伏尼凝血素α (Veyvondi)

概览

Vonicog alfa is a recombinant von Willebrand factor manufactured by Baxalta. It was FDA approved in December 2015. The gen of von Willebrand factor was first cloned in 1985 by Stuart Orkin and David Ginsburg. By the EMA, vonicog alfa is still under clinical analysis.

作用机制

Von Willebrand factor (vWF) is an important piece of the blood coagulation cascade and it acts by stabilizing the coagulation factor VIII. It also binds to collagen and platelets in blood vessel walls which helps with the formation of a platelet plug during clotting process. The presence of different mutations in vWF causes a common bleeding disorder named von Willebrand disease. The bleeding episodes were usually treated with plasma-derived vWF/factor VIII concentrates which required high doses and long treatment duration. The use of vonicog alfa allows a separate administration of vWF without the presence of the Factor VIII. This separate dosing allows the personalization of the dosage according to the patient's needs. On the same note, the plasma-derived vWF concentrates variably lack ULM due to an in vivo exposure to plasma ADAMTS13 and granulocyte elastases. Vonicog alfa can be delivered as an unaltered vWF and it functions as a replacement of vWF. Thus, it mediates platelet adhesion and aggregation as well as stabilization of the procoagulant factor VIII.

适应症

Vonicog alfa is indicated for the on-demand treatment and control of bleeding episodes in adults previously diagnosed with von Willebrand disease. Vonicog alfa contains only the vWF and thus, its administration offers the flexibility to administer the coagulation factor VIII if needed. The von Willebrand disease is an inherited disorder characterized by the deficiency or misfunction of the von Willebrand factor (vWF). Due to this deficiency, the blood cannot clot properly and the patients that present this disease are prone to prolonged or excessive bleeding. There are three types of this disease, and type 3 is an autosomal recessive inherited disorder marked by very low or absent levels of vWF.

• Bleeding