European Commission Grants Approval for CSL's ANDEMBRY® as a New Treatment for Hereditary Angioedema

CSL's Revolutionary Step in HAE Treatment: ANDEMBRY®

On February 13, 2025, CSL Behring, a leading name in biotechnology, proudly announced that the
European Commission has sanctioned ANDEMBRY®, a groundbreaking treatment aimed at preventing attacks of hereditary angioedema (HAE). This remarkable approval marks a new chapter in the therapeutic landscape for HAE, distinguishing ANDEMBRY as the first and only once-monthly treatment specifically targeting factor XIIa to prevent HAE attacks in both adult and adolescent patients aged 12 and older.

The drug is designed to inhibit plasma protein factor XIIa, the initial catalyst in the inflammatory cascade that triggers these potentially severe and unpredictable attacks associated with HAE. The approval is rooted in the promising findings from the Phase 3 VANGUARD trial, which has validated both the safety and efficacy of ANDEMBRY, reinforcing CSL's ongoing commitment to pioneering treatments for this rare disorder.

A Patient-Centric Approach

ANDEMBRY not only embodies innovative science but also emphasizes patient convenience. It is delivered through a user-friendly pre-filled auto-injector, which allows for subcutaneous self-injection, thus enhancing adherence to treatment schedules. Bill Mezzanotte, Executive Vice President and Head of Research and Development at CSL, remarked on the significance of ANDEMBRY, stating, "This treatment offers long-term control over hereditary angioedema, empowering patients with a convenient administration method that can fit seamlessly into their lives."

Insights on Hereditary Angioedema

Hereditary angioedema is a rare genetic condition affecting approximately 1 in 50,000 people and is characterized by recurrent, debilitating attacks of swelling that can impact various body regions, including the abdomen, larynx, and face. The unpredictability of these episodes poses significant challenges for patients, both physically and psychologically. Experts suggest that the actual cases of HAE may be underreported due to misdiagnosis, indicating a critical need for effective treatments like ANDEMBRY.

Prof. Dr. med Markus Magerl, a leading authority on the condition from Charité University Hospital Berlin, explained that current HAE therapeutic options address downstream effects of the inflammatory cascade but do not prevent its initiation. This is where ANDEMBRY stands out, targeting the root cause instead of merely managing the consequences.

Supporting Evidence from Clinical Trials

The pivotal VANGUARD trial, published in renowned medical journals, provided robust data supporting the approval of ANDEMBRY. It demonstrates that patients treating with ANDEMBRY experience a reduced frequency of HAE attacks, thus translating to a better quality of life. HAE International's President, Henrik Balle Boysen, expressed optimism for the community, stating, "ANDEMBRY is a valuable addition to the HAE treatment protocol, allowing individuals to feel more in control of their health and to pursue their lives without the constant fear of unexpected angioedema episodes."

With this approval, CSL Behring is committed to making ANDEMBRY accessible to patients across the EU and is currently negotiating reimbursement and access pathways to ensure patients can benefit from this innovation. The marketing authorization grants full access across all EU member states and the EEA countries, including Norway, Iceland, and Liechtenstein.

Additionally, ANDEMBRY has received previous approvals by the Australian Therapeutic Goods Administration (TGA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA) earlier in January. CSL is keen to expand this critical treatment to other global markets, with submissions currently ongoing in the United States, Japan, Switzerland, and Canada.

The Way Forward

As CSL Behring navigates these regulatory landscapes, the focus remains on ensuring that the distribution of ANDEMBRY proceeds smoothly, providing patients with a vital new option for managing their hereditary angioedema. The introduction of this innovative therapy is a testament to CSL’s dedication to research and development in rare diseases, promising a future where living with HAE may be less burdensome for those affected.

For ongoing updates and future developments regarding ANDEMBRY and other CSL products, visit CSLBehring.com or follow them on social media for the latest news and breakthroughs in biotechnology.