Can new treatment garadacimab prevent attacks in patients with hereditary angioedema?

This video article presents the results of the VANGUARD phase III study, a pivotal, multicentre, randomized, double-blind, placebo-controlled trial investigating the efficacy and safety of garadacimab (a novel, fully human monoclonal antibody that inhibits activated factor XII), for prevention of hereditary angioedema attacks.

Despite advances in the management of hereditary angioedema, there is an unmet need for convenient prophylactic treatments that show sustained efficacy in preventing hereditary angioedema attacks with a favorable safety profile.

The VANGUARD pivotal phase III study demonstrated that once-monthly subcutaneous administration of garadacimab significantly reduced the number of hereditary angioedema attacks in patients aged 12 years and older compared with placebo and had a favourable safety profile. Most patients treated with garadacimab did not experience any hereditary angioedema attacks through to the end of the treatment period of 6 months. None of the patients who received placebo were protected from hereditary angioedema attacks. These results support the use of garadacimab as a long-term prophylactic therapy for the treatment of hereditary angioedema in adolescents and adults.

Meet the author(s):

Dr. Timothy Craig has been a clinical researcher for more than 23 years and has participated in NHLBI asthma research consortiums, including ACRN and AsthmaNet. He is a leader in the field of allergy and immunology and has written guidelines for international management of urticaria, hereditary angioedema and rhinitis. He has more than 300 publications and around 400 invited lectures.

His clinical research and clinical care focuses include asthma, COPD, alpha-1-antitrypsin deficiency, urticaria, hereditary angioedema, immunodeficiency, vaccine response and other respiratory, allergic and immunologic diseases.

Original article(s):

Craig TJ, Reshef A, Li HH, et al. Efficacy and safety of garadacimab, a factor XIIa inhibitor for hereditary angioedema prevention (VANGUARD): a global, multicentre, randomised, double-blind, placebo-controlled, phase 3 trial [published correction appears in Lancet. 2023;401(10382):1079-1090. doi:10.1016/S0140-6736(23)00350-1. Lancet. 2023 Apr 15;401(10384):1266. doi: 10.1016/S0140-6736(23)00739-0.

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