The following is a press release from Bioverativ. Read the full press release here.
Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, presented initial clinical data for BIVV001, a novel and investigational von Willebrand factor (VWF)-independent factor VIII therapy for people with hemophilia A. Preliminary safety and pharmacokinetic data from the ongoing EXTEN-A Phase 1/2a study showed that a single low dose of BIVV001 extended the half-life of factor VIII to 37 hours with high factor activity levels, and was generally well tolerated. The data were presented at a late-breaking session at the World Federation of Hemophilia (WFH) 2018 World Congress being held in Glasgow, Scotland.
“For decades, scientists have been trying to overcome the von Willebrand factor ceiling, which imposes a limit on the half-life of factor VIII, and these data demonstrate that BIVV001 has finally broken through that ceiling,” said Joachim Fruebis, Senior Vice President of Development at Bioverativ. “Importantly for the hemophilia community, the factor levels seen in this study are unparalleled in hemophilia A, and we are excited about the potential for BIVV001 to transform the treatment paradigm for patients and physicians.”
Factor replacement therapy is fundamental in the treatment of hemophilia A, as it naturally provides what is missing in the body (clotting factor VIII), and it has a consistent and well-characterized safety and efficacy profile. BIVV001 is an investigational factor VIII therapy that has the potential to provide comprehensive protection in all treatment scenarios including management of acute bleeds, perioperative care, emergency situations, and prophylactic use. The role of factor also goes beyond the coagulation cascade and may have a role to play in joint and bone health of people with hemophilia.
Read the press release in its entirety here.