Seven Year Data Reinforce Long-term Efficacy and Safety Review of ADVATE (Antihemophilic Factor [Recombinant])
Abstracts Support Use of Real World Evidence to Advance Treatment and Patient Outcomes
Takeda’s Heritage in Bleeding Disorders and Commitment to Investing in Innovation Support Our Passion for Helping Patients and Our Vision of a Bleed-free World
OSAKA, Japan -- (BUSINESS WIRE) --
Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE:TAK) (“Takeda”) today announced that 14 abstracts from the company’s Hematology portfolio and pipeline are being presented as electronic posters at the Virtual European Association for Haemophilia and Allied Disorders Congress (EAHAD 2021). Data include multiple abstracts reinforcing the long-term efficacy and safety of Takeda’s hematology portfolio in clinical practice, supporting the value of these treatments in a real world setting.
Seven year AHEAD study data [ABS034] showed that prophylactic antihemophilic factor (recombinant), rAHF (ADVATE), achieved lower annualized bleeding rates (ABRs) and annualized joint bleeding rates (AJBRs) than on-demand treatment in all patients with severe hemophilia A. Key outcomes included ABRs (all bleeds), AJBRs, joint health (evaluated by Gilbert score), and adverse events (AEs). AEs occurred in 414/702 (59.0%) patients (serious AEs in 141/702). 12 patients developed de novo FVIII inhibitors (high titer [>5 BU], n=2 [transient]; low titer [≤5 BU], n=10 [1 transient, 9 persistent].1 A separate analysis of patients with moderate or severe hemophilia A and target joints [ABS170] showed that prophylactic rAHF maintained lower bleed rates than on-demand treatment over seven years.2
A further retrospective study [ABS039] investigated the impact of switching patients with moderate or severe hemophilia A in US clinical practice (without inhibitors) from Factor VIII prophylaxis to rurioctocog alfa pegol (ADYNOVATE) or emicizumab. Results showed that there were no statistically significant differences in prophylactic effectiveness between treatments.3
“For rare diseases such as hemophilia, gathering evidence from clinical practice is crucial to advance treatment and reduce gaps in patient care,” commented Professor Margareth Ozelo, Director of the WFH International Haemophilia Training Centre, University of Campinas, Brazil. “These data add to the body of evidence for the management of hemophilia, and reinforce the value of using evidence from real world settings to personalized care, improve patient outcomes, and address the unique needs of every single patient.”
Additional abstracts presented at EAHAD 2021 further reinforce the efficacy and safety of Takeda’s portfolio for both on-demand and prophylaxis treatment in real world settings [ABS093, ABS113, ABS181, ABS070, ABS112], illustrate the benefit of PK-guided, personalized prophylaxis to help support patient outcomes in hemophilia [ABS120, ABS190], and demonstrate our ongoing commitment to innovation in hematology [ABS185].
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About Hemophilia
Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.4 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.5
People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.6 Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.7
About Takeda Hematology
Following its recent acquisition of Shire, Takeda is a leader in hemophilia 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 patients8 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 care of bleeding 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.
E.U. Indication and Important Safety Information
About ADVATE (human coagulation factor VIII (rDNA), octocog alfa)
Please consult the ADVATE Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring (here).
Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in the SmPC or to mouse or hamster proteins.
Special warnings and precautions for use
The product contains traces of mouse and hamster proteins. If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the product immediately and contact their physician.
Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension and anaphylaxis.
The formation of neutralising antibodies (inhibitors) to factor VIII is a known complication in the management of individuals with haemophilia A. These inhibitors are usually IgG immunoglobulins directed against the factor VIII procoagulant activity, which are quantified in Bethesda Units (BU) per mL of plasma using the modified assay.
In general, all patients treated with coagulation factor VIII products should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests. If the expected factor VIII activity plasma levels are not attained, or if bleeding is not controlled with an appropriate dose, testing for factor VIII inhibitor presence should be performed.
After reconstitution this medicinal product contains 0.45 mmol sodium (10 mg) per vial.
Adverse Reactions
|
Very common (≥1/10) |
Factor VIII inhibition in previously untreated patients (PUPs) |
|
Common (≥1/100 to <1/10) |
Headache, Pyrexia |
|
Uncommon (≥1/1000 to <1/100) |
Influenza, Laryngitis, Factor VIII inhibition in previously treated patients (PTPs), Lymphangitis, Dizziness, Dysgeusia, Memory impairment, Migraine, Syncope, Tremor, Eye inflammation, Palpitations, Haematoma, Hot flush, Pallor, Dyspnoea, Abdominal pain upper, Diarrhoea, Nausea, Vomiting, Hyperhidrosis, Pruritus, Rash, Urticaria, Chest discomfort, Chest pain, Chills, Feeling abnormal, Periheral oedema, Vessel puncture site haematoma, Coagulation factor VIII level decreased, Haematocrit decreased, Laboratory test abnormal, Monocyte Count increased, Post procedural complication, Post procedural haemorrhage, Procedural site reaction |
|
Not known |
Anaphylactic reaction, Hypersensitivity, Fatigue, Injection site reaction, Malaise |
For the ADVATE US Prescribing Information, please refer here.
About ADYNOVATE/ADYNOVI®▼ (Pegylated human coagulation factor VIII (rDNA), rurioctocog alfa pegol)
Detailed Safety Information
Please consult the ADYNOVI Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring (here)
Contraindications
Hypersensitivity to the active substance, to the parent molecule octocog alfa or to any of the excipients listed in the SmPC. Known allergic reaction to mouse or hamster protein.
Special warnings and precautions for use
The medicinal product contains traces of mouse and hamster proteins. If symptoms of hypersensitivity occur, patients should be advised to discontinue use of the medicinal product immediately and contact their physician. Patients should be informed of the early signs of hypersensitivity reactions including hives, generalised urticaria, tightness of the chest, wheezing, hypotension, and anaphylaxis.
The formation of neutralising antibodies (inhibitors) against factor VIII is a known complication in the management of individuals with haemophilia A. These inhibitors are usually IgG immunoglobulins directed against the factor VIII procoagulant activity, which are quantified in Bethesda Units (BU) per mL of plasma using the modified assay.
In general, all patients treated with coagulation factor VIII should be carefully monitored for the development of inhibitors by appropriate clinical observations and laboratory tests. If the expected factor VIII activity plasma levels are not attained, or if bleeding is not controlled with an appropriate dose, testing for factor VIII inhibitor presence should be performed.
After reconstitution this medicinal product contains 0.45 mmol sodium (10 mg) per vial.
Adverse Reactions
|
Common (≥1/100 to <1/10) |
Headache, Diarrhoea, Nausea, Rash |
|
Uncommon (≥1/1000 to <1/100) |
Factor VIII inhibition in previously-treated patients (PTPs), Hypersensitivity, Flushing |
For the ADYNOVATE US Prescribing Information, please refer here.
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.
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Medical information
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1 EAHAD 2021. Abstract ABS034. 7 Interim Analysis of Antihemophilic Factor (recombinant) and Joint Health in Patients with Hemophilia A: AHEAD International.
2 EAHAD 2021. Abstract ABS170. Real world Bleeding Rates over 7 Years of Antihemophilic Factor (recombinant) in Patients with Hemophilia A and Target Joints: AHEAD International
3 EAHAD 2021. Abstract ABS039. A Retrospective Study of the Impact of Switching Patients with Haemophilia A to rurioctocog alfa pegol or emicizumab in US Practice.
4 World Federation of Hemophilia. Introduction to hemophilia: what is hemophilia?. World Federation of Hemophilia website. Available at: https://bit.ly/356zUDQ. Last accessed January 2021.
5 20 Anniversary Report on the Annual Global Survey 2018. World Federation of Hemophilia. Available at: https://bit.ly/3hGDUzU. Last accessed January 2021.
6 World Federation of Hemophilia. “Introduction to hemophilia: treatment.” Available here: https://bit.ly/3b7Pfry. Last accessed January 2021.
7 NHS. Haemophilia: treatment. Available here: https://bit.ly/3oqhAwY. Last accessed January 2021.
8 Takeda Website. Rare Diseases. Available at: https://bit.ly/3n9gvIN. Last accessed January 2021.
9 Takeda Website. U.S. Product List. Available at: https://bit.ly/35a6HYF. Last accessed January 2021.
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CONTACT:
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