Vonicog alfa for the treatment of von Willebrand disease

Abstract

ABSTRACTIntroduction: von Willebrand disease (VWD) is the most frequent inherited bleeding disorder. It is caused by the quantitative and/or qualitative abnormalities of von Willebrand factor (VWF), an adhesive protein crucial for platelet-subendothelium interaction and for factor VIII stabilization in plasma. Clinical manifestations are mainly represented by excessive mucocutaneous bleeding and prolonged oozing after surgical procedures. The aim of therapy in VWD is to correct the abnormal platelet adhesion-aggregation as a result of low or dysfunctional VWF and the abnormal intrinsic coagulation owing to low FVIII levels. Current therapeutic options are desmopressin, which releases endogenous VWF from endothelial cells, and exogenous VWF contained in plasma-derived VWF/FVIII concentrates.Areas covered: This paper focuses on the potential therapeutic benefits for VWD of the first recombinant VWF concentrate, vonicog alfa. Topics covered include clinical development, pharmacokinetic profile, safety and efficacy data from Phase 1 and 3 clinical studies of vonicog alpha in patients with clinically severe VWD.Expert opinion: Vonicog alfa may provide advantages in treatment of VWD because it is a recombinant high-purity VWF, with a high efficacy profile when co-administered with FVIII as well as alone. Due to its efficacy even without FVIII, vonicog alfa offers the possibility of a more targeted, personalized replacement therapy based on the needs of each individual patient.