On February 1, 2013, the U.S. Food and Drug Administration approved Ravicti (glycerol phenylbutyrate) for the chronic management of some urea cycle disorders (UCDs) in patients ages 2 years and older.
Urea cycle disorders involve deficiencies of specific enzymes involved in the urea cycle, a series of biochemical steps normally required to remove ammonia from the blood. In people with UCDs, the excess ammonia can travel to the brain and cause brain damage, coma or death.
Ravicti, a liquid taken three times a day with meals, helps dispose of ammonia in the body. It is intended for patients whose UCD cannot be managed by a protein-restricted diet or amino acid supplements alone. Ravicti must be used with a protein-restricted diet and, in some cases, dietary supplements.
The Urea Cycle Disorders Consortium (UCDC) and the National Urea Cycle Disorders Foundation were key leaders and research partners in this study, which was sponsored by Hyperion for Ravicti (glycerol phenylbutyrate).
