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News From the Food and Drug Administration
August 1, 2017

New Hereditary Angioedema Therapy

JAMA. 2017;318(5):416. doi:10.1001/jama.2017.9334

A new medication for hereditary angioedema (HAE), a rare disease that affects an estimated 6000 to 10 000 people in the United States, has received FDA approval.

Marketed as Haegarda, the drug is a C1 esterase inhibitor. People with HAE have insufficient amounts or dysfunctional forms of the plasma protein C1 esterase inhibitor, which is part of the body’s complement system and plays a role in regulating inflammation and coagulation.