BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A

Researchers evaluated the effect of the novel fusion protein BIVV001 on factor VIII activity levels in individuals with hemophilia A. Factor VIII replacement products are known to benefit this patient population but are limited by a short half-life of just 15–19 hours.

Researchers evaluated the effect of the novel fusion protein BIVV001 on factor VIII activity levels in individuals with hemophilia A. Factor VIII replacement products are known to benefit this patient population but are limited by a short half-life of just 15–19 hours. The Phase I/IIa trial involved 16 male adults who underwent previous therapy for severe hemophilia. Each study participant received a single I.V. injection of high-dose or low-dose recombinant factor VIII. After a minimum washout period of 3 days, they then received a single I.V. infusion of BIVV001 at a corresponding dose. High levels of factor VIII were sustained with the treatment, with a half-life up to four times that associated with recombinant factor VIII. In the higher-dose group, mean factor VIII level after BIVV001 treatment was in the normal range of greater than or equal to 51% for 4 days, falling to 17% by day 7. With no hypersensitivity or anaphylaxis events reported in the 28 days after treatment administration, the outcomes point to a potential new class of factor VIII replacement therapy administered in weekly intervals.