CSL Behring's ANDEMBRY® Receives Approval for HAE Treatment in Japan

CSL Behring's Landmark Approval for HAE Treatment in Japan

On February 20, 2025, CSL Behring K.K., based in Minato-ku, Tokyo, achieved a significant milestone with the approval of ANDEMBRY® (garadacimab) for the prevention of acute attacks of hereditary angioedema (HAE). This innovative subcutaneous injection, available in a convenient pre-filled pen format, is designed for once-monthly self-administration, providing patients with greater ease and flexibility in managing their condition.

ANDEMBRY® is a groundbreaking monoclonal antibody that specifically inhibits activated Factor XII (FXIIa), a key player in the HAE attack cascade. By targeting this initiating factor, ANDEMBRY offers a distinctive approach compared to existing therapies, many of which act on downstream mechanisms. The approval comes on the heels of successful international clinical trials, specifically the Phase 3 VANGUARD trial, which involved participants from Japan and demonstrated the safety and efficacy of ANDEMBRY in a diverse patient population.

The landscape of HAE treatment has evolved significantly over the past decades, and CSL Behring has been at the forefront of this evolution. With over 40 years of experience in the field, the company has consistently prioritized the needs of HAE patients. Bill Mezzanotte, MD, CSL's Executive Vice President and Head of Research and Development, emphasized the importance of ANDEMBRY, stating it represents a major advancement in the management of this challenging condition. He noted that this treatment not only aims to improve health outcomes but also enhances the overall quality of life for individuals living with HAE.

Hereditary angioedema is a rare genetic disorder characterized by recurrent episodes of swelling, which can be painful and unpredictable. Attacks can involve various parts of the body, including the abdomen, face, larynx, and limbs, and can lead to serious complications, including airway obstruction. Reports indicate that approximately 430 patients in Japan are diagnosed with HAE, with global estimates suggesting about 2,500 cases in the country, underscoring the need for effective treatment options.

The approval of ANDEMBRY relies on robust data collected from the VANGUARD trial and its extension studies, with findings published in reputable medical journals. To ensure broader understanding and accessibility, CSL Behring has made plain language summaries of these trial results available to patients and caregivers in both English and Japanese.

Dr. Rose Fida, Executive Director and Regional Lead for CSL in Japan and China, expressed optimism about the transformative potential of ANDEMBRY for the Japanese HAE community. With its novel mechanism of action and user-friendly delivery system, ANDEMBRY is poised to redefine how patients manage their condition.

As Japan's first fully human anti-FXIIa monoclonal antibody, ANDEMBRY not only represents a technological advancement but also a commitment from CSL to prioritize patient care and comfort in chronic disease management. The product build on considerable research efforts made at CSL’s Bio21 facility, reinforcing the company's dedication to innovative biopharmaceutical solutions.

Alongside its Japanese approval, ANDEMBRY has also recently secured clearance from health authorities in Australia, the United Kingdom, and the European Union, showcasing CSL Behring's global presence and commitment to treating rare diseases.

CSL's mission focuses on improving lives by providing innovative medicines, and the introduction of ANDEMBRY into Japan is a testament to this commitment. With a new treatment option now available, patients with hereditary angioedema can look forward to a more manageable and less disruptive future.