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)

Rhea-AI Summary

CSL Behring has received approval from Japan's MHLW for ANDEMBRY® (garadacimab), a first-in-class monoclonal antibody treatment for preventing hereditary angioedema (HAE) attacks. The treatment, administered via pre-filled pen for once-monthly subcutaneous injection, works by inhibiting activated Factor XII, the initiating factor in the HAE pathway.

The approval follows successful results from the international Phase 3 VANGUARD trial, with the detailed results published in The Lancet (April 2023) and Allergy (October 2024). ANDEMBRY has already received approvals in Australia, the United Kingdom, and the European Union.

HAE is a rare genetic disorder affecting approximately 430 diagnosed patients in Japan, with an estimated total patient population of 2,500 based on global prevalence rates of 1 in 50,000 people. The condition causes recurrent and unpredictable attacks of angioedema in multiple body sites.

Positive

• First-in-class monoclonal antibody treatment approved for HAE prevention
• Novel once-monthly subcutaneous administration via pre-filled pen
• Multiple regulatory approvals across major markets (Japan, EU, UK, Australia)
• Addresses an underserved market with 2,500 potential patients in Japan

Negative

• None.

• 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.

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.

Media Contact
Valerie Bomberger, CSL
Office: +1 610-291-5388 
Mobile: +1 267-280-3829 
Email: valerie.bomberger@cslbehring.com 

In Australia: 
Brett Foley, CSL
Mobile: +61 461 464 708
Email: brett.foley@csl.com.au

Investor Relations:
Chris Cooper, CSL
Mobile: +61 455 022 740
Email: chris.cooper@csl.com.au

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SOURCE CSL

FAQ

What is the significance of ANDEMBRY's approval for CSLLY in Japan?

ANDEMBRY is the first-in-class monoclonal antibody treatment for HAE prevention in Japan, offering once-monthly subcutaneous administration via pre-filled pen, representing a significant market opportunity with 2,500 potential patients.

How many patients could benefit from ANDEMBRY (CSLLY) in Japan?

Currently, 430 patients are diagnosed and receiving treatment in Japan, with an estimated total potential patient population of 2,500 based on global prevalence rates of 1 in 50,000 people.

What clinical trials supported ANDEMBRY's approval for CSLLY?

The approval was based on the Phase 3 VANGUARD trial, with results published in The Lancet (April 2023) and its open-label extension study published in Allergy (October 2024).

Which other markets have approved ANDEMBRY (CSLLY) besides Japan?

ANDEMBRY has received regulatory approvals in Australia, the United Kingdom, and the European Union prior to its Japanese approval.

How does ANDEMBRY (CSLLY) work in treating HAE?

ANDEMBRY works by inhibiting activated Factor XII (FXIIa), which is the initiating factor in the HAE pathway, preventing the cascade of events leading to angioedema attacks.