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CSL Receives Approval in Japan for ANDEMBRY® (garadacimab) Subcutaneous (S.C.) Injection 200mg Pens, a Novel Human Anti-Activated Factor XII Monoclonal Antibody for the Prevention of Acute Attacks of Hereditary Angioedema (HAE)
ANDEMBRY® is a first-in-class monoclonal antibody treatment that inhibits activated Factor XII (FXIIa), the initiating factor in the HAE pathway, and offers the first pre-filled pen presentation enabling once-monthly subcutaneous administration
The approval is based on the results of the international pivotal Phase 3 VANGUARD trial, which included HAE patients from Japan
CSL is dedicated to improving the lives of those with HAE – a community that we have proudly supported for more than 40 years
TOKYO, Feb. 20, 2025 /PRNewswire/ -- CSL Behring K.K. (Headquarters: Minato-ku, Tokyo; President and Representative Director: Izumi Yoshida) today announced that it has received manufacturing and marketing approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for ANDEMBRY® (garadacimab) Subcutaneous (S.C.) Injection 200mg Pens. The product is approved for the prevention of acute attacks of hereditary angioedema (HAE) and is the first pre-filled pen presentation for once-monthly subcutaneous administration for long-term prophylaxis of HAE. The approval in Japan follows additional recent approvals received in Australia, the United Kingdom, and the European Union.
CSL (PRNewsfoto/CSL)
ANDEMBRY is the first fully human monoclonal antibody in Japan designed to inhibit activated Factor XII (Factor XIIa), which initiates the cascade of events leading to angioedema at various sites of the body.
"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," said Bill Mezzanotte, MD, Executive Vice President, Head of R&D, CSL. "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 optimization. This milestone is the result of decades of dedication, and we extend our gratitude to the colleagues, physicians and patients who made this possible 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 are often painful and can occur in multiple sites of the body, including the abdomen, larynx, face, and extremities. HAE is designated as one of Japan's intractable diseases under the category of "Primary Immunodeficiency Syndrome." Reports indicate that approximately 430 patients in Japan are currently diagnosed and receiving treatment. According to global data, the prevalence of HAE is estimated to be 1 in 50,000 people, suggesting there may be approximately 2,500 patients in Japan.
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). A plain language summary of the VANGUARD trial findings has also been published to facilitate understanding of patients and caregivers of the clinical trial data. This summary is accessible in multiple languages, including English and Japanese.
"ANDEMBRY is a breakthrough therapy as the first and only treatment targeting activated Factor XII, the key initiator of HAE attacks," said Dr. Rose Fida, Executive Director and Regional Lead, CSL R&D Japan & China. "With its novel mechanism, once-monthly subcutaneous dosing and easy-to-use pre-filled pen, ANDEMBRY is set to transform the way HAE is managed in Japan."
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, and by the European Union's European Commission (EC) on February 10, 2025.
About "ANDEMBRY® S.C. Injection 200mg Pens"
Trade name
ANDEMBRY® S.C. Injection 200mg Pens
Indications or effects
Prevention of acute attacks of Hereditary Angioedema (HAE)
Dosage and administration
In general, administer subcutaneously the initial loading dose 400 mg of Garadacimab (Genetical Recombination), followed by 200 mg once a month for adults and pediatric patients aged 12 years and older.
Date of approval
February 20, 2025
Manufacturing and marketing
CSL Behring K.K.
About CSL Behring K.K. CSL Behring is a global leader in developing and delivering high-quality medicines that treat people with rare and serious diseases. In Japan, our core focus areas include immunology and rare diseases, hemophilia, as well as critical care and hemostasis. For more information, please visit https://www.cslbehring.co.jp.
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.
