FDA Approves Treatment for Rare Swelling Disease

test_tubes_flaskBiopharmaceutical company Dyax Corporation has received regulatory approval to market its drug ecallantide (Kalbitor) for a rare genetic disorder called hereditary angioedema (HAE). The drug is approved to treat patients age 16 and up. Shares in Dyax rose 37 percent following the announcement.

HAE causes potentially life-threatening swelling of the face, feet, lungs, digestive tract, airway and other parts of the body. It is caused by a deficiency of C1-INH, a blood protein that helps regulate the function of certain immune system and blood clotting pathways. Swelling in the digestive tract can cause nausea, vomiting and diarrhea. Swollen airways can obstruct breathing and lead to suffocation. About 10,000 people in the U.S. suffer from HAE.

The U.S. Food and Drug Administration initially rejected Kalbitor in March, requesting additional safety data. The most serious side effect of Kalbitor is anaphylaxis, an allergic reaction that can close a patient’s airways and cause suffocation.

Kalbitor is the first approved product for Cambridge, Mass.-based Dyax, and only the second HAE treatment on the market. In October, CSL Behring‘s Berinert was approved for the treatment of acute facial and abdominal attacks of HAE. Berinert is a plasma-derived therapy that supplies patients with additional C1-INH.

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