This investigational siRNA-based therapy shows promise in reducing bleeding episodes in males with severe hemophilia A or B with or without inhibitors and without thrombophilia.
Replacement with clotting factor concentrates remains the standard of care for hemophilia but requires frequent infusions. Management of patients who develop inhibitors (antibodies) against exogenously administered clotting factor is complicated by bleeding diathesis and relies on infusion of bypassing agents. Fitusiran, an investigational small interfering RNA therapeutic, is designed to rebalance hemostasis by reducing antithrombin synthesis in patients with hemophilia.
Two industry-sponsored, multinational, open-label, phase 3 randomized trials evaluated the safety and efficacy of fitusiran in patients with and without inhibitors. In both trials, males aged ≥12 years with severe hemophilia A or B who were receiving treatment with on-deman…
Reviewing Author
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute