FDA Accepts BLA for Garadacimab as a Once-Monthly Treatment for Hereditary Angioedema


Previously, garadacimab was granted an orphan drug designation as a therapy for hereditary angioedema by both the FDA and EMA.

The FDA has accepted a Biologics License Application (BLA) for garadacimab (CSL312; CSL Behring) as a once-monthly prophylactic treatment for hereditary angioedema (HAE), a rare, genetic, and potentially life-threatening condition that causes debilitating and unpredictable swelling of the abdomen, larynx, face, and extremities. In addition, the European Medicines Agency (EMA) has accepted submission of the Marketing Authorization Application (MAA) for garadacimab.1

Man experiencing swelling in face

Image credit: Ольга Симонова | stock.adobe.com

If approved, garadacimab would be the first treatment for HAE in the United States and in Europe to target activated Factor XII (FXIIa). This novel, first-in-class, recombinant monoclonal antibody targets activated FXII. FXIIa, a plasma protein, initiated the kallikrein-kinin cascade of HAE attacks, and by targeting FXIIa, garadacimab inhibits the cascade of HAE attacks at the top rather than other HAE therapies which target downstream mediators. Previously, garadacimab was granted an orphan-drug designation as a therapy for individuals with HAE by both the FDA and EMA.1

The BLA and MAA come after results from the pivotal, multicenter, randomized, double-blind, parallel-group VANGUARD trial that evaluated the long-term efficacy and safety of monthly 200-mg doses of garadacimab as a prophylactic treatment for patients with HAE. Enrolled patients received either garadacimab (400-mg dose of subcutaneous garadacimab as 2 200-mg injections) or volume-matched placebo, followed by 5 additional self-administered monthly doses of 200-mg subcutaneous garadacimab or volume-matched placebo. The primary endpoint was the investigator-assessed time-normalized number of HAE attacks per month during the 6-month treatment period, with safety being evaluated in patients who received at least 1 dose of garadacimab or placebo.1,2

The study results found that during the 6-month treatment period, the mean number of investigator-confirmed HAE attacks per month was significantly lower in the garadacimab group (0.27; 95% CI 0.05-0.49) than in the placebo group (2.01; 1.44-2.57; p < 0·0001). The mean number of HAE attacks per month in the garadacimab group was 0 (IQR: 0-0.31) and 1.35 (IQR: 1.00-3.20) in the placebo group. With this milestone, garadacimab is a step closer to delivering first-in-class recombinant monoclonal antibody therapy for patients living with HAE.1,2

"…we are extremely proud that our first homegrown recombinant monoclonal antibody is progressing our commitment to support HAE patients in need," said Emmanuelle Lecomte Brisset, PharmD, senior vice president and global head of regulatory affairs at CSL, in a press release. "We believe that garadacimab has the potential to become a promising therapy in the prevention of HAE attacks and we look forward to working closely with global health regulators throughout the review process."1


1. CSL Behring. CSL's Garadacimab, a First-in-Class Factor XIIa Inhibitor, Receives FDA and EMA Filing Acceptance. News release. December 14, 2023. Accessed December 18, 2023. https://www.prnewswire.com/news-releases/csls-garadacimab-a-first-in-class-factor-xiia-inhibitor-receives-fda-and-ema-filing-acceptance-302016048.html

2. 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 Apr 15;401(10384):1266]. Lancet. 2023;401(10382):1079-1090. doi:10.1016/S0140-6736(23)00350-1

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