Hereditary Angioedema (HAE)
HAE is a life threatening genetic condition based on a lack of C1-esterase inhibitor (C1-INH), resulting in excessive levels of a protein called bradykinin which causes blood vessels to dilate. HAE patients suffer from rapid and painful attacks in the abdomen, arms, legs, feet, hands, lips, eyes, tongue, or throat and vary in frequency from a few attacks a year in mild cases to two or more attacks per month in severe forms of the disease. Attacks often do not have a known cause, and mortality rates for events that involve an airway have been reported to be as high as 30%. There are approximately 1 in 10,000 to 1 in 50,000 patients worldwide with HAE, with approximately 10,000 patients diagnosed across major markets1. We estimate approximately 80% are moderate to severe patients. HAE patients are managed either by acute treatments (multiple approved options) or by preventive therapy which is costly and burdensome, requiring infusions every three or four days.
- Overview of epidemiology, pathophysiology, and disease progression in hereditary angioedema. Lumpy WR1. Am J Manag Care. 2013 Jun;19(7 Suppl):s103-10.