Fitusiran

Overview

siRNA therapeutic (brand Qfitlia) silencing antithrombin expression to rebalance hemostasis in hemophilia; dosed every couple of months but creates a 3-4 month irreversible reduction in antithrombin, raising surgical thrombosis management concerns for a well-informed patient population. Fitusiran (Qfitlia) is a short interfering RNA that knocks down antithrombin expression, reducing a key anticoagulant to rebalance hemostasis in hemophilia; it is dosed every couple of months at a low dose and is fairly well tolerated, but requires dose adjustment due to its potent mechanism. Fitusiran creates a prolonged state of reduced antithrombin lasting approximately three to four months per injection with no ability to rapidly reverse the effect, raising concerns about managing thrombosis risk in patients requiring major surgery during the dosing window. (Treatment Landscape (35:00-40:00))