FDA Licenses For Marketing New Therapy for Rare Genetic Disease

The FDA has 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... [read full story]