FDA Approves C1 Esterase Inhibitor to Treat Genetic Disease

Officials with the FDA have approved the first C1 esterase inhibitor (human) for subcutaneous administration to prevent hereditary angioedema (HAE) attacks in adolescent and adult patients.

Haegarda (CSL Behring LLC) is a human plasma-derived, purified, pasteurized, lyophilized concentrate prepared from large pools of human plasma from US donors. Haegarda is indicated for routine prophylaxis to prevent HAE attacks, but is not indicated for treatment of acute HAE attacks.

The efficacy of Haegarda was demonstrated in a multicenter controlled clinical trial, according to a news release from the FDA. The study included 90 subjects ranging in age from 12 to 72 years old with symptomatic HAE. Patients were randomly assigned to receive twice per week subcutaneous doses of either 40 IU/kg or 60 IU/kg, and the treatment effect was compared to a placebo treatment period. During the 16-week treatment period, patients in both treatment groups experienced a significantly reduced number of HAE attacks compared to their placebo treatment period.

The most common side effects included injection site reactions, hypersensitivity (allergic) reactions, nasopharyngitis, and dizziness. Haegarda should not be used in individuals who have experienced life-threatening hypersensitivity reactions, including anaphylaxis, to a C1-INH preparation or its inactive ingredients.

Haegarda received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs to treat rare diseases or conditions.

HAE, which is caused by having insufficient amounts of a plasma protein called C1-esterase inhibitor (or C1-INH), affects approximately 6,000 to 10,000 people in the United States. People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract or airway. These attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection.


FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease [news release]. Bethesa, Md. FDA website. Accessed at: https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm564332.htm

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