- Discovered and developed in Australia by CSL scientists, ANDEMBRY is CSL’s first monoclonal antibody treatment; reinforces company’s commitment to providing a wide range of treatment modalities to address patients’ needs
- This regulatory approval for registration in Australia is the first in the world for ANDEMBRY
MELBOURNE, Australia I January 24, 2025 I Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the registration of ANDEMBRY® (garadacimab) for routine prevention of recurrent hereditary angioedema (HAE) attacks in patients aged 12 years and older with C1-esterase inhibitor deficiency or dysfunction.1
ANDEMBRY® is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) and is a once-monthly treatment approved for registration in Australia to treat HAE, a form of bradykinin-mediated angioedema. HAE is a rare, debilitating, and potentially life-threatening genetic disorder affecting approximately 1 in 50,000 people globally.2
ANDEMBRY® inhibits the plasma protein factor XIIa, which when activated, initiates the cascade of events leading to oedema at various sites of the body. The treatment is administered subcutaneously using a pre-filled pen (auto-injector), which allows for self-administration.1
It was discovered and developed by scientists at CSL’s Melbourne-based Research and Development laboratories, with manufacturing for clinical programs completed at CSL’s Broadmeadows Manufacturing Facility.
“Today’s news represents a significant milestone for people living with hereditary angioedema and exemplifies the CSL patient-centric ambition of improving quality of life through novel treatment options and convenient administration,” said Paul McKenzie, Chief Executive Officer and Managing Director, CSL. “As an Australian company with a global footprint, we take great pride that ANDEMBRY® is our first monoclonal antibody treatment and was discovered in our labs in Australia. We look forward to making this medicine accessible to patients in the future, to address unmet needs in the HAE community.”
“HAE is a condition characterised by unpredictable swelling attacks that can cause significant disability, loss of work and school time and can be life threatening if airway swelling occurs,” said Constance Katelaris, Professor of Immunology and Allergy at Campbelltown Hospital. “It is estimated that over 500 Australians are living with HAE, which impacts their quality of life, leading to increased rates of anxiety and depression in these patients. The regulatory approval of a new treatment option is an important step towards improving health outcomes of Australians living with this condition.”
The TGA approval for registration was granted following evaluation of the efficacy and safety data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study.1
“ANDEMBRY® is a testament to CSL’s commitment to pursuing innovations that disrupt the standard of care in areas we know well and providing novel mechanisms to address unmet needs,” said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. “Our homegrown ANDEMBRY® highlights our scientific and technical capabilities and our promise to improving the lives of those with HAE – a community that we have proudly supported for over 40 years. Thank you to everyone who contributed to this achievement.”
ANDEMBRY® has been recommended by the Pharmaceutical Benefits Advisory Committee for funding on Australia’s Pharmaceutical Benefits Scheme.3 CSL looks forward to continuing to work through the reimbursement process with decision-makers to make the treatment accessible to patients.
ANDEMBRY® is also under review by regulatory agencies in the European Union, United States, Japan, Switzerland, Canada and the United Kingdom.
About HAE
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death.
About ANDEMBRY®
ANDEMBRY® is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) and is a once-monthly treatment approved in Australia to treat HAE, a form of bradykinin-mediated angioedema.1 ANDEMBRY® inhibits the plasma protein factor XIIa, which when activated, initiates the cascade of events leading to oedema (swelling) at various sites of the body.1 ANDEMBRY® is CSL Behring’s first product evaluated and approved for registration through Australia’s work-sharing ACCESS pathway.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit www.CSL.com.
References
- Andembry Australian Consumer Medicine Information. 2025. Available at: https://www.tga.gov.au/resources/artg/443611. Accessed January 2025
- Ghazi A et al., Biologics. 2013;7:103-13. doi: 10.2147/BTT.S27566
- Australian Department of Health and Aged Care. 2024. November PBAC Outcomes. Available at: https://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/pbac-outcomes/recommendations-made-by-the-pbac-november-2024. Accessed January 2025
SOURCE: CSL
Post Views: 113
- Discovered and developed in Australia by CSL scientists, ANDEMBRY is CSL’s first monoclonal antibody treatment; reinforces company’s commitment to providing a wide range of treatment modalities to address patients’ needs
- This regulatory approval for registration in Australia is the first in the world for ANDEMBRY
MELBOURNE, Australia I January 24, 2025 I Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the registration of ANDEMBRY® (garadacimab) for routine prevention of recurrent hereditary angioedema (HAE) attacks in patients aged 12 years and older with C1-esterase inhibitor deficiency or dysfunction.1
ANDEMBRY® is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) and is a once-monthly treatment approved for registration in Australia to treat HAE, a form of bradykinin-mediated angioedema. HAE is a rare, debilitating, and potentially life-threatening genetic disorder affecting approximately 1 in 50,000 people globally.2
ANDEMBRY® inhibits the plasma protein factor XIIa, which when activated, initiates the cascade of events leading to oedema at various sites of the body. The treatment is administered subcutaneously using a pre-filled pen (auto-injector), which allows for self-administration.1
It was discovered and developed by scientists at CSL’s Melbourne-based Research and Development laboratories, with manufacturing for clinical programs completed at CSL’s Broadmeadows Manufacturing Facility.
“Today’s news represents a significant milestone for people living with hereditary angioedema and exemplifies the CSL patient-centric ambition of improving quality of life through novel treatment options and convenient administration,” said Paul McKenzie, Chief Executive Officer and Managing Director, CSL. “As an Australian company with a global footprint, we take great pride that ANDEMBRY® is our first monoclonal antibody treatment and was discovered in our labs in Australia. We look forward to making this medicine accessible to patients in the future, to address unmet needs in the HAE community.”
“HAE is a condition characterised by unpredictable swelling attacks that can cause significant disability, loss of work and school time and can be life threatening if airway swelling occurs,” said Constance Katelaris, Professor of Immunology and Allergy at Campbelltown Hospital. “It is estimated that over 500 Australians are living with HAE, which impacts their quality of life, leading to increased rates of anxiety and depression in these patients. The regulatory approval of a new treatment option is an important step towards improving health outcomes of Australians living with this condition.”
The TGA approval for registration was granted following evaluation of the efficacy and safety data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study.1
“ANDEMBRY® is a testament to CSL’s commitment to pursuing innovations that disrupt the standard of care in areas we know well and providing novel mechanisms to address unmet needs,” said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. “Our homegrown ANDEMBRY® highlights our scientific and technical capabilities and our promise to improving the lives of those with HAE – a community that we have proudly supported for over 40 years. Thank you to everyone who contributed to this achievement.”
ANDEMBRY® has been recommended by the Pharmaceutical Benefits Advisory Committee for funding on Australia’s Pharmaceutical Benefits Scheme.3 CSL looks forward to continuing to work through the reimbursement process with decision-makers to make the treatment accessible to patients.
ANDEMBRY® is also under review by regulatory agencies in the European Union, United States, Japan, Switzerland, Canada and the United Kingdom.
About HAE
HAE is a rare and potentially life-threatening genetic condition that occurs in about 1 in 10,000 to 1 in 50,000 people. HAE is caused by deficient or dysfunctional C1-INH, a protein in the blood that helps to control inflammation. Inadequate amounts of properly functioning C1-INH can lead to the accumulation of fluid in body tissues, causing considerable swelling referred to as angioedema. HAE attacks can affect many parts of the body and can spread to multiple sites, including the face, abdomen, larynx, and extremities. Patients who have abdominal attacks of HAE can experience extreme pain, diarrhea, nausea, and vomiting caused by swelling of the intestinal wall. HAE attacks that involve the face or throat can result in airway closure, asphyxiation and, if left untreated, death.
About ANDEMBRY®
ANDEMBRY® is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) and is a once-monthly treatment approved in Australia to treat HAE, a form of bradykinin-mediated angioedema.1 ANDEMBRY® inhibits the plasma protein factor XIIa, which when activated, initiates the cascade of events leading to oedema (swelling) at various sites of the body.1 ANDEMBRY® is CSL Behring’s first product evaluated and approved for registration through Australia’s work-sharing ACCESS pathway.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a global biotechnology company with a dynamic portfolio of lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest. For inspiring stories about the promise of biotechnology, visit CSLBehring.com/Vita and follow us on Twitter.com/CSL. For more information about CSL, visit www.CSL.com.
References
- Andembry Australian Consumer Medicine Information. 2025. Available at: https://www.tga.gov.au/resources/artg/443611. Accessed January 2025
- Ghazi A et al., Biologics. 2013;7:103-13. doi: 10.2147/BTT.S27566
- Australian Department of Health and Aged Care. 2024. November PBAC Outcomes. Available at: https://www.pbs.gov.au/info/industry/listing/elements/pbac-meetings/pbac-outcomes/recommendations-made-by-the-pbac-november-2024. Accessed January 2025
SOURCE: CSL
Post Views: 113
