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12:00 AM
Jun 27, 2011
 |  BioCentury  |  Regulation

Firazyr tips the scales

FDA panel backs Shire's Firazyr - potential game-changer for acute HAE attacks

Following last week's positive FDA panel vote, Shire plc's Firazyr icatibant is poised to become the first product approved in the U.S. for self administration to treat acute attacks of hereditary angioedema in adults. Uptake could be rapid, as physicians at FDA's Pulmonary-Allergy Drugs Advisory Committee touted the compound's potential to treat acute HAE attacks more quickly and with fewer side effects than current drugs.

The company's robust dataset persuaded the panel that a novel measure of primary endpoints in Phase III trials of Firazyr was meaningful: the committee voted 12-1 in favor of approval, and 11-1 with one abstention that the data support self administration.

HAE is an autosomal-dominant condition in which individuals have either low levels of complement 1 (C1) esterase inhibitor or mutated C1 that is non-functional.

Symptoms include dramatic swelling at various sites, including the face, airways and gastrointestinal tract. The attacks can occur spontaneously and typically last for one to four days. Stress and anxiety can trigger HAE attacks.

Laryngeal attacks are life-threatening - a patient can die of asphyxia in a matter of minutes. Of the 6,000-30,000 HAE patients in the U.S., about 50% will experience a laryngeal attack. If not treated, the mortality rate is about 30%.

Marcus Maurer, a professor of dermatology and allergy at the Charite Universitaetsmedizin Berlin who spoke on behalf of Shire at the meeting, said HAE patients who carry Firazyr with them tend to have fewer attacks. He said many patients have told him anecdotally that they live with less stress and fear of having an attack since they can manage it themselves.

The drug was approved in the EU in 2008 to treat acute HAE attacks, with a label extension approved in February to allow for self administration.

Robust data

Going into the meeting, FDA had questions about the whether the company's novel endpoint based...

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