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Takeda Drives Continued Scientific Leadership Through Real-World Evidence in Rare Hematological Diseases at ASH 2021

- Nine abstracts across Takeda’s hematology portfolio and pipeline demonstrate Takeda’s commitment to patient-centric, innovative care that aims to drive greater treatment outcomes

- Includes data exploring ADYNOVATE® [Antihemophilic Factor (Recombinant), PEGylated] in previously untreated patients (PUPs) with severe hemophilia A and an exploratory joint health biomarker analysis of the PROPEL study

- Design of a phase 3b clinical study will be published. The study will evaluate the safety and efficacy of TAK-755: the first and only investigational rADAMTS13 replacement therapy in clinical development.

- Design of the first clinical study examining TAK-755’s therapeutic potential in sickle cell disease (SCD)

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced the presentation of nine company-sponsored abstracts at the 63rd American Society of Hematology (ASH) Annual Meeting being held December 11-14, 2021 in Atlanta, Georgia and virtually. The abstracts span Takeda’s extensive hematology portfolio and research and development (R&D) pipeline, featuring data on multiple products and investigational candidates, supporting Takeda’s commitment to helping healthcare professionals improve the way they treat patients as well as delivering innovative solutions for the future.

“At Takeda, we are advancing a promising pipeline of potentially transformative molecules for multiple rare and life-threatening hematological disorders, while also expanding the body of evidence to support our current portfolio of treatments for people living with rare bleeding disorders,” said Neil Inhaber, Head, Global Medical Affairs, Rare Genetics and Hematology. “The data presented at this year’s ASH annual meeting demonstrate Takeda’s commitment to advancing the science in hematology and personalized treatment strategies that aim to address many different needs within the bleeding disorders community.”

Highlights of data to be presented during ASH include:

ADYNOVATE® [Antihemophilic Factor (Recombinant), PEGylated] data in previously untreated patients with severe hemophilia A

Data from the first prospective study evaluating the safety, immunogenicity, and efficacy of ADYNOVATE in previously untreated patients (PUPs), including those younger than six years old, with severe hemophilia A will be presented [Abstract #3184].1

Analysis of joint health from the PROPEL study

Takeda will present post-hoc data from a randomized clinical trial in severe hemophilia A patients on FVIII prophylaxis —the PROPEL study. Six different biomarkers of joint health were measured in 98 patients before and after prophylactic treatment with ADYNOVATE [Abstract #2102].2

This novel biomarker data is the first in a randomized clinical trial to understand the importance of prophylaxis for joint health in patients with hemophilia A, as well as the first study examining longitudinal biomarker levels in a prospective randomized study of severe hemophilia A patients undergoing FVIII prophylaxis at two trough levels (1-3% and 8-12%) to examine the correlation between clinical outcomes and surrogate markers. The results also showed improvement in specific biomarker levels from baseline to study completion [Abstract #2102].2

A cost analysis of prophylaxis with factor VIII replacement and emicizumab in real-world settings

Real-world evidence (RWE) allows healthcare professionals to better understand how medications work in routine clinical practice, potentially advancing patient-centric treatment, particularly in hematological disorders. Takeda is presenting real-world data extracted from the IQVIA PharMetrics® Plus database (2015-2020), a longitudinal U.S. commercial health plan database, analyzing prophylaxis with factor VIII (FVIII) replacement and emicizumab in billed annualized bleed rate and all-cause cost of switching from FVIII to emicizumab [Abstract #3028].3

“This real-world study helps to illuminate important therapeutic and cost impacts of different treatment options for patients with hemophilia A,” commented study author, Katharine Batt, MD, MSc. “These findings are incredibly important for healthcare providers, as the data can begin to help them better understand different treatment options, the long-term implications of use and the best treatment fit for their patients, thereby offering a more personalized form of patient care.”

Design of studies further exploring the therapeutic potential of recombinant ADAMTS13 replacement (TAK-755) in cTTP and sickle cell disease (SCD)

Takeda is continuing to assess the potential of TAK-755, a recombinant ADAMTS13 replacement therapy, in patients with severe cTTP, an ultra-rare and potentially life-threatening ADAMTS13 deficiency driven thrombotic disorder.4 The design of a phase 3b open-label continuation study that aims to evaluate the long-term safety and efficacy of TAK-755 for prophylactic and on-demand treatment of severe cTTP will be made available in the ASH program [accepted for online publication only].4

Takeda will also present the design of the first phase 1 study assessing safety, tolerability, pharmacokinetics, and pharmacodynamics of recombinant ADAMTS13 (TAK-755) in patients with SCD [Abstract #3118].5

rADAMTS13 (TAK-755), is an investigational compound that has not been approved for use by U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), or any other regulatory authorities.

The Takeda Hematology team is proud to, once again, have a significant presence at ASH along with Takeda Oncology. A full list of all company-sponsored abstracts can be found here.

About Hemophilia

Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.6 Hemophilia A is more common than hemophilia B; in 2018, hemophilia A affects about 173,711 people, whereas hemophilia B affects about 34,289 people worldwide.7

People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.8 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.9

About Takeda Hematology

Takeda is a leader in bleeding disorders with the longest heritage and market-leading portfolio, backed by established safety and efficacy profiles with decades of real-world experience. We have 70+ years driving innovation for patients10 and a broad portfolio of 11 products across multiple bleeding disorders. Our experience as a leader in hematology means we are well prepared to meet today’s needs as we pursue future developments in the care of blood disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and full protection against bleeds, and more personalized patient care.

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, diarrhea, rash, nausea, dizziness and urticaria.

Click here for Full Prescribing Information.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetic and Hematology, 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

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1 ASH 2021. Abstract [3184]. Immunogenicity, Efficacy and Safety of Rurioctocog Alfa Pegol in Previously Untreated Patients with Severe Hemophilia: Interim Results from an Open-Label Multicenter Clinical Trial.

2 ASH 2021. Abstract [2102]. Prophylactic Treatment with Rurioctocog Alfa Pegol Results in a Dose-Dependent Normalization of Biomarkers of Joint Health in Severe Hemophilia a: An Exploratory Analysis from the Propel Study.

3 ASH 2021. Abstract [3028]. A Real-world Study of Pre-Post Annualized Bleed Rates and All Cause Costs Among Non-inhibitor Patients with Hemophilia A Switching from FVIII Prophylaxis to Emicizumab.

4 Blood (2021) 138 (Supplement 19). Recombinant ADAMTS13 for Patients with Severe Congenital Thrombotic Thrombocytopenic Purpura: Design of a Phase 3b Open-Label Continuation Study of Prophylactic and On-demand Treatment.

5 ASH 2021. Abstract [3118]. Recombinant ADAMTS13 for Patients with Sickle Cell Disease: Design of a Phase 1 Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics study.

6 World Federation of Hemophilia. Introduction to hemophilia: what is hemophilia?. World Federation of Hemophilia website. Available at: Last accessed January 2021.

7 20 Anniversary Report on the Annual Global Survey 2018. World Federation of Hemophilia. Available at: Last accessed June 2021.

8 World Federation of Hemophilia. “Introduction to hemophilia: treatment.” Available here: Last accessed June 2021.

9 NHS. Haemophilia: treatment. Available here: Last accessed June 2021.

10 Takeda Website. Rare Diseases. Available at: Last accessed June 2021.


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