Bringing new therapy options to the hemophilia community

Abstract

In patients with hemophilia A, outcomes have improved dramatically over the last few decades due to several advances in care, including the availability of factor VIII (FVIII) concentrates. Current research has focused on enhancing the properties of FVIII concentrates and other coagulation factor products using recombinant DNA technology. However, there are several challenges to the development of new products for hemophilia patients, including the relative rarity of the disease, rapidly evolving standards of care, and the varying requirements of regulatory authorities around the world. In the development of two innovative coagulation factor products (recombinant single-chain factor VIII [rVIII-SingleChain] and a recombinant fusion protein linking coagulation factor VIIa with albumin [rVIIa-FP]), these issues have been addressed through novel clinical trial designs, including an ongoing three-part study evaluating the safety, efficacy, and pharmacokinetics of rVIII-SingleChain in patients with severe hemophilia A, and a randomized, placebo-controlled, dose-escalation study of rVIIa-FP in healthy volunteers. The design of these trials is intended to answer as many important clinical questions as possible while limiting the burden on hemophilia patients.