Octapharma has received the US Food and Drug Administration (FDA) approval for Balfaxar (prothrombin complex concentrate, human-lans), marketed as octaplex in Europe and Canada.

Balfaxar is a blood coagulation factor replacement product, indicated for the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist therapy in adults with the need for urgent surgery or invasive procedures.

It is a non-activated four-factor prothrombin complex concentrate (4F-PCC) that contains vitamin K-dependent factors, Factor II (prothrombin), Factor VII, Factor IX and Factor X, along with antithrombotic Proteins C and S.

Octapharma is offering the lyophilised powder that restores the levels of clotting factors deficient due to warfarin therapy, with sterile water for injection and the new transfer device, nextaro.

The nextaro transfer device includes an optimised vial housing that enables precentering of the vial during mounting, and optimised contamination protection with two integrated filters.

Octapharma said that healthcare professionals preferred nextaro device compared to a widely used competitive transfer device, in a user preference study.

Octapharma president Flemming Nielsen said: “The FDA approval of Balfaxar establishes a new therapy for medical providers when their patients need a 4F-PCC product.

“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.”

The FDA approval is supported by the LEX-209 clinical trial, which compared the efficacy and safety of Balfaxar compared to a control 4F-PCC (Kcentra), at 24 sites in the US and Europe.

In Phase 3, randomised, double-blind, multicentre study, 208 patients were randomised to receive either Balfaxar or control 4F-PCC.

Octapharma is recruiting for two additional Phase 3 studies of Balfaxar, LEX-210 and LEX-211.

LEX-210 will evaluate OCTAPLEX in patients with acute major bleeding on DOAC therapy.

LEX-211 will compare the 4-factor prothrombin complex with frozen plasma in cardiac surgery.

LEX-209 principal investigator Ravi Sarode said: “Balfaxar met the primary endpoint of haemostatic efficacy and was non-inferior to the comparator, Kcentra, in patients on a vitamin K antagonist undergoing urgent surgery with significant bleeding risk.

“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.

“International Normalized 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.”