Emerging treatments

Emicizumab

Emicizumab, a bispecific monoclonal antibody that mimics the function of factor VIII by bridging activated factor IX and factor X, is being explored in patients with severe VWD.[80] Emicizumab is currently approved for the management of haemophilia A. Case reports of use in patients with type 3 VWD demonstrate improvements in haemostasis.[80][81]

Rondoraptivon pegol

Rondoraptivon pegol, an investigational pegylated aptamer that binds to von Willebrand factor (VWF) and reduces its clearance, improved VWF levels and thrombocytopenia in one phase 2 study of patients with type 2B VWD.[82]

VGA039

VGA039 is an investigational long-acting monoclonal antibody against protein S. Preliminary studies show enhanced thrombin generation, and it is under investigation for patients with VWD.[83][84]

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