Rare Disease Report

FDA Approves Subcutaneous C1 Esterase Inhibitor for HAE Prophylaxis

JUNE 22, 2017
James Radke
The U.S. Food and Drug Administration (FDA) has approved CSL Behring’s  Haegarda, a C1 Esterase Inhibitor that can be administered subcutaneously, to prevent Hereditary Angioedema (HAE) attacks.
The subcutaneous route makes it much easier for patients and caregivers to manage HAE. All other C1 Esterase Inhibitors (Cinryze for preventing attacks; Berinert to treat acute attacks; and Ruconest to treat acute attacks) are administered intravenously.
HAE is a rare genetic disorder in which patients experience uncontrolled swelling of their extremities, face, and abdomen. The attacks can be life threatening if swelling of the airways occurs.
HAE patients lack a properly functioning C1 esterase inhibitor and that can result in fluid from the blood leaking into connective tissue, causing severe pain and swelling. The attacks can be spontaneous but may be linked to triggers such as stress or infections.
Per the FDA, "The approval of Haegarda provides a new treatment option for adolescents and adults with Hereditary Angioedema," said Peter Marks, M.D., Ph.D., director of FDA’s Center for Biologics Evaluation and Research. "The subcutaneous formulation allows patients to administer the product at home to help prevent attacks."
Haegarda is a human plasma-derived, purified, pasteurized, lyophilized (freeze-dried) concentrate prepared from large pools of human plasma from U.S. donors. Haegarda is indicated for routine prophylaxis to prevent HAE attacks in patients, 12 years and older, with HAE.
The approval of Haegarda was based on a clinical trial in which 90 patients with symptomatic HAE were randomized to receive subcutaneous doses of Haegarda (40 IU/kg or 60 IU/kg) or placebo twice a week for 16 weeks. The study observed that patients in both Haegarda treatment groups had significantly fewer HAE attacks compared to the placebo group.
The most common side effects included injection site reactions, hypersensitivity (allergic) reactions, nasopharyngitis (swelling of the nasal passages and throat) and dizziness.

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