Lancet: Garadacimab is effective in preventing edema episodes in patients with C1 esterase inhibitor deficiency hereditary angioedema

Hereditary angioedema is an autosomal dominant disorder characterized by recurrent, unpredictable, submucosal edema of the skin and mucous membranes, which can be life-threatening by asphyxiation in the presence of Hericium edema
.

There is currently no cure, and the only way to prevent it is through medication

Hereditary angioedema is an autosomal dominant disorder characterized by recurrent, unpredictable, submucosal edema that can be life-threatening by asphyxiation in the presence of Hericium edema

Hereditary angioedema is currently known to be associated with dysregulation of the kallikrein-kinin system
.

Factor XII (FXII) is a key elicitor of the kallikrein-kinin system, which induces the production of bradykinin, a central mediator of angioedema

Garadacimab (CSL Behring) is a first-line fully human immunoglobulin G4 monoclonal antibody targeting activated FXII to prevent attacks in patients with C1-esterase inhibitor-deficiency hereditary angioedema (HAE-C1-INH)

This is a double-blind, placebo-controlled Phase 2 study that recruited patients aged 18-65 years from multiple countries who had at least 4 severe attacks in the 3 months prior to enrollment
.

After a 4-8 week run-in period, patients were randomized into four groups (1:1:1:1) to receive placebo, Garadacimab 75mg, 200mg or 600mg (every 4 weeks)

Between October 29, 2018, and August 28, 2019, a total of 54 patients were screened, of whom 32 were randomized into four groups: 8 in the placebo group, 9 in the Garadacimab 75 mg group, and 8 in the Garadacimab 200 mg group.
Garadacimab 600mg group 7th
.

The median age of patients tested was 39.

The number of edema episodes per month in each group

The number of edema episodes per month in each group

The median number of seizures per month during the 12-week dosing period was 4.
6, 0, 0, and 0.
3 in the placebo, Garadacimab 75 mg, Garadacimab 200 mg, and Garadacimab 600 mg groups, respectively
.

Compared with placebo, the incidence of edema episodes was significantly reduced after Garadacimab intervention (200mg group, decreased by 100%, p=0.

The median number of seizures per month during the 12-week dosing period was 4.

adverse events

adverse events

In conclusion, Garadacimab (200 mg or 600 mg) administered every four weeks significantly reduced edema episodes in patients with C1-esterase inhibitor-deficiency hereditary angioedema and was well tolerated
.

Therefore, Garadacimab is a good option for preventing attacks in patients with HAE-C1-INH and warrants further evaluation in a phase 3 trial

Garadacimab (200 mg or 600 mg) every four weeks significantly reduced edema episodes in patients with C1 esterase inhibitor-deficiency hereditary angioedema and was well tolerated

Original source:

Original source:

Timothy Craig, et al.

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