Garadacimab

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.”

– ANDEMBRY ® , the first and only once-monthly treatment targeting factor XIIa to prevent attacks in HAE patients, inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body – ANDEMBRY reinforces CSL’s decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection – Supported by the Phase 3 VANGUARD trial, this marks the third regulatory approval for ANDEMBRY, which was also recently approved by Australia’s Therapeutic Goods Administration (TGA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) in January MARBURG, Germany I February 13, 2025 I Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced that the European Commission (EC) has approved ANDEMBRY ® (garadacimab), the first and only once-monthly treatment targeting factor XIIa to prevent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older. ANDEMBRY inhibits plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body. ANDEMBRY reinforces CSL’s decades-long commitment to delivering innovative treatment modalities to the HAE community and comes with a convenient patient-centric pre-filled pen (auto-injector) enabling subcutaneous self-injection. “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,” said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. “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 optimization and our decades-long journey to bring this innovation to patients. Thank you to all the colleagues, physicians and patients who contributed to this exciting milestone for HAE patients and CSL.” HAE is a rare, chronic, debilitating, and potentially life-threatening genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks of HAE are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE occurs in about 1 in 50,000 people of any ethnic group. “The physical and emotional toll of HAE is substantial, and the true prevalence of the disease could be higher than currently recorded due to patient misdiagnoses,” said Prof. Dr. med Markus Magerl, MD, Head of Clinical Trials, Charité University Hospital Berlin, Institute of Allergology. “The unpredictable nature of HAE is one of the most daunting aspects for many patients, who never know when an attack might occur. Current HAE preventive therapies work at various downstream steps in the cascade, but none prevent the cascade at its very start like ANDEMBRY.” The approval of ANDEMBRY is based on the efficacy and safety data from the pivotal international Phase 3 VANGUARD trial and its open-label extension study. The detailed results of the VANGUARD trial were published in The Lancet in April 2023 and the primary results of the ongoing open-label extension study were published in Allergy (October 2024). “Garadacimab, a novel once monthly subcutaneous treatment that inhibits activated Factor XII, is a welcome addition to the HAE treatment landscape,” said Henrik Balle Boysen, President, HAE International (HAEi). “People with HAE now have another choice for lessening the burden associated with this lifelong condition and realizing the community’s shared goal of experiencing life to the fullest.” CSL Behring will make ANDEMBRY available for eligible people upon completion of access and reimbursement negotiations. The centralized marketing authorization of ANDEMBRY is valid in all European Union (EU) member states as well as in the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway. ANDEMBRY is currently under review by regulatory agencies in the United States, Japan, Switzerland and Canada. About ANDEMBRY ® (garadacimab) ANDEMBRY (garadacimab) is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb) that has completed Phase 3 clinical development as a new type of once-monthly subcutaneous prophylactic treatment for attacks related to HAE, a form of bradykinin-mediated angioedema. ANDEMBRY is CSL’s first homegrown recombinant monoclonal antibody to gain approval. It was discovered and optimized by scientists at CSL’s Bio21-based research site, with formulation and manufacturing for the clinical programs completed at the CSL Broadmeadows Biotech Manufacturing Facility. ANDEMBRY uniquely inhibits the plasma protein, FXIIa. When FXII is activated, it initiates the cascade of events leading to edema formation. By targeting FXIIa, ANDEMBRY inhibits this cascade at the top as compared to other HAE therapies that target downstream mediators. As of February 2025, ANDEMBRY has been approved by the Australian Therapeutic Goods Administration (TGA) on January 14, 2025, the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) on January 24, 2025. Warnings: Do not use ANDEMBRY ® if you are allergic to garadacimab, or any of the ingredients listed at the end of this leaflet. Always check the ingredients to make sure you can use this medicine. Precautions: Patients should inform their doctor if they have any other medical conditions or take any medicines for any other condition. Patients should inform their doctor if they are pregnant or intend to become pregnant or are breastfeeding or intend to breastfeed. Interactions: Patients should inform their doctor if they are taking any other medicines, including any medicines, vitamins or supplements that you buy without a prescription from your pharmacy, supermarket or health food shop. Side effects: The most commonly observed side effects associated with ANDEMBRY ® in the phase 3 clinical trial were injection site reactions (2/39, 5.1%) including redness, bruising and itchiness. 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 . SOURCE: CSL Behring