How well does Cablivi(Caplacizumab) work?

CABLIVI exerts its therapeutic effect in acquired Thrombotic Thrombocytopenic Purpura (aTTP) by specifically inhibiting von Willebrand factor-mediated platelet adhesion and aggregation, a key pathological process in this condition, when used alongside plasma exchange and immunosuppression.

Mechanism and Therapeutic Impact of CABLIVI in aTTP

Body content:CABLIVI is indicated for adult patients with acquired Thrombotic Thrombocytopenic Purpura (aTTP), functioning as an adjunct to plasma exchange and immunosuppressive therapy. Its therapeutic efficacy stems from its action as a von Willebrand factor (vWF)-directed antibody fragment. By binding to the A1-domain of vWF, caplacizumab-yhdp inhibits the interaction between vWF and platelets. This targeted inhibition prevents the formation of platelet-rich microthrombi in small blood vessels, which is a hallmark of aTTP. Consequently, CABLIVI helps to reduce platelet consumption, leading to faster platelet count normalization, and mitigates end-organ damage associated with microvascular thrombosis. Clinical studies suggest it contributes to reducing the time to response and the recurrence of aTTP episodes. The precise quantitative impact on specific clinical endpoints beyond general improvement and relapse reduction is not detailed in this summary but is established in its clinical development.