The TGA has approved CSL's locally discovered and developed monoclonal antibody of the rare genetic disorder hereditary angioedema (HAE) attacks.
HAE, a form of bradykinin-mediated angioedema, is a rare, debilitating, and potentially life-threatening genetic disorder affecting approximately 1 in 50,000 people globally. It is caused by the excessive production of bradykinin due to a deficiency in C1 esterase inhibitor (C1-INH), leading to swelling in various parts of the body, including the face, limbs, and potentially the airways.
ANDEMBRY (garadacimab) is a once-monthly novel Factor XIIa-inhibitory monoclonal antibody administered subcutaneously using a pre-filled pen.
The TGA has approved ANDEMBRY to prevent HAE attacks in patients aged 12 years and older with C1-esterase inhibitor deficiency or dysfunction.
The Pharmaceutical Benefits Advisory Committee recommended ANDEMBRY to be listed on the PBS at its November 2024 meeting. The company said it looks forward to working through the reimbursement process with decision-makers.
ANDEMBRY was discovered and developed by scientists at CSL’s Melbourne-based research and development laboratories. It was then manufactured for clinical programs at its facility in Broadmeadows.
“ANDEMBRY is a testament to CSL’s commitment to pursue innovations that disrupt the standard of care in areas we know well and provide novel mechanisms to address unmet needs,” said Dr Bill Mezzanotte, executive vice president and 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 which we have proudly supported for over 40 years. Thank you to everyone who contributed to this achievement.”
“I realised we had something special when our lead scientist on this programme came into my office and showed me some data related to the screening they'd been doing. There was one particular candidate that had just the activity that we were looking for, and that candidate ultimately turned out to be the molecule that we optimised, brought through clinical trials and is now registered in Australia,” said Dr Andrew Nash, CSL’s chief scientific officer.
“Working on something right from the beginning in 2009 in the discovery phase, and now seeing it registered is something that all the scientists aspire to and it's been a real privilege,” said Dr Veronika Rayzman, senior scientist at CSL.
“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, CSL CEO and managing director. “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.”
TGA approval of ANDEMBRY was granted based on data from the pivotal placebo-controlled Phase 3 VANGUARD trial and its open-label extension study.
CSL said ANDEMBRY is also under review by regulatory agencies in the European Union, United States, Japan, Switzerland, Canada and the United Kingdom.