Octapharma USA presents bleeding disorders research at virtual ASH annual meeting


PARAMUS, N.J. (December 1, 2020) – Octapharma USA will present its most recent rare bleeding disorders research initiatives during the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, a virtual medical congress to be held December 5 – 8.

Three scientific posters will be presented during the ASH meeting that focus on investigative treatment options utilizing NUWIQ®, Antihemophilic Factor (Recombinant) Lyophilized Powder for Solution for Intravenous Injection and WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection.

“Octapharma is committed to advancing clinical research that will help enhance the lives of people with bleeding disorders,” said Octapharma USA President Flemming Nielsen. “We look forward to sharing the latest research developments with the medical community.”

Ellis J. Neufeld, MD, PhD, of St. Jude Children’s Research Hospital, Memphis, Tenn., will provide oral and poster abstracts for “Long-Term Prophylaxis with Simoctocog Alfa for the Management of Hemophilia A: Immunogenicity, Efficacy, and Safety Results from the NuProtect PUP Extension Study.” The research is focused on evaluating the risk of factor VIII (FVIII) inhibitor development in pediatric patients during FVIII treatment. Prophylaxis with FVIII is the gold standard approach for management of hemophilia A, and long-term bleed prevention remains the key goal for patients and clinicians. The NuProtect extension study assessed the long-term immunogenicity, hemostatic efficacy and tolerability of NUWIQ® in 48 pediatric patients. No children with severe hemophilia A developed FVIII inhibitors during the study. Principal investigators: Ri Liesner, MD, Haemophilia Comprehensive Care Centre, Great Ormond Street Hospital for Children, London, United Kingdom, and Dr. Neufeld.

Another poster is entitled “Use of a Von Willebrand Factor/Coagulation Factor VIII Complex for Treatment of Refractory Inherited Platelet Disorders.” Inherited platelet disorders are recognized as an important cause of mild to severe bleeding in both children and adults. Treatment of platelet disorders is mainly supportive. Normal hemostasis requires von Willebrand factor (VWF) and FVIII to support platelet adhesion and aggregation at sites of vascular injury. Four patients with qualitative platelet disorders, three with delta storage pool deficiencies and one with Bernard-Soulier syndrome achieved clinically relevant bleeding resolution with WILATE®. Notably, these patients were refractory to other therapies. Principal investigator: Amber Federizo, APRN, FNP-BC, Hemostasis and Thrombosis Center of Nevada, Las Vegas, Nev.

Design of the Von Willebrand Factor in Pregnancy (VIP) Study” is a description of planned VWF in pregnancy research. This study will provide a better understanding of post-partum hemorrhage in women with von Willebrand disease (VWD) relative to VWD diagnosis, VWF levels, and other laboratory assessments of VWF associated parameters. The study evaluates the effectiveness of WILATE® in targeting VWF/FVIII during delivery and the immediate 72-hour postpartum period. Principal investigator: Jill M. Johnsen, MD, Bloodworks Research Institute and Department of Medicine, University of Washington, both in Seattle, Wash.

For more information on the ASH annual meeting, please visit hematology.org.


About NUWIQ®

NUWIQ®, Antihemophilic Factor (Recombinant) Lyophilized Powder for Solution for Intravenous Injection is a recombinant antihemophilic factor [blood coagulation factor VIII (Factor VIII)] indicated in adults and children with Hemophilia A for: on-demand treatment and control of bleeding episodes; perioperative management of bleeding; and routine prophylaxis to reduce the frequency of bleeding episodes. NUWIQ® is not indicated for the treatment of von Willebrand Disease.


NUWIQ® is contraindicated in patients who have manifested life-threatening hypersensitivity reactions, including anaphylaxis, to the product or its components.


Hypersensitivity reactions, including anaphylaxis, are possible. Should symptoms occur, discontinue NUWIQ® and administer appropriate treatment. Development of Factor VIII neutralizing antibodies (inhibitors) may occur. If expected plasma Factor VIII activity levels are not attained, or if bleeding is not controlled with an appropriate dose, perform an assay that measures Factor VIII inhibitor concentration. Monitor all patients for Factor VIII activity and development of Factor VIII inhibitor antibodies.

For complete prescribing information, please visit nuwiqusa.com.


WILATE®, von Willebrand Factor/Coagulation Factor VIII Complex (Human) Lyophilized Powder for Solution for Intravenous Injection, is indicated in children and adults with von Willebrand disease for on-demand treatment and control of bleeding episodes; and perioperative management of bleeding. WILATE® is indicated in adolescents and adults with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes; and on-demand treatment and control of bleeding episodes.


Do not use in patients with known hypersensitivity reactions, including anaphylactic or severe systemic reaction, to human plasma-derived products, any ingredient in the formulation, or components of the container.


Anaphylaxis and severe hypersensitivity reactions are possible; thromboembolic events may occur; monitor plasma levels of FVIII activity; development of neutralizing antibodies to FVIII and to VWF, especially in VWD type 3 patients, may occur; WILATE® is made from human plasma and carries the risk of transmitting infectious agents.

For complete prescribing information, please visit wilateusa.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 http://www.octapharmausa.com.


Anita Callari

Yankee Public Relations

[email protected]


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