The Medical Letter on Drugs and Therapeutics
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1541
Haegarda - A Subcutaneous C1 Esterase Inhibitor for Prevention of Hereditary Angioedema
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The FDA has approved Haegarda (CSL Bering), a human plasma-derived C1 esterase inhibitor (C1INH), for prophylaxis against hereditary angioedema (HAE) attacks in adolescents and adults. Haegarda is the fourth C1INH to be approved by the FDA and the second to be approved for HAE prophylaxis. Unlike the other C1INHs, Haegarda is self-administered subcutaneously rather than intravenously.

THE DISEASE — HAE, an autosomal dominant disorder with an estimated prevalence of 1:10,000-50,000, is usually caused by a mutation of the C1INH gene (types 1 and 2). A deficiency in endogenous or functional C1INH leads to an overproduction of bradykinin, a vasoactive substance that increases vascular permeability and is believed to be the mediator of acute angioedema attacks. Patients experience recurrent, often unpredictable attacks of angioedema lasting 2-5 days, typically involving the extremities, GI ... more      

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Title: Haegarda - A Subcutaneous C1 Esterase Inhibitor for Prevention of Hereditary Angioedema
Article code: 1541d
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