Prophylactic use of an anti-activated factor XII monoclonal antibody, garadacimab, for patients with C1-esterase inhibitor-deficient hereditary angioedema: a randomised, double-blind, placebo-controlled, phase 2 trial
Phase II study (n=32) found garadacimab at the 200mg and 600mg dose (given every 4 weeks following initial IV load and dose at 6 days) reduced the number of monthly attacks versus placebo (reduced by 100% [95% CI 98–101]; p=0.0002 for 200mg and 93% [54–110]; p=0.0003) for 600mg).
Source:
The Lancet
SPS commentary:
A related comment notes this study provides the first clinical evidence that FXIIa inhibition has a good safety profile and supports the hypothesis that it can be an effective prophylactic therapy in this setting. They conclude that pending a Phase III study investigating its use in a larger and more diverse patient population, the results are promising.