FDA approves first subcutaneous C1 esterase Inhibitor to treat rare genetic disease

FDA

22 June 2017 - The U.S. FDA today approved Haegarda, the first C1 esterase inhibitor (human) for subcutaneous administration to prevent hereditary angioedema attacks in adolescent and adult patients.

The subcutaneous route of administration allows for easier at-home self-injection by the patient or caregiver, once proper training is received.

Hereditary angioedema (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 U.S. 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.

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Michael Wonder

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Michael Wonder

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Outcome , US , Orphan drug , Blood product