The European Commission (EC) has approved CSL’s 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 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.”
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.
