On July 6, the clinical trial application for recombinant human coagulation factor VIII Fc–von Willebrand factor–XTEN fusion protein (BIVV001) for injection, submitted by Sanofi/Bioverativ in China, was accepted by the Center for Drug Evaluation (CDE).
BIVV001 is a novel recombinant factor VIII therapy for the treatment of severe hemophilia A. By fusing recombinant coagulation factor VIII with Fc, VWF, and XTEN polypeptides, BIVV001 extends its half-life to more than 40 hours, which is three times that of conventional recombinant factor VIII.
Source: Sanofi Official Website
Patients require only a single injection of BIVV001 to maintain near-normal factor VIII levels for the subsequent 3.5 days. This therapy has been granted orphan drug designation by both the U.S. Food and Drug Administration (FDA) and the European Union. Sanofi initiated Phase III clinical trials for this therapy in the fourth quarter of 2019, with plans to submit a marketing application to the FDA in the first half of 2022.
Source: Sanofi Official Website
Hemophilia A is a hemorrhagic disorder caused by congenital deficiency of coagulation factor VIII. Patients primarily present with a bleeding tendency, involving widespread sites that often recur, potentially leading to hematoma formation and joint deformity; intracranial hemorrhage is a common cause of death. Phase I clinical trials demonstrated that patients receiving once-weekly BIVV001 treatment experienced no bleeding episodes during the treatment period and for 10 days thereafter, with good tolerability.
Source: Sanofi Official Website