Shire wins FDA approval for rare disease drug

August 25, 2011

Shire plc (NASDAQ: SHPGY) has won U.S. Food and Drug Administration approval for its drug Firazyr to treat hereditary angioedema (HAE), adding to its stable of treatments for rare diseases.

The UK-based pharmaceutical company, whose rare disease unit, Human Genetic Therapies, is based in Lexington, Mass., will compete head-to-head with fellow Massachusetts company Dyax Corp. (Nasdaq: DYAX), which won approval for its HAE treatment, called Kalbitor, in February of 2010. Uptake of Dyax’s drug has so far been disappointing, but Shire says its new treatment has the advantage of being approved for self-administration, which means patients will not have to travel to a health care facility in order to get treatment for acute attacks of the disease. Firazyr is supplied in a pre-filled syringe that can be stored at room temperature (up to 77° Fahrenheit), making it portable and accessible for immediate treatment of HAE attacks.

Dyax’s drug has also been slapped with a black box warning – the most serious issued by the FDA – because of the risk of anaphylaxis. Shire executives, in a briefing call with the Boston Business Journal last week, said clinical trials studying Firazyr did not show similar risks.

Shire’s rare disease franchise includes two competitors to Cambridge, Mass.-based Genzyme Corp.: Vpriv, approved to treat Gaucher disease, and a competitor of Cerezyme, and Replagal, to treat Fabry disease, which is a competitor of Fabrazyme in Europe, but not yet approved in the U.S. Genzyme is owned by Sanofi SA (NYSE: SNY)

“Shire has extensive experience meeting the unique needs of patients with rare diseases due to our leadership in this area,” Shire HGT President Sylvie Gregoire said in a statement. “With Firazyr now approved in 38 countries, we are pleased to bring both this novel treatment and our comprehensive OnePath patient service and support program to U.S. HAE patients.”

Hereditary angiodema is estimated to affect between 5,000 and 10,000 patients, and its chief symptom is severe swelling, similar to hives, of the extremities, abdomen or throat, which can be life-threatening.

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