Nonacog Beta Pegol: A Review in Haemophilia B

摘要

Abstract Nonacog beta pegol [Refixia ? (EU)] is an intravenously-administered, glycoPEGylated recombinant factor IX (FIX), with an extended terminal half-life. It is approved in the EU for the treatment and prophylaxis of bleeding in patients aged?≥?12?years with haemophilia B. The therapeutic efficacy and safety of nonacog beta pegol was demonstrated in the phase 3 Paradigm trials in previously treated adolescents and adults with haemophilia B. In Paradigm 2, nonacog beta pegol showed good haemostatic effects when treating bleeds on-demand, and reduced annualized bleeding rates when used as a once-weekly prophylaxis. It also improved some health-related quality of life measures in adult patients. The longer-term efficacy of nonacog beta pegol was demonstrated in the open-label extension Paradigm 4 trial. In Paradigm 3, nonacog beta pegol effectively maintained intraoperative and postoperative haemostasis. Nonacog beta pegol was well tolerated in phase 3 clinical trials in patients with haemophilia B, with no evidence of FIX inhibitor formation, allergic reactions or thromboembolic complications. In conclusion, nonacog beta pegol is effective and well tolerated in the on-demand, prophylaxis and perioperative settings in adolescent and adults with haemophilia B. Its extended half-life allows for once-weekly prophylaxis. Therefore, nonacog beta pegol is a useful additional treatment option for patients with haemophilia B.