Experiences From the First Year of Using Garadacimab to Treat Hereditary Angioedema and Acquired Angioedema

Background

Garadacimab, a first-in-class, recombinant monoclonal antibody targeting activated FXII, is available in Germany for long-term prophylaxis (LTP) of hereditary angioedema (HAE) since March 2025. We aimed to examine garadacimab real-life treatment outcomes at our Berlin Angioedema Center of Reference and Excellence (ACARE), the reasons for switching therapies, and approaches to tailoring treatment individually.

Methods

In total, 29 patients (27 patients with HAE and 2 patients with angioedema due to acquired C1 inhibitor deficiency) were treated in regular care with garadacimab; 3 patients discontinued garadacimab due to lack of efficacy after loading dose or second dose and were excluded from analysis due to insufficient follow up. Ultimately, 26 patients were monitored for 26 weeks (median) using the angioedema control test (AECT) and the angioedema quality of life questionnaire (AE-QoL). In seven patients, garadacimab from the open-label-VANGUARD study was continued in regular care. Patients who had previously participated in the study—provided they were symptom-free—were offered the option, upon transitioning to routine care, to extend the interval between injections by alternating between three and four days. Patients who were switched to garadacimab in routine care were offered the same option if they were completely symptom-free 2 months after starting the therapy.

Results

Overall, 16 patients switched from previous LTP to garadacimab mostly at the end of their individual treatment interval and due to the prospect of higher efficacy and convenience. The injection interval could be extended in most patients (median, 42 days, range 31–56). The mean AECT and AE-QoL improved clinically meaningfully, regardless of previous treatment (9.2–14.4 and 35.5–14.9 points, respectively), however, improvement in patients switched from previous LTP was less pronounced when compared to patients that switched from on-demand treatment (11.2–14.5 vs. 6.7–14.2 points and 29.4–14.3 vs. 43.1–15.7 points, respectively). No side effects were reported.

Conclusions

Garadacimab showed high efficacy and safety and the gradual extension of injection intervals presented underline its potential for longer dosing intervals that may further reduce treatment burden.