Octapharma USA presents bleeding disorders research at virtual ASH annual meeting

December 01, 2020

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

Yankee Public Relations

Related Hemophilia Articles from Brightsurf:

A new cell & gene therapy approach to treat common bleeding disorder
WFIRM researchers have developed an optimized cellular platform for delivering Factor 8 to better treat patients with hemophilia A.

Early treatment linked to reduced joint damage in young adults with severe hemophilia A
Children with severe hemophilia A who receive early prophylaxis are less likely to have joint damage in young adulthood than those who begin treatment later, according to a new study in Blood Advances.

Hemophilia three times more prevalent than thought
For the World Federation of Hemophilia, Iorio assembled an international team of researchers from France, US and UK to perform a meta-analysis of the registry data in countries with the most comprehensive registries of hemophilia, which were Australia, Canada, France, Italy, New Zealand and the UK.

Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia
Hematology researchers have further refined how a treatment currently used on an urgent basis to control bleeding in hemophilia patients holds promise as a preventive treatment as well.

Single injection treats hemophilia B for life, in proof-of-concept study
Salk researchers have demonstrated in mice that hemophilia B can be treated for life with one single injection containing disease-free liver cells that can produce their missing clotting factor.

Groundbreaking gene therapy trial set to cure hemophilia
A 'cure' for hemophilia is one step closer, following results of a groundbreaking gene therapy trial led by Queen Mary University of London and the NHS in London.

Inflammatory factors linked to inhibition of factor VIII gene therapy in hemophilia A
As a gene therapy cure for hemophilia A moves closer to reality, a new study sheds light on a challenging complication in which a host autoimmune response inhibits the production of normal clotting factor VIII from the transferred FVIII gene.

Spark Therapeutics and Pfizer announce publication in The New England Journal of Medicine of Interim Data from phase 1/2 clinical trial of investigational gene therapy for Hemophilia B
Spark Therapeutics (NASDAQ: ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, and Pfizer Inc.

Disruptive technology for the treatment of hemophilia
An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87 percent.

Researchers improve vbectors for delivering hFVIII gene therapy to treat Hemophilia A
A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward into development.

Read More: Hemophilia News and Hemophilia Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.