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Ravicti is used to treat urea cycle disorders (UCD), genetic diseases that prevent the body from getting rid of ammonia. The resulting buildup of the toxic substance can lead to brain damage and death, so forgoing treatment is not an option. Horizon Pharma (NASDAQ:HZNP) sells Ravicti at an annual cost per patient of nearly $794,000.
There are an estimated 2,000 individuals in the U.S. who suffer from urea cycle disorders that could potentially be treated by Ravicti. Horizon's drug treats seven types of UCDs, though it's unknown whether Ravicti is safe and effective for treating an especially rare type known as N-acetylglutamate synthetase (NAGS) deficiency.
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