First patient dosed in phase III XTEND-Kids study with efanesoctocog alfa in children with haemophilia A.- Swedish Orphan Biovitrum.

Swedish Orphan Biovitrum AB (publ) announced that the first patient has been dosed in the phase III, open-label, interventional XTEND-Kids study of efanesoctocog alfa (BIVV001) in paediatric subjects with severe haemophilia A . The XTEND-Kids study is designed to investigate the efficacy, safety and pharmacokinetics of efanesoctocog alfa as once-weekly prophylaxis in approximately 65 previously treated patients less than 12 years of age. Duration of treatment in the study is 52 weeks. Efanesoctocog alfa is being developed and will be commericalized in partnership with Sanofi. Factor VIII (FVIII) replacement therapy as a single agent has been shown in clinical studies and real-world experience to control and prevent bleeding effectively in a variety of settings including surgery. Efanesoctocog alfa represents a potential new class of FVIII replacement therapy with high sustained factor levels, and the first recombinant FVIII therapy that is designed to be von Willebrand factor (VWF) independent, which extends half-life. It is currently in clinical development and has the potential to offer extended protection with once weekly dosing, thus addressing an unmet need for people living with severe haemophilia A. EXTEND-Kids:XTEND-Kids is an open-label, non-randomised interventional, single-arm study. Participants will receive a weekly prophylactic dose of efanesoctocog alfa for 52 weeks. XTEND-Kids will evaluate efficacy, safety and pharmacokinetics in approximately 65 previously treated patients less than 12 years of age with severe haemophilia A.