Octapharma USA today announced that Balfaxar (prothrombin complex concentrate, human-lans; marketed in Europe and Canada as octaplex) has received U.S. Food and Drug Administration (FDA) approval for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adult patients with need for urgent surgery or invasive procedures.
Balfaxar helps restore blood coagulation by replenishing the levels of clotting factors that are deficient due to warfarin therapy. Balfaxar is a non-activated four factor prothrombin complex concentrate (4F-PCC) containing vitamin K-dependent factors: Factor II (prothrombin), Factor VII, Factor IX and Factor X, as well as antithrombotic Proteins C and S.
The FDA approval is supported by the clinical trial LEX-209 (ClinicalTrials.gov Identifier: NCT02740335), which compared the efficacy and safety of Balfaxar head-to-head with a control 4F-PCC (Kcentra). The Phase III, randomised, double-blind, multicenter study was performed at 24 sites in the U.S. and Europe and randomised 208 patients to Balfaxar (N=105) or control 4F-PCC (N=103).
“Balfaxar met the primary endpoint of hemostatic efficacy and was non-inferior to the comparator, Kcentra, in patients on a vitamin K antagonist undergoing urgent surgery with significant bleeding risk,” said LEX-209 Principal Investigator Ravi Sarode, M.D. “The primary objective was met at the prespecified interim analysis and the study was stopped due to statistically significant efficacy results indicating that Balfaxar was non-inferior to Kcentra. Balfaxar demonstrated effective hemostasis in 94.6% of patients versus 93.5% of patients for Kcentra. International Normalised Ratio (INR) reductions and vitamin K dependent coagulation factor increases supported the primary endpoint and PCC dosing and duration of infusion were also similar. The safety profile was similar between treatment arms and consistent with previous studies.”
More than 2.4 million U.S. patients are prescribed warfarin to prevent blood clots from forming following a heart attack, heart valve surgery, stroke, deep vein thrombosis/pulmonary embolism, or for certain types of irregular heartbeat (atrial fibrillation). The main side effect of warfarin is an increased risk of bleeding particularly for patients undergoing urgent surgery or invasive procedures.
“The FDA approval of Balfaxar establishes a new therapy for medical providers when their patients need a 4F-PCC product,” said Octapharma USA President Flemming Nielsen. "Octapharma is committed to providing patients with life-saving and life-enhancing therapies for critical care medicine. We are confident Balfaxar will be a welcomed treatment for physicians who need to quickly restore patients’ coagulation.”
Balfaxar, a lyophilised powder for reconstitution, will be provided with sterile water for injection and the new transfer device, nextaro. The transfer device includes an optimised vial housing that enables precentering of the vial during mounting, and optimised contamination protection with two integrated filters. Nextaro was preferred by healthcare professionals versus a widely used, competitive transfer device in an Octapharma user preference study.