- Key learnings from real-world data and retrospective studies from nine abstracts are presented at the 62nd ASH Annual Meeting & Exposition, taking place virtually this year
- Takeda continues to gather and apply real-world evidence in the treatment of hemophilia, von Willebrand disease (VWD) and sickle cell disease (SCD) and advance personalized treatment in its ongoing commitment to people living with bleeding disorders
Cambridge, Mass. and Osaka, Japan, December 7, 2020 – Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”), today presented five hematology poster presentations and four abstracts at the 62nd American Society of Hematology (ASH) Annual Meeting & Exposition that underscore its commitment to advancing treatments for rare bleeding disorders.
Real-World Evidence Demonstrates Importance of Patient Centric Care
Real-world evidence (RWE) from studies across rare bleeding disorders demonstrate the crucial role understanding patterns of care and the patient’s holistic experience outside of rigorous clinical studies play in advancing patient-centric treatment in bleeding disorders. Several of Takeda’s studies presented at ASH generate real-world evidence to better understand clinical management of treatment and associated disease outcomes in hemophilia A and VWD.
“Real-world evidence allows healthcare professionals to understand how medications work in routine clinical practice and can advance patient-centric treatment in bleeding disorders,” said Dr. Michael Tarantino, CEO, Chief Medical Officer and President, The Bleeding and Clotting Disorders Institute and Professor of Pediatrics and Medicine at University of Illinois College of Medicine-Peoria. “For example, real world data presented at ASH describe patient satisfaction data with ADYNOVATE. This data help us better understandthe true patient experience when using our products.”
Insights generating RWE at ASH 2020 are captured in the following posters:
Advancing Personalized Treatment in Bleeding Disorders
In addition to a real-world evidence suite at ASH 2020, Takeda is highlighting its commitment to advancing individualized treatment in rare hematology, by highlighting the importance of a tailored dosing regimen of hemophilia based on patients’ different pharmacokinetic (PK) profiles, patient preferences in hemophilia A treatment and patient clinical demographics in sickle cell disease (SCD). For VWD, we are a long way from advancing a PK-guided dosing regimen but are looking at a variety of personal characteristics, such as bodyweight, in order to advance pharmacokinetics in VWD treatment.
“Personalized treatments provide the potential to drive enhanced outcomes while improving both the patient and HCP experience,” comments Wolfhard Erdlenbruch, M.D., PhD, Vice President, Head Global Medical Affairs Hematology. “For rare bleeding disorders, there has been transformative steps around PK modelling to build a more personalized approach to factor therapy, but the journey towards personalization is still being defined, as indicated by this year’s data presented at ASH.”
Additional insights showcased at ASH 2020 include:
ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated] was first approved by the Food and Drug Administration (FDA) in the U.S. followed by approval in Japan, Canada, and Colombia, and is approved as ADYNOVI® in the 28 Member States of the European Union (EU) as well as Iceland,
Liechtenstein, Norway and Switzerland. In Europe ADYNOVI is approved for the treatment and prophylaxis of bleeding in patients 12 years and above with hemophilia A.
ADYNOVATE Important Information
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:
ADYNOVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
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).
WARNINGS & PRECAUTIONS
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.
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.
For more information, please refer to the ADYNOVI Summary of Product Characteristics here.
For US specific safety information, please refer to the ADYNOVATE US Prescribing Information here.
ADVATE is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. ADVATE is approved in the EU for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency) in all age group.
ADVATE is currently approved in over 70 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Bahrain, Brazil, Brunei, Chile, China, Colombia, Ecuador, GCC, Hong Kong, Iceland, India, Iraq, Israel, Japan, Kazakhstan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Thailand, Tunisia, Turkey, UAE, Ukraine, Uruguay, Venezuela, Vietnam.
ADVATE Important Information
ADVATE [Antihemophilic Factor (Recombinant)] Important Information
ADVATE is a recombinant antihemophilic factor indicated for use in children and adults with
hemophilia A (congenital factor VIII deficiency) for:
ADVATE is not indicated for the treatment of von Willebrand disease.
DETAILED IMPORTANT RISK INFORMATION
Patients who have life-threatening hypersensitivity reactions, including anaphylaxis, to mouse or
hamster protein or other constituents of the product.
WARNINGS & PRECAUTIONS
Allergic-type hypersensitivity reactions, including anaphylaxis, have been reported with ADVATE.
Symptoms include dizziness, paresthesia, rash, flushing, facial swelling, urticaria, dyspnea, pruritus, and vomiting. Discontinue ADVATE if hypersensitivity symptoms occur and administer appropriate emergency treatment.
Neutralizing antibodies (inhibitors) have been reported following administration of ADVATE
predominantly in previously untreated patients (PUPs) and previously minimally treated patients
(MTPs). Monitor all patients for the development of factor VIII inhibitors by appropriate clinical observation and laboratory testing. If expected plasma factor VIII activity levels are not attained, or if bleeding is not controlled with an expected dose, perform an assay that measures factor VIII inhibitor concentration.
anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.
For more information, please refer to the ADVATE Summary of Product Characteristics here.
For US specific safety information, please refer to the ADVATE US Prescribing Information here.
VEYVONDI is indicated in adults (age 18 and older) with von Willebrand disease (VWD), when desmopressin (DDAVP) treatment alone is ineffective or not indicated for the treatment of hemorrhage and treatment/prevention of surgical bleeding. VEYVONDI should not be used in the treatment of hemophilia A.1
For full EU Summary of Product Characteristics, including approved indication(s) and important safety information about marketed products, please visit here.
In the US the product has been approved under the trade name VONVENDI® [von Willebrand factor (Recombinant)] and is indicated for use in adults (age 18 and older) diagnosed with VWD for on-demand treatment and control of bleeding episodes or perioperative management of bleeding.
VONVENDI Important Information
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:
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).
WARNINGS AND PRECAUTIONS
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 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.
For more information, please refer to the VEYVONDI Summary of Product Characteristics here.
For US specific safety information, please refer to the VONVENDI US Prescribing Information here.
About FEIBA (Factor VIII Inhibitor Bypassing Activity)
FEIBA® (factor VIII inhibitor bypassing activity) is indicated for the treatment of spontaneous bleeding and cover of surgical interventions in haemophilia A patients with factor VIII inhibitors and in non-haemophiliacs with acquired factor VIII inhibitors as well as for prophylaxis in haemophilia A patients with high responding inhibitors and frequent joint bleeding.
Please consult the FEIBA Summary of Product Characteristics (SmPC) here before prescribing, particularly in relation to dosing and treatment monitoring.
Contraindications are hypersensitivity to the product, disseminated intravascular coagulation (DIC) and acute thrombosis or embolism (including myocardial infarction).
The Adverse Drug Reactions (ADRs) occurring in the highest frequency (common, greater than or equal to 1/100 to <1/10) were hypersensitivity, headache, dizziness, hypotension, rash, hepatitis B surface antibody positive.
Other symptoms of hypersensitivity reactions to plasma-derived products include lethargy and restlessness.
FEIBA [Anti-Inhibitor Coagulant Complex] Indications and Detailed Important Risk Information
Indications for FEIBA
FEIBA is an Anti-Inhibitor Coagulant Complex indicated for use in hemophilia A and B patients with inhibitors for:
FEIBA is not indicated for the treatment of bleeding episodes resulting from coagulation factor deficiencies in the absence of inhibitors to coagulation factor VIII or coagulation factor IX.
Detailed Important Risk Information for FEIBA
WARNING: EMBOLIC AND THROMBOTIC EVENTS
FEIBA is contraindicated in patients with:
WARNINGS AND PRECAUTIONS
Thromboembolic events (including venous thrombosis, pulmonary embolism, myocardial infarction, and stroke) can occur, particularly following the administration of high doses (>200 units/kg/day) and/or in patients with thrombotic risk factors.
Patients with DIC, advanced atherosclerotic disease, crush injury, septicemia, or concomitant treatment with recombinant factor VIIa have an increased risk of developing thrombotic events due to circulating tissue factor or predisposing coagulopathy. Potential benefit of treatment should be weighed against potential risk of these thromboembolic events.
Infusion should not exceed a single dose of 100 units/kg and daily doses of 200 units/kg. Maximum injection or infusion rate must not exceed 2 units/kg/minute. Monitor patients receiving >100 units/kg for the development of DIC, acute coronary ischemia and signs and symptoms of other thromboembolic events. If clinical signs or symptoms occur, such as chest pain or pressure, shortness of breath, altered consciousness, vision, or speech, limb or abdomen swelling and/or pain, discontinue FEIBA and initiate appropriate diagnostic and therapeutic measures.
Safety and efficacy of FEIBA for breakthrough bleeding in patients receiving emicizumab has not been established. Cases of thrombotic microangiopathy (TMA) were reported in a clinical trial where subjects received FEIBA as part of a treatment regimen for breakthrough bleeding following emicizumab treatment. Consider the benefits and risks with FEIBA if considered required for patients receiving emicizumab prophylaxis. If treatment with FEIBA is required for patients receiving emicizumab, the hemophilia treating physician should closely monitor for signs and symptoms of TMA. In FEIBA clinical studies TMA has not been reported.
Hypersensitivity and allergic reactions, including severe anaphylactoid reactions, can occur. Symptoms include urticaria, angioedema, gastrointestinal manifestations, bronchospasm, and hypotension. Reactions can be severe and systemic (e.g., anaphylaxis with urticaria and angioedema, bronchospasm, and circulatory shock). Other infusion reactions, such as chills, pyrexia, and hypertension have also been reported. If signs and symptoms of severe allergic reactions occur, immediately discontinue FEIBA and provide appropriate supportive care.
Because FEIBA is made from human plasma it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent.
FEIBA contains blood group isohemagglutinins (anti-A and anti-B). Passive transmission of antibodies to erythrocyte antigens, e.g., A, B, D, may interfere with some serological tests for red cell antibodies, such as antiglobulin test (Coombs test).
Most frequently reported adverse reactions observed in >5% of subjects in the prophylaxis trial were anemia, diarrhea, hemarthrosis, hepatitis B surface antibody positive, nausea, and vomiting.
Serious adverse reactions seen are hypersensitivity reactions and thromboembolic events, including stroke, pulmonary embolism and deep vein thrombosis.
Consider possibility of thrombotic events when systemic antifibrinolytics such as tranexamic acid and aminocaproic acid are used with FEIBA. No adequate and well-controlled studies of combined or sequential use of FEIBA and recombinant factor VIIa, antifibrinolytics, or emicizumab, have been conducted. Use of antifibrinolytics within approximately 6 to 12 hours after FEIBA is not recommended.
Clinical experience from an emicizumab clinical trial suggests that a potential drug interaction may exist with emicizumab.
Please see FEIBA full Prescribing Information, including BOXED
WARNING on Embolic and Thrombotic Events
For more information, please refer to the FEIBA Summary of Product Characteristics here.
For US specific safety information, please refer to the FEIBA US Prescribing Information here.
Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.1 Hemophilia A is more common than hemophilia B; in 2018, hemophilia A affects about 158,225 people, whereas hemophilia B affects about 31,247 people worldwide.2 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.1,2
About von Willebrand disease (VWD)
VWD is the most common inherited bleeding disorder, affecting up to one percent of the U.S. population.3 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.3 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.3 Bleeding caused by VWD varies greatly among patients with this disease.4
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 Limited
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.
+81 (0) 3-3278-2095
Media outside Japan
+1 (781) 421-1741
For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.
The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.
This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could” “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: https://www.takeda.com/investors/reports/sec-filings/ or at www.sec.gov. Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results.