attention to those that we could define extraclinical needs, psychosocial support, first to parents, to foster the acceptance of the disease, awareness programs and increased knowledge and visibility of hemophilia to public opinion.” -Pfizer press office photos –(ITALPRESS).
