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Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)
Guy Young, Alok Srivastava, Kaan Kavakli, Cecil Ross, Jameela Sathar, Huyen Tran, Runhui Wu, Jing Sun, Stacey Poloskey, Zhiying Qui, Salim Kichou, Shauna R. Andersson, Baisong Mei, Savita Rangarajan Abstract
Introduction
Hemophilia A and B are rare bleeding disorders characterized by ineffective clot formation due to impaired thrombin generation as a result of deficiency of FVIII or FIX, respectively. Fitusiran is a subcutaneously (SC) administered investigational siRNA therapeutic targeting antithrombin to restore thrombin generation and rebalance hemostasis in people with hemophilia A or B, with or without inhibitors. Here, we present the safety and efficacy of fitusiran prophylaxis for PwHI in a phase 3 study (ATLAS-INH; NCT03417102).