Eptacog beta: a novel recombinant human factor VIIa for the treatment of hemophilia A and B with inhibitors

Hemophilia A and B are X-linked recessive disorders caused by the deficiency of Factor VIII or factor IX, respectively. Bleeding episodes are treated with factor replacement therapy. The most serious complication of this treatment is the development of inhibitors. In such patients, bypassing agents, such as activated recombinant human factor VII (rhFVIIa) or plasma-derived activated prothrombin complex concentrates, are administered to prevent or treat bleeding episodes. The high cost of the current bypassing agents limits their availability in emerging countries. Areas covered: Authors reviewed the published data on the development and clinical testing of eptacog beta, a new second-generation rhFVIIa produced in the milk of transgenic rabbits. The available data indicate that activated eptacog beta exhibits structural (N- and O- glycosylation), pharmacodynamic and pharmacokinetic characteristics similar to activated eptacog alfa, its main competitor, but binds slightly better to platelets and HUVEC, and it is safe and effective. Expert commentary: This critical review of available data on activated eptacog beta shows that it represents an alternative source of rhFVIIa at potentially lower cost with easily expandable manufacturing capacity that could contribute to cover the future patient needs.

Hemophilia; bypassing agent; eptacog beta; inhibitors; milk; recombinant activated FVII; transgenic rabbits.