− These Data Demonstrate Breadth of Takeda’s Portfolio and Pipeline with Three Hematology Presentations at the Virtual Summit
OSAKA, Japan-Monday 15 June 2020 [ AETOS Wire ]
(BUSINESS WIRE)-- Takeda Pharmaceutical Company Limited, (“Takeda”) (TSE: 4502/NYSE:TAK) today announced a scientific update from the AHEAD real-world study investigating the long-term outcomes associated with ADVATE [antihemophilic factor (recombinant), rAHF] in patients with hemophilia A, presented as an oral presentation at the World Federation of Hemophilia Virtual Summit 2020 (WFH 2020). The update is one of the three abstracts being presented at WFH 2020 from Takeda’s Hematology portfolio and pipeline. Echoing Takeda’s theme at the virtual summit – “It’s Personal” – the company also shared data from a physician survey in China, providing insights into optimizing personalized care and hemophilia patient management in centers of excellence (COE).
“It’s Personal”: Takeda is relentless in its pursuit of a bleed-free world
“Takeda’s vision for a bleed-free world is as important today as ever,” commented Dr. med. Wolfhard Erdlenbruch, M.D., Vice President Head of Global Medical Affairs Hematology, Takeda. “The data we presented at WFH 2020 continue to add to the body of evidence for the management of hemophilia, a bleeding disorder that affects at least 210,000 people worldwide as of 2018 and potentially a lot more.i,ii As a leader in Hematology, Takeda is committed to improving patient outcomes, and the findings of our studies demonstrate the value personalized strategies can offer to patients as part of the ongoing management of their bleeding disorder.”
Prophylaxis for hemophilia A is the standard of care treatment for patients because it can help prevent spontaneous bleeds, as even a single bleed may cause joint damage and impact their quality of life.iii,iv
Interim analysis results from the AHEAD real-world outcomes study [abstract MED‐FP‐005 (363)] demonstrate that the number of hemophilia A patients who were able to achieve zero bleeds increased over the years by receiving rAHF. For those receiving prophylaxis, the number of patients with zero bleeds increased from 34% in year 1 to 53% in year 6. For those receiving on-demand treatment, it increased from 28% in year 1 to 38% in year 6. The results also show that those receiving rAHF prophylaxis generally reported slightly better health-related quality of life (HRQoL) than those receiving rAHF on-demand. The results support the role of rAHF as an important option for hemophilia A, a condition which requires life-long monitoring and management.v
“The results of the interim analysis from the AHEAD real-world outcomes study support the long-term effectiveness of rAHF for hemophilia A patients, which was maintained in a six-year observational period and bolstered by consistent HRQoL scores,” added Prof. Dr. med. Johannes Oldenburg, Chairman and Director, Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Germany, who is the researcher leading the study. “Every patient is unique, and these results support a tailored and personalized approach to optimize care for patients with bleeding disorders, delivered in partnership with their treating physician.”
Takeda aims to help advance more personalized, connected care for patients
Takeda presented two additional abstracts at WFH 2020 – available on the summit’s iPoster Gallery (within the ‘Open to all’ and ‘HCP’ sections respectively) – supporting its personalized care approach for patients with hemophilia.
Multi-department collaboration for the management of hemophilia Avi [iPoster MTD-PO-057]
A substantial number of patients with hemophilia A live in China, due to the large population.i Results from a physician survey explored the Center of Excellence (COE) model for the optimization of multi-department collaboration for patients with hemophilia A in China; low patient awareness of the need for treatment/prophylaxis and fewer than 40% of patients received prophylaxis, demonstrated the need for improvements in both public and physician awareness and knowledge of hemophilia. Results showed that a COE model may improve collaboration across departments, optimize patient management and thereby improve outcomes.
Real-world safety and effectiveness of Rixubis in patients with hemophilia B in South Koreavii [iPoster MED-PO-018]
Data from a prospective, post-marketing surveillance study evaluating the safety and efficacy profile of Rixubis [Nonacog gamma, recombinant FIX concentrate] demonstrated the safety and efficacy profile for bleeds, perioperative/surgery, and prophylaxis in adult and pediatric patients with hemophilia B in the real-world setting in South Korea. The study showed that 86.6% (123/142) of hemostatic effectiveness assessments for nonacog gamma were reported as good or excellent, and of the 11 adverse events reported, all were mild in severity, with 10 resolved/recovered events not related to nonacog gamma, and one event (inhibitory antibody development) unconfirmed.
“The findings presented at WFH 2020 highlight our commitment to patients living with bleeding disorders, and are adding to the bank of scientific evidence supporting the importance of personalized care in Hematology,” Dr. Erdlenbruch concluded. “In addition to the AHEAD study, results from the physician survey in China supported the rationale for cross-department collaboration to optimize patient care. All the results from WFH 2020 emphasize the importance of understanding each patient and offering tailored support for those with bleeding disorders who may have differing needs.”
ABOUT ADVATE
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 groups.
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 SAFETY INFORMATION FOR EUROPEviii
Please consult the ADVATE Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring.
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, Peripheral 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 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.
About RIXUBIS
RIXUBIS is an injectable medicine used to replace clotting factor IX that is missing in adults and children with hemophilia B.
RIXUBIS SAFETY INFORMATION FOR EUROPEix
Please consult the RIXUBIS Summary of Product Characteristics (SmPC) here before prescribing, particularly in relation to dosing and treatment monitoring.
Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in the SmPC. Known allergic reaction to hamster protein.
Special warnings and precautions for use
Traceability
In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.
Hypersensitivity
Allergic type hypersensitivity reactions have been reported with RIXUBIS. The product contains traces of hamster proteins. If symptoms of hypersensitivity occur, patients or their caregivers 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 risk is highest during the early phases of initial exposure to factor IX concentrates in previously untreated patients (PUPs), in particular in patients with high-risk gene mutations. There have been reports in the literature showing an association between the occurrence of a factor IX inhibitor and allergic reactions, in particular in patients with a high-risk gene mutation. Therefore, patients experiencing allergic reactions should be evaluated for the presence of an inhibitor. In case of shock, standard medical treatment for shock should be implemented.
Inhibitors
After repeated treatment with human coagulation factor IX (rDNA) products, patients should be monitored for the development of neutralising antibodies (inhibitors) that should be quantified in Bethesda Units (BU) using appropriate biological testing. There have been reports in the literature showing a correlation between the occurrence of a factor IX inhibitor and allergic reactions. Therefore, patients experiencing allergic reactions should be evaluated for the presence of an inhibitor. It should be noted that patients with factor IX inhibitors may be at an increased risk of anaphylaxis with subsequent challenge with factor IX. Because of the risk of allergic reactions with factor IX concentrates, the initial administrations of factor IX should, according to the treating physician’s judgement, be performed under medical observation where proper medical care for allergic reactions could be provided.
Nephrotic syndrome
Nephrotic syndrome has been reported following attempted immune tolerance induction in haemophilia B patients with factor IX inhibitors.
Thromboembolism
Because of the potential risk of thrombotic complications, clinical surveillance for early signs of thrombotic and consumptive coagulopathy should be initiated with appropriate biological testing when administering this product to patients with liver disease, to patients post-operatively, to new-born infants, or to patients at risk of thrombotic phenomena or DIC. In each of these situations, the benefit of treatment with RIXUBIS should be weighed against the risk of these complications.
Cardiovascular events
In patients with existing cardiovascular risk factors, substitution therapy with FIX may increase the cardiovascular risk.
Catheter-related complications
If a central venous access device (CVAD) is required, risk of CVAD-related complications including local infections, bacteraemia and catheter site thrombosis should be considered.
Excipient related considerations
This medicine contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially ‘sodium free’. Depending on body weight and Posology of RIXUBIS, patients could receive more than one vial. This should be taken into consideration if the patient is on a controlled sodium diet.
Elderly
Clinical studies of RIXUBIS did not include subjects aged 65 and over. It is not known whether they respond differently from younger subjects. As for all patients, dose selection for an elderly patient should be individualised.
Paediatric population
The listed warnings and precautions apply both to adults and children.
Adverse Reactions
Adverse Drug Reactions, from clinical trials and spontaneous reports
MedDRA Standard System Organ Class
Adverse reactions
Frequency per Patient
Immune system disorders
Hypersensitivity
Not known
Nervous system disorders
Dysgeusia
Common
Musculoskeletal and connective tissue disorders
Pain in extremity
Common
For more information, please refer to the RIXUBIS Summary of Product Characteristics here.
For US specific safety information, please refer to the RIXUBIS US Prescribing Information here.
About Hemophilia
Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.x 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.i
People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.xi Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.xii
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 patientsxiii and a broad portfolio of 11 products across multiple bleeding disorders.xiv 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.
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.
Important Notice
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.
Forward-Looking Statements
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.
###
References
i 20th Anniversary Report on the Annual Global Survey 2018. World Federation of Hemophilia. Available at: http://www1.wfh.org/publications/files/pdf-1731.pdf. Last accessed May 2020.
ii Iorio, A., et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019 Oct 15;171(8):540-546.
iii Gringeri, A., Ewenstein, B. & Reininger, A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia. 2014;20:459-463.
iv Carcao, M. & Srivastava, A. Factor VIII/factor IX prophylaxis for severe hemophilia. Semin Hematol. 2016;53:3-9.
v WFH 2020 Virtual Summit. Abstract MED‐FP‐005 (363). Effectiveness, safety, and health‐related quality of life outcomes in patients with hemophilia A receiving antihemophilic factor (recombinant) in a real‐world setting: results of a 6‐year interim analysis of the AHEAD International study.
vi WFH 2020 Virtual Summit. iPoster MTD-PO-057. Multi-department collaboration for the management of hemophilia A: a physician survey in China.
vii WFH 2020 Virtual Summit. iPoster MED-PO-018. Safety and Effectiveness of Rixubis in Patients with Hemophilia B in South Korea: A Real-World, Prospective, Post-marketing Surveillance Study.
viii European Medicines Agency. Advate Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/advate-epar-product-information_en.pdf. Last accessed May 2020.
ix European Medicines Agency. Rixubis Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/rixubis-epar-product-information_en.pdf. Last accessed May 2020.
x World Federation of Hemophilia. “Introduction to hemophilia: what is hemophilia?” World Federation of Hemophilia website. Available at: https://elearning.wfh.org/elearning-centres/introduction-to-hemophilia/#what_is_hemophilia. Last accessed May 2020.
xi World Federation of Hemophilia. “Introduction to hemophilia: treatment.” Available here: https://elearning.wfh.org/elearning-centres/introduction-to-hemophilia/#hemophilia_treatment. Last accessed May 2020.
xii NHS. “Haemophilia: treatment.” Available here: https://www.nhs.uk/conditions/haemophilia/treatment/. Last accessed May 2020.
xiii Takeda Website. Rare Diseases. Available at: https://www.takeda.at/what-we-do/areas-of-focus/rare-diseases/. Last accessed May 2020.
xiv Takeda Website. U.S. Product List. Available at: https://www.takeda.com/en-us/what-we-do/product-portfolio/. Last accessed May 2020.
Contacts
Media Contacts:
Japanese Media
Kazumi Kobayashi
kazumi.kobayashi@takeda.com
+81 (0) 3-3278-2095
Media outside Japan
Linda Calandra
linda.calandra1@takeda.com
+1-617-301-2092
Permalink : https://www.aetoswire.com/news/takeda-provides-updates-on-its-hemophilia-studies-at-wfh-2020-reinforcing-its-commitment-to-putting-patients-first-and-supporting-personalized-care-for-people-with-bleeding-disorders/en
OSAKA, Japan-Monday 15 June 2020 [ AETOS Wire ]
(BUSINESS WIRE)-- Takeda Pharmaceutical Company Limited, (“Takeda”) (TSE: 4502/NYSE:TAK) today announced a scientific update from the AHEAD real-world study investigating the long-term outcomes associated with ADVATE [antihemophilic factor (recombinant), rAHF] in patients with hemophilia A, presented as an oral presentation at the World Federation of Hemophilia Virtual Summit 2020 (WFH 2020). The update is one of the three abstracts being presented at WFH 2020 from Takeda’s Hematology portfolio and pipeline. Echoing Takeda’s theme at the virtual summit – “It’s Personal” – the company also shared data from a physician survey in China, providing insights into optimizing personalized care and hemophilia patient management in centers of excellence (COE).
“It’s Personal”: Takeda is relentless in its pursuit of a bleed-free world
“Takeda’s vision for a bleed-free world is as important today as ever,” commented Dr. med. Wolfhard Erdlenbruch, M.D., Vice President Head of Global Medical Affairs Hematology, Takeda. “The data we presented at WFH 2020 continue to add to the body of evidence for the management of hemophilia, a bleeding disorder that affects at least 210,000 people worldwide as of 2018 and potentially a lot more.i,ii As a leader in Hematology, Takeda is committed to improving patient outcomes, and the findings of our studies demonstrate the value personalized strategies can offer to patients as part of the ongoing management of their bleeding disorder.”
Prophylaxis for hemophilia A is the standard of care treatment for patients because it can help prevent spontaneous bleeds, as even a single bleed may cause joint damage and impact their quality of life.iii,iv
Interim analysis results from the AHEAD real-world outcomes study [abstract MED‐FP‐005 (363)] demonstrate that the number of hemophilia A patients who were able to achieve zero bleeds increased over the years by receiving rAHF. For those receiving prophylaxis, the number of patients with zero bleeds increased from 34% in year 1 to 53% in year 6. For those receiving on-demand treatment, it increased from 28% in year 1 to 38% in year 6. The results also show that those receiving rAHF prophylaxis generally reported slightly better health-related quality of life (HRQoL) than those receiving rAHF on-demand. The results support the role of rAHF as an important option for hemophilia A, a condition which requires life-long monitoring and management.v
“The results of the interim analysis from the AHEAD real-world outcomes study support the long-term effectiveness of rAHF for hemophilia A patients, which was maintained in a six-year observational period and bolstered by consistent HRQoL scores,” added Prof. Dr. med. Johannes Oldenburg, Chairman and Director, Institute of Experimental Haematology and Transfusion Medicine, University Clinic Bonn AöR, Germany, who is the researcher leading the study. “Every patient is unique, and these results support a tailored and personalized approach to optimize care for patients with bleeding disorders, delivered in partnership with their treating physician.”
Takeda aims to help advance more personalized, connected care for patients
Takeda presented two additional abstracts at WFH 2020 – available on the summit’s iPoster Gallery (within the ‘Open to all’ and ‘HCP’ sections respectively) – supporting its personalized care approach for patients with hemophilia.
Multi-department collaboration for the management of hemophilia Avi [iPoster MTD-PO-057]
A substantial number of patients with hemophilia A live in China, due to the large population.i Results from a physician survey explored the Center of Excellence (COE) model for the optimization of multi-department collaboration for patients with hemophilia A in China; low patient awareness of the need for treatment/prophylaxis and fewer than 40% of patients received prophylaxis, demonstrated the need for improvements in both public and physician awareness and knowledge of hemophilia. Results showed that a COE model may improve collaboration across departments, optimize patient management and thereby improve outcomes.
Real-world safety and effectiveness of Rixubis in patients with hemophilia B in South Koreavii [iPoster MED-PO-018]
Data from a prospective, post-marketing surveillance study evaluating the safety and efficacy profile of Rixubis [Nonacog gamma, recombinant FIX concentrate] demonstrated the safety and efficacy profile for bleeds, perioperative/surgery, and prophylaxis in adult and pediatric patients with hemophilia B in the real-world setting in South Korea. The study showed that 86.6% (123/142) of hemostatic effectiveness assessments for nonacog gamma were reported as good or excellent, and of the 11 adverse events reported, all were mild in severity, with 10 resolved/recovered events not related to nonacog gamma, and one event (inhibitory antibody development) unconfirmed.
“The findings presented at WFH 2020 highlight our commitment to patients living with bleeding disorders, and are adding to the bank of scientific evidence supporting the importance of personalized care in Hematology,” Dr. Erdlenbruch concluded. “In addition to the AHEAD study, results from the physician survey in China supported the rationale for cross-department collaboration to optimize patient care. All the results from WFH 2020 emphasize the importance of understanding each patient and offering tailored support for those with bleeding disorders who may have differing needs.”
ABOUT ADVATE
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 groups.
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 SAFETY INFORMATION FOR EUROPEviii
Please consult the ADVATE Summary of Product Characteristics (SmPC) before prescribing, particularly in relation to dosing and treatment monitoring.
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, Peripheral 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 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.
About RIXUBIS
RIXUBIS is an injectable medicine used to replace clotting factor IX that is missing in adults and children with hemophilia B.
RIXUBIS SAFETY INFORMATION FOR EUROPEix
Please consult the RIXUBIS Summary of Product Characteristics (SmPC) here before prescribing, particularly in relation to dosing and treatment monitoring.
Contraindications
Hypersensitivity to the active substance or to any of the excipients listed in the SmPC. Known allergic reaction to hamster protein.
Special warnings and precautions for use
Traceability
In order to improve the traceability of biological medicinal products, the name and the batch number of the administered product should be clearly recorded.
Hypersensitivity
Allergic type hypersensitivity reactions have been reported with RIXUBIS. The product contains traces of hamster proteins. If symptoms of hypersensitivity occur, patients or their caregivers 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 risk is highest during the early phases of initial exposure to factor IX concentrates in previously untreated patients (PUPs), in particular in patients with high-risk gene mutations. There have been reports in the literature showing an association between the occurrence of a factor IX inhibitor and allergic reactions, in particular in patients with a high-risk gene mutation. Therefore, patients experiencing allergic reactions should be evaluated for the presence of an inhibitor. In case of shock, standard medical treatment for shock should be implemented.
Inhibitors
After repeated treatment with human coagulation factor IX (rDNA) products, patients should be monitored for the development of neutralising antibodies (inhibitors) that should be quantified in Bethesda Units (BU) using appropriate biological testing. There have been reports in the literature showing a correlation between the occurrence of a factor IX inhibitor and allergic reactions. Therefore, patients experiencing allergic reactions should be evaluated for the presence of an inhibitor. It should be noted that patients with factor IX inhibitors may be at an increased risk of anaphylaxis with subsequent challenge with factor IX. Because of the risk of allergic reactions with factor IX concentrates, the initial administrations of factor IX should, according to the treating physician’s judgement, be performed under medical observation where proper medical care for allergic reactions could be provided.
Nephrotic syndrome
Nephrotic syndrome has been reported following attempted immune tolerance induction in haemophilia B patients with factor IX inhibitors.
Thromboembolism
Because of the potential risk of thrombotic complications, clinical surveillance for early signs of thrombotic and consumptive coagulopathy should be initiated with appropriate biological testing when administering this product to patients with liver disease, to patients post-operatively, to new-born infants, or to patients at risk of thrombotic phenomena or DIC. In each of these situations, the benefit of treatment with RIXUBIS should be weighed against the risk of these complications.
Cardiovascular events
In patients with existing cardiovascular risk factors, substitution therapy with FIX may increase the cardiovascular risk.
Catheter-related complications
If a central venous access device (CVAD) is required, risk of CVAD-related complications including local infections, bacteraemia and catheter site thrombosis should be considered.
Excipient related considerations
This medicine contains less than 1 mmol sodium (23 mg) per vial, that is to say essentially ‘sodium free’. Depending on body weight and Posology of RIXUBIS, patients could receive more than one vial. This should be taken into consideration if the patient is on a controlled sodium diet.
Elderly
Clinical studies of RIXUBIS did not include subjects aged 65 and over. It is not known whether they respond differently from younger subjects. As for all patients, dose selection for an elderly patient should be individualised.
Paediatric population
The listed warnings and precautions apply both to adults and children.
Adverse Reactions
Adverse Drug Reactions, from clinical trials and spontaneous reports
MedDRA Standard System Organ Class
Adverse reactions
Frequency per Patient
Immune system disorders
Hypersensitivity
Not known
Nervous system disorders
Dysgeusia
Common
Musculoskeletal and connective tissue disorders
Pain in extremity
Common
For more information, please refer to the RIXUBIS Summary of Product Characteristics here.
For US specific safety information, please refer to the RIXUBIS US Prescribing Information here.
About Hemophilia
Hemophilia is a chronic disease that causes longer-than-normal bleeding due to absent or deficient clotting factor in the blood.x 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.i
People with hemophilia, working closely with their healthcare professionals, can live healthy lives with proper care and adequate treatment.xi Treatment regimens typically include on-demand and/or regular prophylactic infusions of factor replacement therapy to control or prevent the risk of bleeding.xii
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 patientsxiii and a broad portfolio of 11 products across multiple bleeding disorders.xiv 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.
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.
Important Notice
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.
Forward-Looking Statements
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.
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References
i 20th Anniversary Report on the Annual Global Survey 2018. World Federation of Hemophilia. Available at: http://www1.wfh.org/publications/files/pdf-1731.pdf. Last accessed May 2020.
ii Iorio, A., et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males: A Meta-analytic Approach Using National Registries. Ann Intern Med. 2019 Oct 15;171(8):540-546.
iii Gringeri, A., Ewenstein, B. & Reininger, A. The burden of bleeding in haemophilia: is one bleed too many? Haemophilia. 2014;20:459-463.
iv Carcao, M. & Srivastava, A. Factor VIII/factor IX prophylaxis for severe hemophilia. Semin Hematol. 2016;53:3-9.
v WFH 2020 Virtual Summit. Abstract MED‐FP‐005 (363). Effectiveness, safety, and health‐related quality of life outcomes in patients with hemophilia A receiving antihemophilic factor (recombinant) in a real‐world setting: results of a 6‐year interim analysis of the AHEAD International study.
vi WFH 2020 Virtual Summit. iPoster MTD-PO-057. Multi-department collaboration for the management of hemophilia A: a physician survey in China.
vii WFH 2020 Virtual Summit. iPoster MED-PO-018. Safety and Effectiveness of Rixubis in Patients with Hemophilia B in South Korea: A Real-World, Prospective, Post-marketing Surveillance Study.
viii European Medicines Agency. Advate Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/advate-epar-product-information_en.pdf. Last accessed May 2020.
ix European Medicines Agency. Rixubis Summary of Product Characteristics. Available at: https://www.ema.europa.eu/en/documents/product-information/rixubis-epar-product-information_en.pdf. Last accessed May 2020.
x World Federation of Hemophilia. “Introduction to hemophilia: what is hemophilia?” World Federation of Hemophilia website. Available at: https://elearning.wfh.org/elearning-centres/introduction-to-hemophilia/#what_is_hemophilia. Last accessed May 2020.
xi World Federation of Hemophilia. “Introduction to hemophilia: treatment.” Available here: https://elearning.wfh.org/elearning-centres/introduction-to-hemophilia/#hemophilia_treatment. Last accessed May 2020.
xii NHS. “Haemophilia: treatment.” Available here: https://www.nhs.uk/conditions/haemophilia/treatment/. Last accessed May 2020.
xiii Takeda Website. Rare Diseases. Available at: https://www.takeda.at/what-we-do/areas-of-focus/rare-diseases/. Last accessed May 2020.
xiv Takeda Website. U.S. Product List. Available at: https://www.takeda.com/en-us/what-we-do/product-portfolio/. Last accessed May 2020.
Contacts
Media Contacts:
Japanese Media
Kazumi Kobayashi
kazumi.kobayashi@takeda.com
+81 (0) 3-3278-2095
Media outside Japan
Linda Calandra
linda.calandra1@takeda.com
+1-617-301-2092
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