Posted: 5 February 2025
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.
