Octapharma Announces Availability of Balfaxar® For Warfarin Reversal in Urgent Surgery & Invasive Procedures

Octapharma is Committed to Supplying Hospitals and Medical Providers with New Prothrombin Complex Concentrate.

Octapharma USA has announced Balfaxar® (prothrombin complex concentrate, human-lans; marketed in Europe and Canada as octaplex®) is now available. The new non-activated four factor prothrombin complex concentrate (4F-PCC), approved by the U.S. Food and Drug Administration (FDA) this summer, is indicated 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.

"The availability of Balfaxar^® brings a welcome new treatment option for medical providers around the country," said Josh Goldstein, MD, PhD, Professor of Emergency Medicine at Harvard Medical School and Massachusetts General Hospital. "More than 2.4 million U.S. patients are prescribed warfarin to prevent blood clots from forming in patients who are at high risk.1 Unfortunately, the main side effect of warfarin is an increased risk of bleeding, which can complicate urgent surgery or invasive procedures.2Clinical research has demonstrated that Balfaxar^® is extremely effective in restoring blood coagulation by replenishing the levels of clotting factors that are deficient in those patients on warfarin."

Balfaxar^® is a non-activated 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.

"We are excited to announce the availability of Balfaxar^® and are committed to providing the medical community with a valuable life-saving therapy," said Octapharma USA President Flemming Nielsen. "Octapharma has the production flexibility to ensure consistent supply for hospitals and medical providers now and in the future. We look forward to working with our critical care medicine partners to ensure patients receive the absolute best treatments available."

The FDA approval of Balfaxar^® was 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, randomized, double-blind, multicenter study randomized 208 patients to Balfaxar^® (N=105) or control 4F-PCC (N=103).

Balfaxar^® met the primary endpoint of hemostatic efficacy 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. Balfaxar^® demonstrated effective hemostasis in 94.6% of patients versus 93.5% of patients for 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.3

To study Balfaxar^® further in other clinical scenarios, Octapharma is recruiting for two additional Phase III studies:

• LEX-210: Study of OCTAPLEX in Patients With Acute Major Bleeding on DOAC Therapy With Factor Xa Inhibitor (ClinicalTrials.gov Identifier: NCT04867837)

• LEX-211: Active-control Randomised Trial Comparing 4-factor Prothrombin Complex Concentrate With Frozen Plasma in Cardiac Surgery (ClinicalTrials.gov Identifier: NCT05523297)

For more information on LEX-210 and LEX-211, please visit ClinicalTrials.gov.

Kcentra^® is a registered trademark of CSL Behring GmbH.

About Balfaxar^®

Balfaxar^® (prothrombin complex concentrate, human-lans) is a blood coagulation factor replacement product indicated 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 an urgent surgery/invasive procedure.

WARNING: ARTERIAL and VENOUS THROMBOEMBOLIC COMPLICATIONS
Patients being treated with Vitamin K antagonists (VKA) therapy have underlying disease states that predispose them to thromboembolic events. Potential benefits of reversing VKA should be weighed against the potential risks of thromboembolic events, especially in patients with the history of a thromboembolic event. Resumption of anticoagulation should be carefully considered as soon as the risk of thromboembolic events outweighs the risk of acute bleeding.

• Both fatal and non-fatal arterial and venous thromboembolic complications have been reported with Balfaxar^® in clinical trials and post marketing surveillance. Monitor patients receiving Balfaxar^® for signs and symptoms of thromboembolic events.

• Balfaxar^® may not be suitable in patients with thromboembolic events in the prior 3 months.

About the Octapharma Group

Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines.

Octapharma employs more than 11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Hematology, Immunotherapy and Critical Care.

Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centers across Europe and the US. The company's American subsidiary, Octapharma USA, is located in Paramus, N.J. For more information, please visit octapharmausa.com.

References

1 - ClinCalc.com, Warfarin: Drug Usage Statistics, United States, 2013 - 2020, accessed June 19, 2023.

2 - Mayo Clinic, Warfarin side effects: Watch for interactions, accessed June 19, 2023.

3 - Ravi Sarode, Joshua N. Goldstein, Gregory Simonian, Truman J. Milling Jr; A Phase 3, Prospective, Randomized, Double-Blind, Multicenter, Non-Inferiority Study Comparing Two Four-Factor Prothrombin Complex Concentrates for Reversal of Vitamin K Antagonist-Induced Anticoagulation in Patients Needing Urgent Surgery with Significant Bleeding Risk. Blood 2022; 140 (Supplement 1): 352–353. doi: https://doi.org/10.1182/blood-2022-168890