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Takeda to Present Rare Bleeding Disorders Research at Thrombosis & Hemostasis Summit of North America (THSNA) 2020

October 27, 2020
Cambridge, Mass. and Osaka, Japan, October 27, 2020 – Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented four abstracts at the Thrombosis and Hemostasis Summit of North America (THSNA), highlighting Takeda’s commitment to advancing personalized care in people living with bleeding disorders.

These studies may help provide further data to healthcare providers regarding treatment personalization in patients with hemophilia and von Willebrand’s disease (VWD).

“When living with a bleeding disorder that is complex, rare and lifelong, it is critically important to strive for personalized care, to help patients find an appropriate therapy option for them.” states Dr. Jonathan C. Roberts, MD, Associate Medical Director, Associate Research Director at the Bleeding & Clotting Disorders Institute, and Assistant Professor of Pediatrics, University of Illinois College of Medicine at Peoria, Peoria, Ill. “The opportunity for therapeutic individualization with factor products provides hope that bleeding disorder patients can live life with treatment plans that help to address their clinical needs.”

Takeda’s presence at THSNA 2020 includes the following poster presentations, which are intended for scientific discussion only:

Hemophilia A and B

  • A phase 3b, open-label, multicenter, continuation study of rurioctocog alfa pegol prophylaxis in previously treated patients with severe hemophilia A: clinical outcomes in patients with target joints by age group
    • This phase 3b Continuation study (NCT01945593) evaluated the clinical course and outcomes of patients with target joints across different age groups treated with extended half-life PEGylated recombinant FVIII, rurioctocog alfa pegol (TAK-660). 1
  • Implementing GOAL-Hēm, a Goal Attainment Scaling Instrument
    • Goal Attainment Scaling (GAS) is an individualized outcome measure for enabling patients and caregivers to identify and track goals. GOAL-Hēm, a hemophilia-specific goal menu categorized in 3 domains (hemophilia management, complications, and impact on life), was studied in patients to evaluate whether its use influenced time to complete goal-setting vs. traditional opened-ended GAS interviews, and to learn patient and caregiver implementation preferences. 2
  • Efficacy and Safety Results from a Phase 3, Randomized, Multicenter Study of Rurioctocog Alfa Pegol Pharmacokinetic-Guided Prophylaxis Targeting Two Factor VIII Trough Levels in Patients With Severe Hemophilia A (PROPEL Study): A Consideration for Personalized Prophylaxis
    • This phase 3, randomized, multicenter study investigated the clinical impact of pharmacokinetic (PK)-guided prophylaxis with extended half-life recombinant factor VIII (rFVIII) rurioctocog alfa pegol (TAK-660) targeting 1-3% or 8-12% FVIII troughs and the related increased FVIII exposure in previously treated patients with severe hemophilia A. 3

Von Willebrand Disease

  • Population Pharmacokinetics of Recombinant von Willebrand Factor in Patients with von Willebrand Disease or Severe Hemophilia A
    • This study aims to characterize the population pharmacokinetics (PK) of rVWF and predict effects such as VWF activity in patients with VWD or hemophilia A. This population PK model represents the first stage in development of a pharmacokinetic/pharmacodynamic model to describe VWF activity. Development of a population PK/PD model may help support personalized rVWF dosing strategies for patients with this heterogenous disease. 4


ADYNOVATE Professional Important Information10

ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] Important Information

Indications and Limitation of Use


ADYNOVATE is a human antihemophilic factor indicated in children and adults with hemophilia A (congenital factor VIII deficiency) for:

  • On-demand treatment and control of bleeding episodes
  • Perioperative management
  • Routine prophylaxis to reduce the frequency of bleeding episodes

ADYNOVATE is not indicated for the treatment of von Willebrand disease.




Prior anaphylactic reaction to ADYNOVATE, to the parent molecule (ADVATE [Antihemophilic Factor (Recombinant)]), mouse or hamster protein, or excipients of ADYNOVATE (e.g. Tris, mannitol, trehalose, glutathione, and/or polysorbate 80).


Hypersensitivity Reactions

Hypersensitivity reactions are possible with ADYNOVATE. Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with other recombinant antihemophilic factor VIII

products, including the parent molecule, ADVATE. Early signs of hypersensitivity reactions that can

progress to anaphylaxis may include angioedema, chest tightness, dyspnea, wheezing, urticaria, and pruritus. Immediately discontinue administration and initiate appropriate treatment if hypersensitivity reactions occur.

Neutralizing Antibodies

Formation of neutralizing antibodies (inhibitors) to factor VIII can occur following administration of ADYNOVATE. Monitor patients regularly for the development of factor VIII inhibitors by appropriate clinical observations and laboratory tests. Perform an assay that measures factor VIII inhibitor concentration if the plasma factor VIII level fails to increase as expected, or if bleeding is not controlled with expected dose.


The most common adverse reactions (≥1% of subjects) reported in the clinical studies were headache and nausea.


Prescribing Information: https://www.shirecontent.com/PI/PDFs/ADYNOVATE_USA_ENG.pdf


VONVENDI Professional Important Information11

VONVENDI [von Willebrand factor (recombinant)] Important Information


VONVENDI [von Willebrand factor (recombinant)] is a recombinant von Willebrand factor (rVWF) indicated for use in adults (age 18 and older) diagnosed with von Willebrand disease (VWD) for:

  • On-demand treatment and control of bleeding episodes
  • Perioperative management of bleeding


Detailed Important Risk Information


Do not use in patients who have had life-threatening hypersensitivity reactions to VONVENDI or its components (tri-sodium citrate-dihydrate, glycine, mannitol, trehalosedihydrate, polysorbate 80, and hamster or mouse proteins).


Embolism and Thrombosis

Thromboembolic reactions, including disseminated intravascular coagulation, venous thrombosis, pulmonary embolism, myocardial infarction, and stroke, can occur, particularly in patients with known risk factors for thrombosis, including low ADAMTS13 levels. Monitor for early signs and symptoms of thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis, and syncope, and institute prophylaxis measures against thromboembolism based on current recommendations.

In patients requiring frequent doses of VONVENDI in combination with recombinant factor VIII, monitor plasma levels for FVIII:C activity because sustained excessive factor VIII plasma levels can increase the risk of thromboembolic events.

One out of 80 subjects treated with VONVENDI in clinical trials developed proximal deep vein thrombosis in perioperative period after total hip replacement surgery.

Hypersensitivity Reactions

Hypersensitivity reactions have occurred with VONVENDI. These reactions can include anaphylactic shock, generalized urticaria, angioedema, chest tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia, pruritus, restlessness, blurred vision, wheezing and/or acute respiratory distress. Discontinue VONVENDI if hypersensitivity symptoms occur and administer appropriate emergency treatment.

Neutralizing Antibodies (Inhibitors)

Inhibitors to VWF and/or factor VIII can occur. If the expected plasma levels of VWF activity (VWF:RCo) are not attained, perform an appropriate assay to determine if anti-VWF or antifactory VIII inhibitors are present. Consider other therapeutic options and direct the patient to a physician with experience in the care of either VWD or hemophilia A.

In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI therapy may not be effective and infusion of this protein may lead to severe hypersensitivity reactions. Since inhibitor antibodies can occur concomitantly with anaphylactic reactions, evaluate patients experiencing an anaphylactic reaction for the presence of inhibitors.


In clinical trials, the most common adverse reactions observed in ≥2% of subjects (n=80) were generalized pruritus, vomiting, nausea, dizziness, and vertigo. One subject treated with VONVENDI in perioperative setting developed deep vein thrombosis after total hip replacement surgery.


Please see VONVENDI full Prescribing Information: 


About Hemophilia
Hemophilia is a challenging chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.5, 10, 11 Hemophilia A is more common than hemophilia B; hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.6 People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment. Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.5,6

About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population. VWD is caused by a deficiency or dysfunction of von Willebrand factor (VWF), one of several types of proteins in the blood that are needed to facilitate proper blood clotting.7 Due to this defect or deficiency in VWF, blood is not able to clot effectively in people with VWD, which may result in heavy menstrual periods, easy bruising or frequent nose bleeds. Bleeding caused by VWD varies greatly among patients with this disease.8, 11

About Takeda Hematology  

Takeda is a leader in hemophilia with the longest heritage and a market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years of experience driving innovation for patients and a broad portfolio of 11 products across multiple bleeding disorders.9 Our experience as leaders in hematology means we are well prepared to meet today’s needs as we pursue future developments in the treatment of bleeding disorders. Together with the hematology community, we are committed to raising expectations for the future, including earlier diagnosis, earlier and better protection against bleeds, and more personalized patient care.

About Takeda Pharmaceutical Company

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.


For more information, visit https://www.takeda.com.




David Murdoch

[email protected]

+1 781-421-1741






  1. Chowdary et al., (2019). A phase 3b, open-label, multicenter, Continuation study of rurioctocog alfa pegol prophylaxis in previously treated patients with severe hemophilia A: clinical outcomes in patients with target joints by age group. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #147.
  2. Stanley et al., (2019). Implementing GOAL-Hēm, a Goal Attainment Scaling Instrument. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #14.
  3. Radulescu, et al., (2019). Efficacy and Safety Results from a Phase 3, Randomized, Multicenter Study of Rurioctocog Alfa Pegol Pharmacokinetic-Guided Prophylaxis Targeting Two Factor VIII Trough Levels in Patients With Severe Hemophilia A (PROPEL Study): A Consideration for Personalized Prophylaxis. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #15.
  4. Bauer, et al., (2019). Population Pharmacokinetics of Recombinant von Willebrand Factor in Patients with von Willebrand Disease or Severe Hemophilia A. In proceedings from the Thrombosis & Hemostasis Societies of North America; October 27-30, 2020; Chicago, IL. Abstract #125.
  5. World Federation of Hemophilia. “What is hemophilia?” World Federation of Hemophilia website. http://www.wfh.org/en/page.aspx?pid=646. Last Accessed April 2019.
  6. World Federation of Hemophilia. Report on the Annual Global Survey 2017. World Federation of Hemophilia website. http://www1.wfh.org/publications/files/pdf-1714.pdf Last Accessed April 2019.
  7. National Hemophilia Foundation. "Von Willebrand Disease." National Hemophilia Foundation website. https://www.hemophilia.org/Bleeding-Disorders/Types-of-Bleeding-Disorders/Von-Willebrand-Disease.  Last accessed January 2020. 
  8. National Hemophilia Foundation. "VWD Summit Highlights." HEMAWARE website. https://hemaware.org/story/vwd-summit-highights . Last accessed January 2020.
  9. Takeda Website. Rare Diseases. Website: https://www.takeda.com/what-we-do/areas-of-focus/rare-diseases/ Last Accessed June 2020.
  10. ADYNOVATE Professional Important Information, Takeda. November 2017.
  11. VONVDENDI Professional Important Information, Takeda. January 2019.


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