Hereditary diseases

FDA Approves New Method for Treating Hereditary Angioedema

A subcutaneously administered C1 Esterase Inhibitor, Haegarda, was recently approved by the US Food and Drug Administration for the prevention of Hereditary Angioedema (HAE) attacks in adult and adolescent patients.

Haegarda is a human plasma-derived, purified, pasteurized, lyophilized concentrate that is indicated for routine prophylaxis to prevent HAE attacks. The new subcutaneous route will make it easier for patients or caregivers to administered the drug at home after receiving the proper training.
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Data from a multicenter, controlled clinical trial was used to determine the efficacy of Haegarda. Ninety subjects from 12 to 72 years of age were included in the study and randomly assigned to receive doses of either 40 IU/kg or 60 IU/kg of Haegarda two times per week for 16 weeks, followed by a placebo treatment period. Patients in both treatment groups experienced a significantly reduced number of HAE attacks during the 16 weeks of treatment compared with the placebo period.

Common adverse effects associated with Haegarda included injection site reactions, hypersensitivity reactions, nasopharyngitis, and dizziness. Patients who have experienced life-threatening hypersensitivity reactions to a C1-INH preparation or its inactive ingredients should not receive Haegarda.

—Melissa Weiss

Reference:

FDA approves first subcutaneous C1 Esterase Inhibitor to treat rare genetic disease [press release]. FDA. June 22, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm564332.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed June 23, 2017.