
CSL, a biotechnology company, has received the US Food and Drug Administration (FDA) approval for Andembry (garadacimab-gxii) to prevent hereditary angioedema (HAE) in patients aged 12 and older.
Andembry is a monoclonal antibody that inhibits Factor XIIa, the initial protein in the HAE pathway, effectively preventing the cascade of events that lead to an HAE attack.
Unlike other therapies that target downstream mediators, Andembry intervenes at the top of the cascade.
HAE is characterised by recurrent and unpredictable attacks of angioedema, affecting areas such as the abdomen, larynx, face, and extremities.
Andembry is the first treatment to target Factor XIIa, offering once-monthly dosing to prevent HAE attacks, a rare and potentially life-threatening genetic disorder.
It is administered as a subcutaneous self-injection, delivered in 15 seconds or less via an autoinjector with a citrate-free formula.
CSL executive vice president and R&D head Bill Mezzanotte said: “Andembry, the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method.
Andembry underscores our long-standing and enduring commitment to better the lives of the patients we serve, including those suffering from HAE.
“I’d like to thank all the physicians, patients, and my colleagues who contributed to this exciting milestone for HAE patients and CSL.”
The FDA approval is based on data from the Phase 3 VANGUARD trial, which demonstrated that 62% of patients treated with Andembry remained attack-free.
The trial also showed a median reduction of more than 99% in HAE attacks and a least squares mean reduction of 89.2% compared to placebo.
In addition, the trial revealed a 99% median reduction and an 88% mean reduction in attacks requiring on-demand therapy.
Moderate or severe attacks saw a 99% median reduction and a 90% mean reduction. Common adverse reactions included nasopharyngitis and abdominal pain.
An interim analysis of an ongoing open-label extension study, published in October 2024, indicated that Andembry maintains a favourable long-term safety profile.
It also showed that the drug provides sustained reductions in HAE attacks.
CSL Behring plans to commercialise Andembry in the US by the end of this month, is already been approved in Australia, the EU, Japan, Switzerland, the UK, and the UAE.
Furthermore, the company plans to launch the Andembry Connect app to support healthcare professionals and patients in accessing treatment.