The director of the hemostasis and thrombosis program at Children’s Hospital Los Angeles discussed advantages of the siRNA therapeutic.
“We had a decent number of patients with hemophilia B with inhibitors in the study. That is a very small population in the United States, roughly 150 patients. These patients have no good alternatives to preventing bleeding and few alternatives to treating bleeding. That's the group of patients that continues to really suffer and has been left behind... by the innovations we've had with emicizumab and factor therapy in hemophilia A.”
Findings from the phase 3 ATLAS-INH study (NCT03417102) of fitusiran were presented at the 63rd Annual American Society of Hematology (ASH) Meeting, December 11-14, 2021, by Guy Young, MD, director, Hemostasis and Thrombosis Program, Children’s Hospital of Los Angeles and professor of Pediatrics, Keck School of Medicine, University of Southern California. Fitusiran reduced bleeding in people with hemophilia A or B with or without inhibitors compared with those only given on-demand treatment. The siRNA therapeutic targets antithrombin and is administered in monthly, prophylactic doses.
Most participants in the study (65.8%) with both hemophilia A and B had treated bleeding events reduced to 0. In general, treated participants also had higher physical health domain health-related quality of life scores (both P <.0001).
GeneTherapyLive spoke with Young to learn more about fitusiran and how the siRNA therapeutic may improve outcomes in hemophilia A and B. He discussed unmet needs in hemophilia B and the decent population included in the ATLAS-INH study.