Long-Term Integrated Safety and Efficacy of Garadacimab for Hereditary Angioedema Prophylaxis.

Authors

Timothy J Craig, Mar Guilarte, Huamin Henry Li, John Anderson, Inmaculada Martinez Saguer, Joshua S Jacobs, Raffi Tachdjian, Henriette Farkas, William H Yang, Isao Ohsawa, Roman Hakl, Maressa Pollen, Ingo Pragst, John-Philip Lawo, Harsha Shetty, Chiara Nenci, Markus Magerl

Published in

Advances in therapy. Jun 10, 2026. Epub Jun 10, 2026.

Abstract

Garadacimab (monoclonal antibody inhibiting activated factor XII [FXIIa]) is approved for long-term prophylaxis (LTP) against hereditary angioedema (HAE) attacks. Due to the novelty of FXIIa inhibition and the lifelong need for HAE LTP, long-term evaluation of garadacimab is important. Pooling data from multiple studies can provide valuable insights into treatment effects over longer durations. We report the integrated summary of safety (ISS) and efficacy (ISE) of garadacimab LTP across the HAE clinical development program.
The ISS comprised data from phase 2 (13-week placebo-controlled period, 75/200/600 mg monthly or 400 mg every 2 weeks; ≥ 44-week open-label period, 200/600 mg monthly; NCT03712228), pivotal phase 3 (6 months; 200 mg monthly; NCT04656418), and ≥ 12-month phase 3 open-label extension (OLE) studies (200 mg monthly; NCT04739059). The ISE comprised data from phase 3 studies. Endpoints included treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), adverse events of special interest (AESIs) per protocol, and efficacy.
At data cutoff (June 15, 2024), median garadacimab exposure (ISS: any dose, n = 172) was 2.5 (range 0.2-5.5) years. Overall rates of TEAEs and garadacimab-related TEAEs were 2.8/patient-year and 0.2/patient-year, respectively. No deaths occurred; four TEAEs led to treatment discontinuation. Eleven patients experienced SAEs (0 garadacimab-related). No garadacimab-related AESIs were reported; one unrelated AESI was observed with garadacimab 600 mg (epistaxis; mild; resolved). The most common garadacimab-related TEAEs were mild/moderate injection-site reactions. In the ISE (median exposure 2.5 [range 0.3-3.2] years), the mean (95% confidence interval) monthly attack rate was 0.2 (0.1, 0.2) with garadacimab (n = 164), corresponding to a 94.9% (93.1, 96.7) reduction from run-in (3.5 [3.2, 3.9]).
These integrated data, representing the longest garadacimab exposure reported to date, confirm the favorable long-term safety profile of garadacimab (exposure ≤ 5.5 years), and durable efficacy with sustained protection against HAE attacks (exposure ≤ 3.2 years).
ClinicalTrials.gov identifiers, NCT03712228, NCT04656418, NCT04739059.

PMID:
42268494
Bibliographic data and abstract were imported from PubMed on 10 Jun 2026.

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