Abstrakt

Recent trial results in recombinant coagulation factors for the treatment of hemophilia B

Julie Jaffray & Guy Young

Hemophilia B is an inherited bleeding disorder, due to a deficiency in factor IX (FIX). Patients require an intravenously injected drug, known as clotting factor concentrate, to prevent or stop bleeding. In the past, patients relied on blood-based products and ultrapurified plasma-derived FIX, which carry the risk of infectious transmission. Technological advancements have led to the development of recombinant FIX (rFIX), which is free from that risk; however, patients still require frequent intravenous injections. Recent advancement involves the development of FIX concentrates with a longer half-life, resulting in the need for less frequent injections. This article will review the rFIX concentrates, BeneFIX® and Rixubis®, as well as the newer, longer acting products, rFIX-Fc (Alprolix™), N9-GP and rFIX-FP.