Australian biotechnology company CSL has received the European Commission (EC) approval for Andembry (garadacimab) to prevent attacks of hereditary angioedema (HAE).
Andembry is a novel Factor XIIa-inhibitory monoclonal antibody (anti-FXIIa mAb), indicated to prevent HAE attacks in adult and adolescent patients aged 12 years and older.
The drug works by inhibiting plasma protein factor XIIa, which initiates the cascade of events leading to angioedema at various sites of the body.
CSL will provide Andembry in the form of a convenient patient-centric pre-filled pen (auto-injector), enabling subcutaneous self-injection.
The EC marketing authorisation is valid in all European Union (EU) member states, along with the European Economic Area (EEA) countries Iceland, Liechtenstein and Norway.
CSL R&D head and executive vice president Bill Mezzanotte said: “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.
“Thank you to all the colleagues, physicians and patients who contributed to this exciting milestone for HAE patients and CSL.”
According to CSL, HAE is a rare, chronic, debilitating, and potentially life-threatening genetic disorder characterised by recurrent and unpredictable attacks of angioedema.
HAE attacks are often painful and can spread to multiple sites of the body, including the abdomen, larynx, face, and extremities.
The EC approval of Andembry is based on the efficacy and safety data from the Phase 3 VANGUARD trial and its open-label extension study.
CSL Behring plans to make Andembry available for eligible people, subject to completion of access and reimbursement negotiations.
Andembry has been approved by the Australian Therapeutic Goods Administration (TGA), UK’s Medicines and Healthcare products Regulatory Agency (MHRA), last month.
The drug is currently under regulatory review in the US, Japan, Switzerland and Canada.
HAE International (HAEi) president Henrik Balle Boysen said: “Garadacimab, 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.”
