FDA Approves Kalbitor for Treating Potentially Life-Threatening Attacks of Hereditary Angioedema

The U.S. Food and Drug Administration approved Kalbitor (ecallantide) on Dec. 1 to treat sudden and potentially life-threatening fluid buildup that can occur in people with a rare genetic condition known as hereditary angioedema (HAE).

HAE is caused by a defect in the blood protein C1 esterase-inhibitor, which plays a role in regulating how certain immune system and blood clotting pathways function. Decreased function of this protein can lead to rapid and serious swelling of the face or other parts of the body, which may result in permanent disfigurement, disability or death. Swelling of the digestive tract may cause excruciating abdominal pain, nausea, and vomiting, while airway swelling puts patients at risk of suffocation. About 10,000 people in the United States have HAE.

“Kalbitor offers another potentially life-saving option for those people with HAE,” said John Jenkins, M.D., director, Office of New Drugs, in the FDA’s Center for Drug Evaluation and Research. “FDA is committed to facilitating the development and approval of safe and effective therapies for patients with rare conditions.”

This is the second drug marketed in the United States to treat HAE attacks. In October 2009 the FDA approved Berinert, a C1-inhibitor derived from blood plasma to treat facial and abdominal attacks of HAE.

Kalbitor is a liquid that is intended to be injected under the skin for patients age 16 and older who experience HAE attacks.

The medication’s most serious side effect is anaphylaxis, a severe allergic reaction that can close a person’s airways and stop them from breathing. Other side effects include headache, nausea, diarrhea, swelling in the nose and throat, fever, and skin irritations.

Kalbitor should only be administered by a healthcare professional with appropriate medical support to manage anaphylaxis.

The drug is marketed by Dyax Corp., Cambridge, Mass. The second HAE drug, Berinert, is marketed by CSL Behring Inc., Marburg, Germany.

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FDA Licenses for Marketing New Therapy for Rare Genetic Disease

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.

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