Efanesoctocog alfa, a novel factor VIII replacement therapy, emerges as one of the options for improved prophylaxis in pediatric patients with hemophilia A.
Severe hemophilia A is conventionally treated with factor VIII concentrates, but effectiveness is limited by the von Willebrand factor (VWF)–imposed half-life ceiling, thereby requiring frequent infusions. Efanesoctocog alfa (ALTUVIIIO; formerly BIVV001) is designed to overcome this limitation by combining a recombinant factor VIII molecule with the Fc domain of human IgG1, the D′D3 domain of VWF, and two XTEN polypeptides.
XTEND-Kids, an international, industry-sponsored, open-label, phase 3 trial, evaluated the safety and efficacy of once-weekly intravenous (IV) efanesoctocog alfa (50 IU/kg) for 52 weeks in 74 children younger than age 12 with severe hemophilia A. All participants had >50 previous exposure days to factor VIII concentrates.…
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