CSL Behring announced that the Food and Drug Administration has accepted for review the Biologics License Application (BLA) of its novel investigational recombinant factor VIII single-chain (rVIII-SingleChain) for the treatment of hemophilia A.
The BLA submission is based on the AFFINITY clinical development program, which includes a phase I/III open-label, multi-center trial examining safety and efficacy. The pharmacokinetics of rVIII-SingleChain compared with recombinant human antihemophilic factor VIII (octocog alfa) was also studied.
In the pivotal clinical trial, rVIII-SingleChain met all primary endpoints. Patients treating prophylactically had a median annualized bleeding rate (ABR) of 1.14 and a median annualized spontaneous bleeding rate (AsBR) of 0.00. The data also showed that, of 848 bleeds treated in the study, 94% were controlled with no more than two infusions of rVIII-SingleChain, with 81% controlled by one infusion.
rVIII-SingleChain is the first and only single-chain factor VIII (FVIII) product in late-stage development for the treatment of hemophilia A. rVIII-SingleChain (also known as CSL627) has a strong affinity for von Willebrand factor, leading to greater stability and integrity of FVIII in circulation.
For more information call (800)504–5434 or visit CSLBehring.com.