The FDA recently approved Andembry (garadacimab-gxii) from CSL for the prophylactic treatment of hereditary angioedema (HAE). This approval marks a significant milestone as Garadacimab is the first and only Factor XIIa inhibitor approved for routine prevention of HAE attacks, offering once-monthly dosing for all patients from the start. Additionally, Andembry is approved for subcutaneous, self-injection delivered in 15 seconds or less via an autoinjector with a citrate-free formula.
Hereditary angioedema is a rare, genetic disorder characterized by unpredictable and potentially life-threatening swelling episodes in various parts of the body, including the face, abdomen, and airways. The condition is caused by a deficiency or dysfunction of the C1 esterase inhibitor, leading to uncontrolled activation of the kallikrein-kinin system and excess bradykinin production.
Garadacimab targets activated Factor XII (Factor XIIa), a key driver in the inflammatory cascade of HAE. Its unique mechanism represents a novel therapeutic class, providing a preventive option without requiring titration or loading doses. In clinical trials, Garadacimab demonstrated statistically significant reductions in HAE attacks with a favorable safety profile. Common side effects reported in trials included injection site reactions, upper respiratory tract infections, and headache. No serious treatment-related adverse events were observed.
Specific cost details have not yet been disclosed publicly as of the approval date.
Andembry will be available in the U.S. through specialty pharmacies.
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U.S. Food and Drug Administration Approves CSL’s ANDEMBRY (garadacimab-gxii), the Only Prophylactic Hereditary Angioedema (HAE) Treatment Targeting Factor XIIa with Once-Monthly Dosing for All Patients From the Start
CLICK HERE to read the full press release