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Caplacizumab is a bivalent single-domain antibody (Nanobody) fragment that specifically targets the A1 domain of von Willebrand factor (vWF). By binding to vWF, it inhibits the interaction between vWF and the glycoprotein Ib-IX-V receptor on platelets, thereby preventing platelet aggregation and microthrombus formation. Caplacizumab is used in combination with plasma exchange and immunosuppressive therapy for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP), a rare autoimmune disorder characterized by microvascular thrombosis due to severe ADAMTS13 deficiency. The drug was developed by Ablynx and is marketed under the brand name Cablivi. It was first approved in Europe in 2018 and subsequently in the United States in 2019 as a first-in-class therapy for adults with aTTP[2][5][6][9].
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