Alprolix (recombinant Factor IX Fc fusion protein): extended half-life product for the prophylaxis and treatment of hemophilia B

Abstract

Hemophilia B is a genetic disease caused by mutation of the gene for coagulation protein Factor IX. When severe, the disease leads to spontaneous life-threatening bleeding episodes. Current therapy requires frequent intravenous infusions of therapeutic recombinant or plasma-derived protein concentrates containing Factor IX. Alprolix™ (recombinant Factor IX Fc fusion protein), is a therapeutic Factor IX preparation that has been engineered for a prolonged half-life in circulation, has completed pivotal clinical trials and has been approved recently in the USA, Canada, Australia and Japan for use in the clinic for patients with hemophilia B. This promising therapy should allow patients to use fewer infusions to maintain appropriate Factor IX activity levels in all clinical settings, and its use may be indicated in both on demand and prophylactic treatments.

Keywords::

• Factor IX
• gene therapy
• genetic coagulation defects
• hemophilia arthropathy
• hemophilia B
• long-acting factors
• prophylaxis

Financial & competing interests disclosure

JM Ducore and JS Powell received clinical trial support from Bayer, Octapharma, Novo Nordisk, Baxter, CSL Behring and Biogen Idec. 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

• Recombinant Factor IX Fc fusion protein prophylaxis resulted in less consumption of factor product compared with current Factor IX (FIX) products and at least comparable annual bleed rates compared with prior FIX prophylaxis regimens.

• The data presented in this review article include the first description of FIX pharmacokinetic simulations, which support the validity of empiric dosing of recombinant Factor IX Fc fusion protein in patients with hemophilia B.

Notes