Garadacimab up for approval as HAE therapy in US, Europe

VANGUARD study tested therapy against a placebo in 64 patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The monthly under-the-skin injection therapy garadacimab will be considered for approval for treating hereditary angioedema (HAE) in both the U.S. and the European Union.

Developer CSL Behring announced that both the U.S. Food and Drug Administration and the European Medicines Agency have agreed to review applications of the therapy for HAE.

“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,” Emmanuelle Lecomte Brisset, senior vice president and global head of regulatory affairs at CSL, said in a company press release.

Recurrent swelling attacks are a feature of the genetic disorder. Garadacimab is an antibody-based therapy designed to prevent swelling by blocking the activity of factor XII (FXII), a protein that spearheads the cascade of events that lead to attacks. If approved, garadacimab would become the first approved HAE therapy that targets activated FXII in both the U.S. and EU.

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VANGUARD data support garadacimab applications

“CSL is a company with a deep heritage in developing innovative treatments for the rare disease community, and we are extremely proud that our first homegrown recombinant monoclonal antibody is progressing our commitment to support HAE patients in need,” Brisset said.

CSL’s applications for garadacimab are based primarily on data from the Phase 3 clinical trial VANGUARD (NCT04656418), which tested it against a placebo in 64 people with HAE, ages 12 and older.

Top-line results announced last year showed the experimental therapy outperformed the placebo at reducing the rate of swelling attacks. The average rate of attacks was 0.27 per month for patients on the therapy, compared with about two a month for those on the placebo.

Full results, published this year, showed nearly two-thirds of patients treated with garadacimab were completely free from swelling attacks over the six-month clinical trial. VANGUARD data also indicated the therapy was generally well tolerated and led to improvements in quality of life for patients.

Most participants elected to continue into an open-label extension study (NCT04739059) where all are receiving long-term treatment with garadacimab at 200 mg a month. The extension study is expected to run through 2025.