Octapharma USA Presents Research on Congenital & Acquired Bleeding Disorders at ASH Annual Meeting
Research evaluates use of fibryga®, Octaplas™, and Octaplex® as investigational therapies
PARAMUS, N.J. (December 3, 2020) – Octapharma USA will present multiple clinical research posters focused on the efficacy and safety of fibryga®, Fibrinogen (Human) Lyophilized Powder for Reconstitution, for Intravenous Use in the treatment of congenital and acquired bleeding disorders during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, a virtual medical congress to be held December 5 – 8.
The research focused on fibryga® addresses the therapy’s safety and efficacy to treat congenital fibrinogen (Factor 1) deficiency, a rare, inherited blood disorder where the blood does not clot normally, during on-demand bleeding episodes and surgical prophylaxis in adults and pediatrics. Additionally, Octapharma will present fibryga® research evaluating the product’s hemostatic efficacy in acquired fibrinogen deficiency, a condition affecting non-surgical and surgical bleeding patients, including cardiac surgery, post-partum hemorrhage and trauma patients.
“Octapharma is determined to advance clinical research and treatment options for people with life-threatening bleeding disorders, including Factor 1 deficiency and other rare conditions,” said Octapharma USA President Flemming Nielsen. “The National Hemophilia Foundation recently updated its treatment guidelines for Factor 1 deficiency to include fibryga® so the ASH research we will be presenting continues to add important clinical treatment data for this therapy. We look forward to sharing the latest research developments with the medical community.”
The fibryga® focused research includes:
“Efficacy and Safety of Human Fibrinogen Concentrate for the Treatment of Patients with Congenital Fibrinogen Deficiency: Combined Results of the FORMA-02 and FORMA-04 Clinical Trials”: Fibryga® was shown to be efficacious for on-demand treatment of bleeding episodes and perioperative prophylaxis in a rare congenital fibrinogen deficiency (CFD) population, across two Phase 3 clinical trials. Hemostatic efficacy was comparable for adult, adolescent and pediatric patients. A favorable safety profile was seen for the treatment of patients with congenital afibrinogenemia with fibryga®. Presenter: Claudia Khayat, MD, BS, Hotel Dieu de France, Beirut, Lebanon.
“Fiirst-2: Prospective, Randomized Study Comparing Administration of Clotting Factor Concentrates with Standard Massive Hemorrhage Protocol in Severely Bleeding Trauma Patients”: FiiRST-2 will determine if early hemostatic therapy with fibryga® and prothrombin complex concentrate (PCC) is superior to standard massive hemorrhage protocol (MHP) packs in bleeding trauma patients. Results could have a major impact on clinical practice and improve management and outcomes in this high-risk group of patients. Presenter: Luis Da Luz, MD, University of Toronto, Canada.
“Real-world Experience with a New Human Fibrinogen Concentrate (Fibryga®) in France for the Treatment of Bleeding Episodes and Surgical Prophylaxis”: Fibryga® was used mostly for bleeding control in specific subpopulations, including patients undergoing cardiac surgery and women with post-partum hemorrhage (PPH). Presenter: François Stephan, Hôpital Marie Lannelongue, Le Plessis Robinson, France.
Octapharma will also present research focused on Octaplas™, Pooled Plasma (Human), Solvent/Detergent (S/D) Treated Solution for Intravenous Infusion, entitled, “Retrospective Analysis of the Efficacy of Solvent/Detergent Treated Pooled Plasma As Compared to Fresh Frozen Plasma in Thrombotic Thrombocytopenic Purpura (TTP): A Single Center Experience.” The study confirmed previously reported efficacy of Octaplas™ for treating suspected/confirmed TTP. There were no significant differences in thromboembolic or bleeding events between patients who received FFP as compared to Octaplas™. Presenter: Catherine Klapheke, University of Rochester Medical Center, Rochester, N.Y.
Finally, ASH attendees will have the opportunity to review research focused on another product, octaplex® Human Prothrombin Complex, freeze dried powder and solvent for solution for injection, which is not licensed in the U.S. The posters are:
“The Association of Prothrombin Complex Concentrates with Transfusion Requirement and Postoperative Outcomes in Cardiac Surgery: A Post-Hoc Analysis of the FIBRES Randomized Controlled Trial”: In cardiac surgical patients with post-cardiopulmonary bypass (CPB) bleeding who received cryoprecipitate or fibryga®, PCC use was associated with decreased red blood cell and platelet transfusion compared to frozen plasma, without a significant increase in adverse events. Presenter: Justyna Bartoszko, MD, MSc, FRCPC, Toronto General Hospital, Canada.
“The FARES Study: A Multicenter, Randomized, Active-Control, Pragmatic, Phase 2 Pilot Study Comparing Prothrombin Complex Concentrate (PCC) versus Frozen Plasma (FP) in Bleeding Adult Cardiac Surgical Patients” will determine the feasibility, and inform the design and primary outcome parameter, of a definitive Phase 3 trial comparing the efficacy and safety of PCC versus FP in bleeding cardiac surgical patients requiring coagulation factor replacement. The pilot study results will also be used in sample size calculations and to aid detection of any early safety issues. Presenter: Keyvan Karkouti, MD, MSc, FRCPC, Toronto General Hospital, University of Toronto, Canada.
Fibryga®, Fibrinogen (Human) Lyophilized Powder for Reconstitution, for Intravenous Use, is a human fibrinogen concentrate indicated for the treatment of acute bleeding episodes in adults and adolescents with congenital fibrinogen deficiency, including afibrinogenemia and hypofibrinogenemia. Fibryga® is not indicated for dysfibrinogenemia. Fibryga® is stored as a lyophilized powder at room temperature or refrigerated temperature [+2°C to + 25°C (36°F to 77°F)] for up to 30 months from the date of manufacture. The product can be reconstituted with sterile water within approximately 5 to 10 minutes with the provided Octajet transfer device and particle filter (17-micron).
Fibryga® is contraindicated in individuals who have manifested severe immediate hypersensitivity reactions, including anaphylaxis, to fibryga® or its components.
WARNINGS AND PRECAUTIONS
Monitor patients for early signs of hypersensitivity or allergic reactions. If necessary, discontinue administration and institute appropriate treatment.
Thrombotic events have been reported in patients receiving human fibrinogen concentrate. Treatment with human fibrinogen concentrate has been associated with thrombosis at target plasma fibrinogen levels that were below 150 mg/dL. The thrombotic risks may be greater when the target fibrinogen plasma level is 150 mg/dL. Weigh the benefits of administration versus the risks of thrombosis.
Fibryga® is made from pooled human plasma. Products made from human plasma may contain infectious agents, e.g., viruses and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
The fibryga® manufacturing process includes a solvent/detergent (S/D) step for virus inactivation, and a nanofiltration step (Planova 20N nanofilter or Pegasus SV4 nanofilter) for virus removal. For complete prescribing information, please visit fibrygausa.com.
Octaplas™ is a solvent/detergent (S/D) treated, pooled human plasma indicated for replacement of multiple coagulation factors in patients with acquired deficiencies due to liver disease or who are undergoing cardiac surgery or liver transplantation; and plasma exchange in patients with thrombotic thrombocytopenic purpura (TTP).
WARNINGS AND PRECAUTIONS
Transfusion reactions can occur with ABO blood group mismatches; high infusion rates can induce hypervolemia with consequent pulmonary edema or heart failure; excessive bleeding due to hyperfibrinolysis can occur due to low levels of alpha 2-antiplasmin; thrombosis can occur due to low levels of Protein S; citrate toxicity can occur with volumes exceeding one milliliter of Octaplas™ per kg per minute; Octaplas™ is made from human blood and may carry the risk of transmitting infectious agents, e.g., viruses and theoretically, the variant Creutzfeldt-Jakob disease and Creutzfeldt-Jakob disease agent.
For complete prescribing information, please visit octaplasus.com.
About the Octapharma Group
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein products manufacturers in the world and has been committed to patient care and medical innovation since 1983. Its core business is the development and production of human proteins from human plasma and human cell lines. Octapharma employs more than 10,000 people worldwide to support the treatment of patients in over 115 countries with products across the following therapeutic areas: Hematology (coagulation disorders), Immunotherapy (immune disorders) and Critical Care. The company’s American subsidiary, Octapharma USA, is located in Paramus, N.J. Octapharma operates three state-of-the-art production sites licensed by the U.S. Food and Drug Administration (FDA), providing a high level of production flexibility. For more information, please visit www.octapharmausa.com.