12:55 PM
Oct 13, 2017
 |  BioCentury  |  Regulation

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 not include data on these measures.

“In a perfect world, longer-lasting therapies would help me live as if I didn’t have HAE at all.”

Unidentified HAE patient

Cinryze and Haegarda are both C1-INH replacement therapies. Cinryze is delivered as a 10-minute IV infusion every three or four days. Some but not all patients use an implanted catheter. Haegarda, approved in June, is given twice weekly as a self-administered subcutaneous injection.

When an attack does occur, the acute therapies Firazyr icatibant, Kalbitor ecallantide and Berinert P have reduced attack severity in patients with abdominal, peripheral and laryngeal swelling. Ruconest conestat alfa, another acute therapy, has not demonstrated efficacy in patients with laryngeal attacks, but has reduced the severity of abdominal and peripheral HAE attacks.

Ruconest from Valeant Pharmaceuticals International Inc. and Pharming Group N.V., and Berinert P from CSL are both IV C1 esterase inhibitors that can be self-administered.


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