Phase III Results Show Balfaxar Non-Inferior to Kcentra in Reversing Warfarin Therapy

Results of a Phase III study (LEX-209) of the safety and efficacy of Balfaxar (prothrombin complex concentrate, human-lans), a blood coagulation factor replacement product made by Octapharma, have been published in JAMA Network Open, according to a press release from Octapharma USA on Aug. 12, 2024 (1,2).

The study investigated Balfaxar’s performance in the urgent reversal of acquired coagulation factor deficiency induced by vitamin K antagonist (VKA, e.g., warfarin) therapy in adults in need of urgent surgeries and other invasive procedures.

Warfarin, as the press release stated, is prescribed to millions of patients in the United States to prevent blood clots from forming after a heart attack, stroke, heart valve surgery, deep vein thrombosis or pulmonary embolism, or certain types of irregular heartbeat (atrial fibrillation) (1). However, because of its anti-coagulant properties, warfarin carries with it an increased risk of bleeding in patients undergoing related procedures.

In the study, Balfaxar was tested against another four-factor prothrombin complex concentrate (4F-PCC), Kcentra, evaluating just over 200 patients in total, almost exactly evenly split between the two products (1,2). (Balfaxar contains vitamin K-dependent factors II (prothrombin), VII, IX, and X, as well as antithrombotic proteins C and S.) Balfaxar demonstrated effective hemostasis in 94.3% of patients, compared with 94.2% for Kcentra. The safety profile, the study said, was in line with previous studies.

“We are pleased that JAMA Network Open has published the results of this important study,” said Ravi Sarode, MD, the study’s principal investigator. “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.”

Results of the LEX-209 trial to date have supported FDA’s approval of Balfaxar, according to the press release (1). The product is available as a lyophilized powder for reconstitution, which is then provided with sterile water as a diluent for injection and a transfer device.

“Octapharma launched Balfaxar in the US earlier this year and the response from the medical community has been outstanding,” said Octapharma USA president Flemming Nielsen. "The journal article provides further reinforcement of the great value this life-saving therapy has for patients. Octapharma is committed to ensuring a consistent Balfaxar supply for hospitals and medical providers now and in the future.”

Octapharma cautioned that reversing VKA therapy could lead to thromboembolic events, particularly in patients who have experienced such events in the past—in fact, Balfaxar is not recommended for people who have had a thromboembolic event in the previous three months. Fatal and non-fatal arterial and venous thromboembolic complications have been reported both in clinical trials involving Balfaxar, and post marketing surveillance (1). Patients who receive Balfaxar are recommended to be monitored for signs and symptoms of thromboembolic events.

References

1. Octapharma USA. JAMA Network Open Publishes Study Results Demonstrating Efficacy & Safety of Balfaxar for Warfarin Reversal in Urgent Surgery & Invasive Procedures. Press Release. Aug. 12, 2024.
2. Sarode, R.; Goldstein, J.N.; Simonian, G.; et al. Vitamin K Antagonist Reversal for Urgent Surgery Using 4-Factor Prothrombin Complex Concentrates: A Randomized Clinical Trial. JAMA Netw. Open 2024, 7 (8) e2424758. DOI: 10.1001/jamanetworkopen.2024.24758