ASH 2021 | Fitusiran significantly reduces bleeding events in patients with hemophilia A or B, with inhibitors

Guy Young, MD, Children’s Hospital Los Angeles & Keck School of Medicine, University of Southern California, Los Angeles, CA, shares the findings of the Phase III ATLAS-INH trial (NCT03417102) of fitusiran prophylaxis in patients with hemophilia A or B, with inhibitors. Fitusiran is a novel siRNA therapeutic targeting antithrombin to restore thrombin generation and rebalance hemostasis. A total of 57 patients receiving on-demand therapy were randomized 2:1 to receive subcutaneous fitusiran prophylaxis or continue with on-demand bypassing agents (BPA). The results showed that treatment with fitusiran significantly reduced annualized bleeding rates compared to those in the BPA arm, with almost two thirds of patients having zero treated bleeding events. These outcomes led to meaningful improvements in health-related quality of life. One patient experienced treatment emergent adverse events that led to discontinuation. A revised fitusiran dosing regimen is under investigation in ongoing clinical studies. This interview took place at the 63rd ASH Annual Meeting and Exposition congress in Atlanta, GA.