Nonacog Beta Pegol: A Review in Haemophilia B

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Funding: The preparation of this review was not supported by any external funding.

Conflicts of interest: Yahiya Syed is a salaried employee of Adis/Springer, is responsible for the article content and declares no relevant conflicts of interest.

Additional information about this Adis Drug Review can be found here.

Abstract

Nonacog beta pegol (Refixia®) 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/adults with haemophilia B. In Paradigm 2, nonacog beta pegol showed good haemostatic effect when treating bleeds on-demand, and reduced annualized bleeding rates when used as a once-weekly prophylaxis. It also improved the health-related quality of life in adult patients. These findings are supported by the open-label extension Paradigm 4 trial. In Paradigm 3, nonacog beta pegol effectively maintained intraoperative and postoperative haemostasis, with minimal FIX consumption. Nonacog beta pegol was well tolerated in 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/adult patients with haemophilia B. Its extended half-life allows for a once-weekly prophylaxis. Therefore, nonacog beta pegol is a useful addition to recombinant FIX preparations with extended half-lives. Access to the full article can be found here.

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