FDA accepts BLA for human 4-factor prothrombin complex concentrate (CSL Behring) for acute Bleeding

The FDA has accepted a Biologics License Application (BLA) for a human 4-factor prothrombin complex concentrate (PCC), from CSL Behring, for the urgent reversal of vitamin K-antagonist therapy (i.e., warfarin) in patients with acute major Bleeding. The submission is based on results from three prospective Phase III clinical trials that evaluated the safety and efficacy of PCC in patients who required urgent reversal of warfarin therapy. The pivotal, acute major bleeding trial demonstrated that PCC was comparable to plasma at 24 hours in patients who required urgent reversal of warfarin therapy (72.4 percent and 65.4 percent, respectively). Additionally, the co-primary efficacy endpoint analysis showed that the 4-factor PCC was superior in achieving target INR correction within 30 minutes at the end of infusion as compared to plasma (62.2 percent and 9.6 percent, respectively). Four-factor PCC was also superior to plasma in rapidly and safely raising the levels of clotting factors II, VII, IX, X, and anticoagulant Proteins C and S at the same 30 minutes post-treatment time point. If approved by the FDA, the 4-factor PCC would be the first agent of its kind available in the US.