FDA Licenses For Marketing New Therapy For Rare Genetic Disease

Main Category: Regulatory Affairs / Drug Approvals
Article Date: 13 Oct 2008 - 5:00 PDT

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The U.S. Food and Drug Administration today licensed for marketing the first product in the United States intended to protect people with hereditary angioedema (HAE), a rare and potentially life-threatening genetic disease. HAE affects about 6,000 to 10,000 individuals in the United States.

The product, called Cinryze, is licensed for the prevention of HAE attacks, which can occur spontaneously or during stress, surgery, or infection in patients diagnosed with the disease. Attacks can produce rapid swelling of the hands, feet, limbs, face, intestinal tract or airway. Swelling of the larynx can lead to asphyxiation.

"Cinryze should greatly enhance treatment options for those with hereditary angioedema and potentially save lives," said Jesse Goodman, M.D., M.P.H., director of the FDA's Center for Biologics Evaluation and Research.

Cinryze is a C1-esterase inhibitor product derived from human plasma. This plasma protein regulates clotting and inflammatory reactions that, when impaired, can lead to local tissue swelling. C1-esterase inhibitor is low or does not function properly in individuals with HAE. In clinical trials, Cinryze was effective in preventing or decreasing the frequency of attacks in most but not all HAE patients. Adverse reactions reported in the study were considered mild or moderate in severity.

Cinryze is administered intravenously and can be used every three or four days for routine prevention of HAE attacks. It is manufactured by Lev Pharmaceuticals Inc., New York, N.Y., through a contract manufacturing agreement with Sanquin Blood Supply Foundation in The Netherlands.

http://www.fda.org