CSL Behring has secured regulatory approval in Japan for Andembry (garadacimab) Subcutaneous (S.C.) Injection 200mg Pens for the prevention of acute hereditary angioedema (HAE) attacks.
The authorisation, granted by Japan’s Ministry of Health, Labour and Welfare (MHLW), introduces the first pre-filled pen designed for once-monthly subcutaneous administration as a long-term prophylactic treatment for HAE.
The treatment is the first fully human monoclonal antibody in Japan designed to inhibit activated Factor XII (Factor XIIa), a key initiator of the biochemical cascade responsible for angioedema episodes in HAE patients.
CSL executive vice president and research and development head Bill Mezzanotte said: “Andembry represents a major advancement in the management of hereditary angioedema, offering people living with this life-threatening condition long-term disease control through a patient-centric and convenient administration method.
“As CSL’s first approved recombinant monoclonal antibody discovered and developed entirely by CSL, Andembry underscores our more than 40-year commitment to HAE research and treatment optimisation.”
HAE is a rare genetic disorder characterised by recurrent and unpredictable swelling episodes that can affect the face, extremities, abdomen, and larynx.
The condition is classified under Japan’s “Primary Immunodeficiency Syndrome” category of designated intractable diseases. Regulatory approval for Andembry in Japan was supported by clinical data from the global Phase 3 VANGUARD trial and its open-label extension study, which evaluated efficacy and safety in patients requiring prophylactic treatment.
CSL Behring developed Andembry as its first proprietary recombinant monoclonal antibody to receive marketing approval. The therapy was discovered and optimised at the company’s Bio21-based research facility, with formulation and clinical manufacturing conducted at the CSL Broadmeadows Biotech Manufacturing Facility.
Andembry’s mechanism of action distinguishes it from existing HAE treatments by targeting Factor XIIa at the start of the cascade, rather than intervening in downstream pathways.
The product has received multiple regulatory approvals in early 2025. Australia’s Therapeutic Goods Administration (TGA) authorised Andembry on 14 January 2025, followed by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on 24 January 2025.
Earlier this month, the European Commission (EC) granted marketing authorisation to Andembry for the prevention of HAE attacks.
