Garadacimab

CSL has a portfolio that includes treatments for haemophilia and immune deficiencies, and influenza vaccines. Credit: © 2023 CSL, All Rights Reserved. CSL has received approval from the European Commission (EC) for Andembry (garadacimab) to prevent recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and above. The therapy targets factor XIIa and is designed to prevent the attacks with a once-monthly, self-administered dose via a pre-filled auto-injector. The approval is supported by the outcomes from the international Phase III VANGUARD trial and its subsequent open-label extension trial. These studies demonstrated the treatment’s safety and efficacy. Andembry’s centralised marketing authorisation means it will be accessible across all European Union member states and in the European Economic Area (EEA) countries Norway, Iceland and Liechtenstein. Beyond Europe, the therapy is under regulatory review in Japan, Canada, Switzerland and the US. CSL research and development head and executive vice-president Bill Mezzanotte stated: “Andembry is a significant advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method. “Andembry, CSL’s first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimisation and our decades-long journey to bring this innovation to patients.” As a factor XIIa-inhibitory monoclonal antibody, Andembry offers a unique approach to HAE treatment by blocking the plasma protein FXIIa, which is responsible for initiating the cascade of events resulting in angioedema. It was developed by the company scientists at the Bio21-based research site and manufactured at the CSL Broadmeadows Biotech facility. The therapy received approvals from the UK’s Medicines and Healthcare products Regulatory Agency and the Australian Therapeutic Goods Administration. CSL has a portfolio that includes treatments for haemophilia and immune deficiencies, influenza vaccines and therapies for iron deficiency and nephrology.

The European Commission (EC) has approved CSL’s Andembry (garadacimab) to prevent attacks of hereditary angioedema (HAE) in patients aged 12 years and older. Estimated to affect one in every 50,000 people worldwide, HAE is a rare and potentially life-threatening genetic disorder characterised by recurrent and unpredictable attacks of swelling (angioedema). These attacks are often painful and can spread to multiple parts of the body, including the abdomen, face, hands and feet, as well as the airways and gastrointestinal tract. CSL’s Andembry is a once-monthly monoclonal antibody designed to target plasma protein factor XIIa, which plays a crucial role in initiating attacks of swelling in HAE patients. The drug is delivered as a subcutaneous injection via a pre-filled pen and may be self-administered following appropriate training. The EC’s decision, which comes less than a month after the Medicines and Healthcare products Regulatory Agency approved Andembry for the same patient population, was based on positive results from the phase 3 VANGUARD trial and its ongoing open-label extension study. In VANGUARD, Andembry was shown to reduce HAE attacks rates from a mean of 2.01 attacks per month at baseline to a mean of 0.27 per month after six months of treatment. Additionally, 62% of Andembry-treated patients achieved attack-free status throughout the treatment period, and the drug was shown to reduce the median number of HAE attacks to zero and lower the mean number of HAE attacks per month by 86.5% compared to placebo. Bill Mezzanotte, executive vice president, head of research and development at CSL, said: “Andembry is a significant advancement in the management of HAE, offering people living with this life-threatening condition long-term control over their disease with a patient-centric, convenient administration method.” CSL outlined that its CSL Behring business unit will make Andembry available for eligible patients “upon completion of access and reimbursement negotiations”. Also commenting on the approval, Henrik Balle Boysen, president of global non-profit network HAE International, said: “[Andembry], a novel once monthly subcutaneous treatment that inhibits activated factor XII, is a welcome addition to the HAE treatment landscape. “People with HAE now have another choice for lessening the burden associated with this lifelong condition and realising the community’s shared goal of experiencing life to the fullest.”