Individuals with hemophilia B experience frequent and spontaneous bleeding episodes into joints and muscles that can lead to severe arthropathy, chronic pain, disability, and diminished quality of life (QoL). Prophylaxis with factor nine (FIX) concentrates may reduce the frequency of bleeding events and improve QoL. Recombinant FIX (rFIX) concentrates are a potentially safer treatment option than plasma-derived FIX products with respect to pathogen transmission risk, but until recently, only one licensed rFIX product was available. We describe a newly approved rFIX concentrate, BAX326 (RIXUBIS; Baxter Healthcare Corporation). Phase III studies of BAX326 demonstrated its efficacy and safety in prophylactic, on-demand, and surgical settings and showed that its pharmacokinetic properties were comparable to those of the licensed comparator. Importantly, prophylaxis with BAX326 significantly improved physical health-related QoL, demonstrating that this new rFIX treatment that has the potential to improve outcomes in hemophilia B patients.
Keywords: hemophilia B; hemostasis; prophylaxis; quality of life; recombinant factor IX; surgery.