Japan Approves CSL Behring's Andembry for HAE Attack Prevention

CSL Behring KK has received the green light from Japan's Ministry of Health, Labour and Welfare (MHLW) to produce and market Andembry pens, a 200mg subcutaneous injection designed to prevent acute episodes of hereditary angioedema (HAE). This disease, marked by sudden and severe swelling in various body parts, can have life-threatening implications, especially if it affects the respiratory tract.

Andembry pens represent a significant advancement, being the first of their kind to offer a pre-filled pen presentation for once-monthly subcutaneous administration. This innovation aims to provide a convenient and effective long-term prophylactic treatment for individuals dealing with this condition. The approval from Japan follows similar endorsements in other regions, including the European Union, Australia, and the United Kingdom.

The decision by the MHLW is supported by data from the international Phase III VANGUARD trial, along with findings from its open-label extension. These studies provide robust evidence of the safety and efficacy of Andembry.

Dr. Rose Fida, who leads research and development for Japan and China at CSL, highlighted the uniqueness of Andembry. She emphasized that it is the first and only treatment targeting activated Factor XII, a crucial component in the chain reaction initiating HAE attacks. This novel mechanism, coupled with the product's user-friendly design and monthly dosing, is expected to revolutionize HAE management in Japan.

Andembry distinguishes itself as the first fully human monoclonal antibody in Japan that targets activated Factor XII (Factor XIIa). By inhibiting FXIIa, Andembry disrupts the process that leads to the formation of oedema, effectively halting the progression of HAE attacks at an early stage. The therapy's journey through Phase III clinical development underscores its potential as a preventative treatment for HAE-related episodes.

The creation of Andembry involved the expertise of CSL's Bio21-based research team, with the formulation and production carried out at the CSL Broadmeadows biotechnology manufacturing facility. This collaborative effort highlights CSL's commitment to advancing treatment options for rare and challenging conditions.

Hereditary angioedema is classified in Japan as part of the "primary immunodeficiency syndrome" list, recognized as an intractable condition. The disease is characterized by recurrent episodes of swelling in areas such as the larynx, face, extremities, and abdomen, posing significant health risks if not managed effectively.

CSL Behring KK operates as a division of the Australian biotechnology company, CSL, which is known for its innovative approaches in the medical field. The recent advancements in Japan also include the approval of a self-amplifying mRNA (sa-mRNA) Covid-19 vaccine, ARCT-154, developed in collaboration with Arcturus Therapeutics. This approval, granted in November 2023, marks another milestone in CSL’s ongoing efforts to address urgent health needs through pioneering biotechnological solutions.

Through these developments, CSL continues to demonstrate its dedication to enhancing patient care and advancing medical science. The introduction of Andembry in Japan is poised to offer a transformative solution for individuals affected by hereditary angioedema, potentially improving their quality of life and offering new hope in the management of this challenging condition.