BIVV001 injection effective in severe hemophilia A
In the phase 1-2a open-label trial, 16 men with severe hemophilia A were treated with a single intravenous injection of recombinant factor VIII with a dose of 25 IU per kilogram of body weight or 65 IU per kilogram. A washout period of at least 3 days followed the injection before patients received a single intravenous injection of BIVV001 of the same dose.
Up to 28 days after the injection of BIVV001 no hypersensitivity or anaphylaxis events occurred and no inhibitors to factor VIII were detected. The geometric mean half-life of BIVV001 was 3-4 times as long as that of recombinant factor VIII.
In the higher dose-group, the mean factor VIII level was ≥51% for four days and 17% at day 7. The researchers concluded this may mean treatments can be given at a weekly interval.
Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. New Engl J Med. 2020;383:1018-1027. DOI: 10.1056/NEJMoa2002699.