Expanding the HAE tool kit

What patients told FDA they want from new HAE therapies

Despite an increasingly crowded market, hereditary angioedema patients who attended FDA’s Patient-Focused Drug Development meeting said there remains room for improvement in dosing convenience and reducing attack frequency.

Two subcutaneous prophylactics that have completed Phase III trials and two oral therapies in earlier clinical development have the potential to meet these patients’ needs.

Hereditary angioedema (HAE) is an autosomal-dominant condition in which individuals have either low levels of C1 esterase inhibitor (C1-INH) or mutated C1-INH that is non-functional.

The disease is characterized by sudden attacks of swelling in different parts of the body. The frequency, location, severity and duration of attacks vary between and within individual patients. Attacks can be fatal when they affect the larynx.

Since 2008, six new drugs have been approved to treat or prevent HAE attacks. The prophylactic treatments Cinryze from Shire plc and Haegarda from CSL Ltd. have reduced the frequency of attacks. Cinryze has also reduced attack severity and duration; Haegarda’s label does

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