Drug intelligence / Profile preview

human von Willebrand factor concentrate

Development stage
Unknown
Lead developer
Takeda
Modality
Recombinant Proteins and Enzymes
Administration
Intravenous
01

Overview

A high-purity, plasma-derived human von Willebrand factor (VWF) concentrate investigated for the treatment of von Willebrand disease (VWD). Developed and studied in Phase I clinical trials sponsored by the National Center for Research Resources (NCRR) and the National Institutes of Health (NIH), this product was specifically designed to evaluate the pharmacokinetics and physiological effects of VWF replacement in isolation from exogenous Factor VIII. The concentrate functions by replacing deficient or dysfunctional VWF, which is essential for platelet adhesion to the vascular subendothelium at sites of injury and for the stabilization of endogenous Factor VIII in the circulation. By providing a purified source of VWF, the therapy aims to correct bleeding time and restore normal hemostatic function in patients with various subtypes of VWD.

Other names
von Willebrand factorhuman von Willebrand factor concentrateVWF concentrate
02

Targets

F8 (Coagulation Factor VIIIa)GP9 (Platelet glycoprotein ib-ix-v complex)ADAMTS13 (A disintegrin and metalloproteinase with thrombospondin motifs 13)COL (Collagen type XXI alpha 1 chain)

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