Trial results of rVIII-SingleChain in haemophilia shows benefits- CSL Behring

CSL Behring presented data from a Phase I/III study on the efficacy and safety of rVIII-SingleChain (recombinant factor VIII single chain) in adolescents and adults with hemophilia A. Overall, patients using rVIII-SingleChain to prevent bleeding (prophylaxis) were well controlled with two to three infusions per week and developed no inhibitors. Patients using rVIII-SingleChain to prevent bleeding had low annualized bleeding rates (median ABR of 1.14), and an annualized spontaneous bleeding rate (AsBR) of 0.00. rVIII-SingleChain had improved pharmacokinetic parameters compared with octocog alfa, the comparator.

Of 848 bleeds treated in the study, 94 percent were successfully controlled with no more than two infusions of rVIII-SingleChain, with 81 percent controlled by only one infusion. The majority of bleeding events treated with rVIII-SingleChain and assessed by investigators (94 percent of 835 assessed bleeding events) were rated as excellent or good. The data are part of the AFFINITY Phase I/III study, an open-label, multi-center trial examining the safety, efficacy and pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alfa). Data were presnted at the 2015 International Society on Thrombosis and Haemostasis (ISTH) Congress.

Comment: rVIII-SingleChain is a novel recombinant single-chain factor VIII (FVIII) construct specifically designed for greater molecular stability. It uses a covalent bond that forms one structural entity, a single chain, to improve the stability of FVIII and provide longer-lasting FVIII activity.