Marketing approval for
orphan drugs, specialty drugs created to treat rare diseases, is always exciting. In 2008, the U.S. Food and Drug Administration (FDA) approved several of these drugs that were particularly important.
The FDA granted marketing approval to Arcalyst (rilonacept) for the treatment of cryopyrin-associated periodic syndromes (CAPS) in adults and children 12 and older. CAPS is a group of inherited autoinflammatory conditions characterized by fever, rash, and joint pain. Intermittent exacerbations or flares can be triggered at any time by exposure to cooling temperatures, stress, exercise, or other unknown stimuli. CAPS affects about 300 people in the United States.
People with
hereditary angioedema (HAE), a rare and potentially life-threatening genetic disease, now have this treatment drug, approved by the FDA in 2008. Cinryze is licensed for the prevention of HAE attacks, in which swelling of body parts such as hands, feet, face, arms, or legs occurs; this can include life-threatening swelling of the airway.
The
seizures involved in
Lennox-Gastaut syndrome, a severe form of epilepsy, are difficult to control. Banzel (rufinamide) was approved by the FDA for treatment of seizures associated with Lennox-Gastaut syndrome in children and adults. In a clinical study, treatment with Banzel reduced total seizures in the syndrome by about a third.
Xenazine is the first drug of any kind approved in the United States to treat any symptoms of
Huntington's disease, an inherited nervous system disorder. It's also the first FDA-approved drug to treat chorea, the jerky, involuntary muscle movement that occurs in people with this disease. Xenazine has been available in a number of countries in Europe for more than 30 years.
