Australia’s largest biotech secures first TGA listing for HAE rare disease

The Therapeutic Goods Administration (TGA) has granted approval for the registration of the first treatment targeting factor XIIa, offering new hope for patients with hereditary angioedema (HAE).

HAE, a rare and potentially life-threatening genetic disorder, affects around 1 in 50,000 people worldwide. CSL’s Andembry (garadacimab) is indicated for the routine prevention of recurrent HAE attacks in patients aged 12 years and older who have C1-esterase inhibitor deficiency or dysfunction.

Paul McKenzie, CEO and Managing Director of CSL, Australia’s largest biotechnology company, said “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.”

Constance Katelaris, Professor of Immunology and Allergy at Campbelltown Hospital, emphasised the impact of HAE on patients’ daily lives.

“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,” she said. “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’s approval follows the evaluation of data from the pivotal Phase 3 VANGUARD trial, a placebo-controlled study, and its open-label extension.

Dr Bill Mezzanotte, Executive Vice President and Head of R&D at CSL, highlighted the significance of Andembry’s approval, saying “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.”

He added, “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.”

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