Innovations in coagulation: improved options for treatment of hemophilia A and B

Abstract

The availability of plasma-derived and recombinant coagulation factor products represents an important breakthrough in the management of patients with hemophilia that has led to improvements in life expectancy and quality of life. Current research has focused on enhancing the pharmacokinetic properties of these products. Half-life extension, for example, could allow for less frequent dosing, which would increase the convenience of therapy and have a meaningful impact on patient compliance and quality of life. On 9 July 2012, CSL Behring sponsored a symposium, entitled “Innovations in coagulation: improved options for treatment of hemophilia A and B”, which took place at the World Federation of Hemophilia 2012 World Congress in Paris, France. Topics addressed new treatment approaches currently in development for the treatment of hemophilia A, hemophilia B, and von Willebrand disease. The articles in this supplement summarize the presentations from the symposium. In the first article of this supplement, Dr. Stefan Schulte of CSL Behring provides an update on the company’s ongoing research aimed at improving currently available therapeutic coagulation factor products. This includes the use of recombinant albumin fusion technology to extend the half-lives of factor VIIa (FVIIa) and factor IX (FIX). Dr. Schulte addresses not only the advantages of using albumin fusion technology to prolong the pharmacokinetic properties of therapeutic proteins but also some of the challenges in designing and developing fusion proteins, and how unique solutions were devised to overcome some of these challenges. Other advances currently in development include a unique recombinant single-chain factor VIII (FVIII) protein (rVIII-SingleChain), which has improved stability and a high affinity for von Willebrand factor, relative to other recombinant FVIIIs. In the second article, Dr. Elena Santagostino details the results of the phase I, first-in-human trial of the recombinant fusion protein linking recombinant FIX with albumin (rIX-FP) in patients with hemophilia B. The results indicate that rIX-FP has an excellent safety profile and significantly improved pharmacokinetic profile