Marstacimab shows long-term safety and efficacy in preventing bleeding events in patients with hemophilia A and B without inhibitors, potentially expanding treatment options.
Marstacimab, a novel, investigational monoclonal antibody, shows long-term safety and efficacy in the prevention of bleeding events in patients with hemophilia A as well as B without inhibitors, potentially adding to the toolbox for hemophilia A and representing a first of its kind therapy for hemophilia B.
The therapy has been granted fast-track and orphan drug status in the United States, in addition to orphan drug status in the European Union for the prevention of hemophilia bleeding episodes. Of the patients, 89 were adult, and 18 were adolescents. Overall, they had a mean age of 29 years; 83 patients had hemophilia A, while 24 had hemophilia B.
In the hemophilia A and B groups combined, those previously treated with on-demand factor replacement therapy had substantial reductions in estimated ABR for treated bleeds from the baseline of 38.0 prior to initiating marstacimab, to 3.2 after 12 months of the treatment in the trial . That reduction was sustained at an ABR of 3.7 after the mean additional 12.5 months in the extension study.
The trends were similar among those with hemophilia B, albeit with lower numbers of patients, consistent with hemophilia B being more rare.
Haemophilia Hemophilia A Haemophilia A Hemophilia B Haemophilia B Hemorrhage Bleeding Prophylaxis Adherence Adherence To Medication Issues For Patients Compliance Drug Compliance Treatment Adherence Adolescent Medicine Teens Teenage Teenager Adolescent Health Adolescents Adolescent Europe European Orphan Drugs Thromboembolism Thrombus Blood Clot Biologic Therapy
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