Cystagon (cysteamine bitartrate) was approved by FDA last Aug. 15 to treat children with a rare kidney disease.
The disease, nephropathic cystinosis, is characterized by the buildup of an amino acid called cystine in the kidneys. Children with the disorder suffer progressive kidney failure and weak bones, and don't reach their normal height.
During the past 15 to 20 years, most children with the condition had to undergo kidney transplants. Before that, most died by age 10.
Studies showed that Cystagon can stop or slow the progressive kidney failure if patients get treatment early--before substantial kidney damage--and take the drug regularly. Although patients treated with the drug maintained growth, they did not catch up to their normal height. The studies included most of the 200 pre-transplant nephropathic cystinosis patients in the United States.
Side effects include nausea, vomiting, loss of appetite, drowsiness, and skin rash.
Cystagon was designated an orphan drug under the Orphan Drug Act, which provides economic incentives to encourage development of drugs to treat rare diseases.
Under the Orphan Grants Program, FDA has provided more than $900,000 to researchers at the University of California in San Diego, the National Institutes of Health, and the University of Michigan to study the effects of the drug in clinical trials.
Cystagon is manufactured by Mylan Pharmaceuticals Inc., Morgantown, W. Va.
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