KING OF PRUSSIA, PA — The U.S. Food and Drug Administration (FDA) has approved ANDEMBRY® (garadacimab-gxii), developed by CSL, as the first treatment targeting factor XIIa for the prevention of hereditary angioedema (HAE) attacks. This milestone marks a significant advancement in managing the rare, chronic, and potentially life-threatening condition, offering patients a more convenient and effective treatment option.
ANDEMBRY is the only prophylactic HAE treatment to use once-monthly subcutaneous dosing from the start. Delivered in under 15 seconds via an autoinjector with a citrate-free formula, the treatment provides long-term disease control while enhancing patient convenience. By targeting factor XIIa, a plasma protein central to the HAE attack cascade, ANDEMBRY inhibits the triggering mechanism of swelling episodes.
“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,” said Bill Mezzanotte, MD, Executive Vice President and Head of R&D at CSL. “This approval underscores our commitment to bettering the lives of patients, including those with HAE, and I thank everyone involved in achieving this milestone.”
The FDA’s decision is supported by data from the Phase 3 VANGUARD trial. The study demonstrated a 99% median reduction in HAE attacks and an 89.2% mean reduction in attack frequency compared to placebo. Furthermore, 62% of patients treated with ANDEMBRY were attack-free for the trial’s duration. Results also showed a 99% median reduction in attacks requiring on-demand therapy and a similar reduction in moderate-to-severe episodes. The most common adverse reactions reported included nasopharyngitis and abdominal pain.
“We’ve made significant progress in treating hereditary angioedema, yet many patients still experience painful and sometimes life-threatening HAE attacks and require frequent injections to manage them,” said Dr. Tim Craig, Professor of Medicine at Penn State University. “This new treatment allows us, for the first time, to inhibit the top of the HAE cascade by targeting factor XIIa.”
A longer-term analysis from an ongoing open-label extension study highlights the safety and sustained effectiveness of ANDEMBRY after a median exposure of 13.8 months.
HAE affects roughly 1 in 50,000 people worldwide, and symptoms include unpredictable and painful swelling episodes across various parts of the body. Effective management of the disease is crucial for improving patients’ quality of life.
“ANDEMBRY, a novel once-monthly subcutaneous treatment that inhibits factor XIIa, is a welcome addition to the HAE treatment landscape,” added Anthony J. Castaldo, CEO and Chairman of the Board for the US HAE Association and HAE International. “This approval gives people with HAE another option to lessen the burden of this lifelong condition, enabling them to live life to the fullest.”
The approval of ANDEMBRY in the U.S. follows regulatory approvals in markets including Australia, the UK, the EU, Japan, and Switzerland. CSL Behring is preparing for its immediate commercial launch, with the treatment expected to be available before the end of June.
Healthcare professionals and patients are encouraged to access ANDEMBRY Connect, which offers comprehensive support and resources for onboarding and managing the new therapy. This FDA approval represents an important step forward in expanding treatment options for HAE patients globally.
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