CSL Behring today announced that the U.S. Food and Drug Administration (FDA) has accepted for standard review its Biologics License Application (BLA) for a human 4-factor prothrombin complex concentrate (PCC) for the urgent reversal of vitamin K-antagonist therapy (i.e., warfarin) in patients with acute major bleeding. If approved by the FDA, the CSL Behring 4-factor PCC would be the first agent of its kind available in the United States.
"The acceptance by FDA of this 4-factor PCC BLA marks an important step toward addressing the urgent unmet need for a rapid and simple way to reverse the effects of warfarin during bleeding emergencies," said Val Romberg, Senior Vice President, Research and Development at CSL Behring. "This 4-factor PCC has the potential to be a significant new tool in stopping excessive bleeding in patients on warfarin, where fresh frozen plasma is the current standard of care."
The CSL Behring BLA 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 (p values<0.0001).