EC Approves CSL's Andembry for Hereditary Angioedema Prevention

The European Commission (EC) has granted approval for CSL's Andembry (garadacimab) as a preventive treatment for hereditary angioedema (HAE) attacks in individuals aged 12 and older. Hereditary angioedema is a rare genetic disorder, affecting approximately one in 50,000 people globally. This condition is characterized by unpredictable and recurring episodes of swelling, known as angioedema, which can be life-threatening. The swelling can target various body parts, including the face, abdomen, hands, feet, airways, and gastrointestinal tract, often causing significant pain.

Andembry is a monoclonal antibody, administered monthly, that focuses on plasma protein factor XIIa. This protein is instrumental in triggering swelling attacks in HAE patients. The medication is designed for subcutaneous injection using a pre-filled pen, allowing for self-administration once patients receive adequate training.

The EC's approval follows closely on the heels of the Medicines and Healthcare products Regulatory Agency's authorization of Andembry for the same demographic. The decision is underpinned by successful outcomes from the phase 3 VANGUARD trial and its ongoing open-label extension. Findings from the VANGUARD trial revealed that Andembry notably reduced HAE attack frequency, from an average of 2.01 attacks per month at the study's outset to just 0.27 after six months of therapy. Moreover, 62% of patients treated with Andembry experienced no attacks throughout the treatment duration. The study also demonstrated an impressive reduction in the median number of attacks to zero and an 86.5% decrease in the average number of monthly attacks compared to placebo.

Bill Mezzanotte, executive vice president and head of research and development at CSL, hailed Andembry as a crucial advancement in HAE management. He emphasized its potential to offer patients enduring control over their condition through a user-friendly, patient-focused administration approach. CSL has stated that its CSL Behring division will make Andembry accessible to eligible patients following the conclusion of access and reimbursement discussions.

Henrik Balle Boysen, president of the global non-profit organization HAE International, also expressed support for the approval. He highlighted Andembry as an innovative monthly subcutaneous treatment that inhibits activated factor XII. Boysen remarked that this development provides individuals with HAE an additional option to alleviate the challenges associated with this lifelong disorder, aligning with the community's collective aim of living life to its fullest potential.