BAX326 (recombinant coagulation factor IX) for the treatment and prophylaxis of hemophilia B

Abstract

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

Financial & competing interests disclosure

J Windyga has received honoraria or research funding from Baxter, Bayer, Behring, Novo Nordisk, Octapharma and Pfizer. BE Abbuehl and AE Hafeman are employees of Baxter Healthcare Corporation, the sponsor of these studies. The authors would like to thank M Zbreski of MedLogix Communications, LLC for editorial assistance with this manuscript. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Prophylaxis with coagulation factor nine (FIX) with plasma-derived FIX or recombinant FIX (rFIX) clotting factor concentrates can decrease the frequency of bleeding episodes, and may help to prevent joint damage and improve health-related quality of life in patients with hemophilia B.

• There is a need for additional rFIX products to increase global supply, lower the cost of rFIX concentrates and expand treatment choices for patients with hemophilia B.

• BAX326, an rFIX product, was recently approved in the USA for routine prophylaxis, control and prevention of bleeding episodes, and perioperative management in adults with hemophilia B.

• Studies of BAX326 in patients with severe or moderately severe hemophilia B demonstrated PK properties that were comparable to those of the comparator rFIX product (BeneFIX^®).

• Prophylactic therapy with BAX326 was shown to be efficacious in reducing the frequency of bleeding episodes, with 43% of patients experiencing zero bleeds over the course of the study.

• Prophylaxis with BAX326 was associated with statistically significant and clinically meaningful improvements in physical health-related quality of life.

• An ongoing study of BAX326 in the surgical setting suggests that it provides safe and efficacious hemostatic control in hemophilia B patients who are undergoing surgical procedures.

• BAX326 has demonstrated safety in patients with hemophilia B, with no incidences of FIX inhibitor development, severe allergic reactions or thrombotic events.

Notes