Eptacog beta, a novel recombinant factor VIIa, for the treatment of hemophilia.

Abstract

Hemophilia A and B are X-linked hereditary bleeding disorders due to factor VIII (FVIII) or factor IX (FIX) deficiency, respectively. Major advancements have been made in the care of patients with hemophilia, yet the development of inhibitors to infused FVIII or FIX continues to be a formidable challenge. The current first-line therapy for acute bleeding episodes in patients diagnosed with inhibitors are bypassing agents including activated prothrombin complex concentrates (aPCCs) and recombinant factor VIIa (rFVIIa). Eptacog beta (SevenFact; LFB Biotechnologies, Hema Biologics) is a new rFVIIa product produced via expression in the milk of transgenic rabbits. This emerging platform has demonstrated numerous cost advantages to traditional cell culture systems including a better ability to scale up production and better protein yields. Eptacog beta is currently approved by the U.S. Food and Drug Administration (FDA) for the on-demand control of bleeding episodes in patients with hemophilia aged 12 to 75 with inhibitors. A potential future expansion of its current label could occur given the recent completion of two major phase III clinical trials evaluating its efficacy in children as well as its use for perioperative management. In this paper, we describe the preclinical and clinical literature documenting the development of eptacog beta and discuss its current and future application for the management of patients with hemophilia and inhibitors.