FDA Approves CSL's Andembry for Hereditary Angioedema Prevention

The US Food and Drug Administration (FDA) has granted approval for CSL's Andembry, a treatment aimed at preventing attacks of hereditary angioedema (HAE). HAE is a rare genetic disorder that can be life-threatening, affecting roughly one in every 10,000 to 50,000 individuals globally. This condition is known for its unpredictable episodes of swelling, which are often painful and can impact various parts of the body, such as the abdomen, face, hands, feet, airways, and gastrointestinal tract.

Andembry, developed by CSL, has been approved for use in both adults and children aged 12 and above. It is a monoclonal antibody specifically designed to target factor XIIa, a plasma protein instrumental in triggering swelling attacks in those with HAE. The drug is unique in its administration method, as it is self-administered through a subcutaneous injection using an auto-injector. Remarkably, it is the first treatment to provide once-monthly dosing from the onset for all patients, according to CSL.

The FDA's approval was based on encouraging results from the phase 3 VANGUARD trial. In this study, 62% of participants receiving Andembry remained free from attacks throughout the treatment period. The trial revealed a significant reduction in HAE attacks, showcasing a median decrease of over 99% and a least squares mean reduction of 89.2% when compared with a placebo.

Furthermore, data from an ongoing open-label extension study indicated that Andembry has a promising long-term safety profile, while continuing to effectively decrease the frequency of HAE attacks. Bill Mezzanotte, executive vice president and head of research and development at CSL, emphasized the groundbreaking nature of Andembry. He noted that it is the first monoclonal antibody developed entirely by CSL and provides individuals with HAE long-term control over their condition, coupled with a convenient administration method.

The CSL Behring business unit plans to roll out Andembry immediately, making it available before the end of June. Tim Craig, a professor of medicine, pediatrics, and biomedical sciences at Penn State University, expressed his approval of the FDA's decision. He highlighted the progress made in treating HAE, acknowledging that despite advancements, many patients still endure painful and potentially life-threatening attacks and frequently require injections for management.

Professor Craig was optimistic about Andembry's potential, as it introduces a novel approach by targeting factor XIIa, thereby inhibiting the start of the HAE cascade for the first time. This new option offers hope for effective management of this condition, providing relief to those who continue to suffer from recurrent HAE attacks despite existing treatments. The approval of Andembry marks a significant advancement in the ongoing fight against hereditary angioedema, offering renewed hope to patients seeking better control over their disease.