Vonicog alfa for the treatment of von Willebrand disease

ABSTRACT

Introduction: 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.

KEYWORDS:

• Bleeding
• factor VIII
• plasma-derived concentrates
• recombinant concentrate
• von Willebrand disease
• von Willebrand factor

Acknowledgements

GC received honoraria to serve in Advisory Boards by CSL Behring and Baxalta.

SL received honoraria to serve in Advisory Boards by CSL Behring and Baxalta.

Declaration of interest

G Castaman has participated on advisory board’s for CSL Behring, Baxalta, Bayer, Kedrion, Novo Nordisk, Pfizer and Sobi. S Linari has participated on the advisory board of CSL Behring, Baxalta, Novo Nordisk and Sobi. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.