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FDA Approves Factor XIIa-Targeting Garadacimab-gxii (ANDEMBRY) for HAE Attacks
The monoclonal antibody garadacimab-gxii offers once-monthly, citrate-free dosing to prevent hereditary angioedema attacks in patients aged ≥ 12 years.
The US Food and Drug Administration (FDA) approved garadacimab-gxii (ANDEMBRY), the only treatment targeting factor XIIa for prophylactic use to prevent hereditary angioedema (HAE) attacks in adults and children aged ≥ 12 years. The global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) announced this approval on June 16, 2025.1
Garadacimab-gxii is the first HAE treatment to offer all patients once-monthly dosing from the start. Delivered via a citrate-free, subcutaneous autoinjector in ≤15 seconds, it targets the top of the HAE cascade to prevent attacks. Garadacimab-gxii was previously approved for HAE attacks in Australia, the United Kingdom, the European Union, Japan, Switzerland, and the United Arab Emirates.
“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 at CSL, in a statement.
HAE is a rare and chronic genetic disorder characterized by recurrent and unpredictable attacks of angioedema. Attacks can be painful—even life-threatening—and can affect several sites on the body, including the abdomen, larynx, face, and extremities. About 1 in 50,000 people have HAE.
The decision was based on pivotal results from the placebo-controlled phase 3 VANGUARD trial, published in The Lancet in April 2023, assessing the efficacy and safety of garadacimab-gxii for the prophylactic treatment of HAE attacks. In VANGUARD, participants were randomized 3:2 to receive a loading dose of 400 mg followed by 200 mg of garadacimab-gxii monthly (n = 39) or volume-matched placebo monthly (n = 25) subcutaneously for 6 months.
The trial found 62% of patients treated with garadacimab-gxii were attack-free during the treatment period, and HAE attacks in the garadacimab-gxii arm were reduced by ≥ 99% compared with placebo.
Participants on garadacimab-gxii achieved ≥ 99% median reduction and an 88% mean reduction in HAE attacks requiring on-demand therapy. Not only that, but participants on garadacimab-gxii had more than a 99% median reduction and a 90% mean reduction in moderate or severe attacks, compared to placebo.
Nasopharyngitis and abdominal pain were common adverse events in the pivotal trial, with an incidence of ≥ 7%.
In addition to the VANGUARD trial, an interim analysis published in October 2024 assessed long-term garadacimab-gxii exposure, a median of 13.8 months. This ongoing open-label extension study demonstrated the favorable long-term safety and efficacy of garadacimab-gxii. In total, 23 participants reported injection-site injections, including injection-site bruising, injection-site erythema, injection-site hematoma, injection-site pruritus, and injection-site urticaria.
“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 Tim Craig, professor of medicine, pediatrics, and biomedical sciences at Penn State University, in a statement. "We now have a new option to manage this condition through a new target, as it allows us, for the first time, to inhibit the top of the HAE cascade by targeting factor XIIa."
Garadacimab-gxii will be available in the US before the end of June.
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