Ravicti Receives Expanded Indication
December 21, 2018 – The U.S. FDA has approved an expanded indication for Ravicti® (glycerol phenylbutyrate) oral liquid, manufactured by Horizon Pharma. The product is now approved to provide chronic management for patients of all ages who have a urea cycle disorder (UCD) that cannot be managed by dietary protein restriction and/or amino acid supplementation alone.
UCDs occur due to genetic mutations that lead to a deficiency in the enzymes responsible for removing nitrogen from the bloodstream. When these enzymes function properly, they remove nitrogen by converting it to urea, which is then expelled from the body through urination. In patients with a UCD, the nitrogen instead remains in the blood as toxic ammonia. This puts patients at risk of brain damage, as the ammonia can travel to the brain through the bloodstream. The onset and severity of UCDs can vary based on the specifics of the genetic mutation and on environmental factors including diet, exercise, exposure to infectious organisms, and the use of certain medications. There is no cure for UCDs, although a liver transplant can have a significant beneficial impact. UCDs are believed to affect one out of every 8,500 births and account for as much as 20% of cases of Sudden Infant Death Syndrome (SIDS).
Ravicti, which was previously indicated for use in patients at least two months of age, helps to manage UCDs by acting as a nitrogen-binding agent. This aids the body in removing toxic ammonia from the bloodstream. The FDA first approved the product 1996. Recommended dosing depends on individual patient needs and treatment history. Ravicti should only be prescribed by physicians experienced in the treatment of UCDs, and should not be used to treat acute hyperammonemia in patients with UCDs. It must be used in conjunction with dietary protein restriction. In some cases, dietary supplements may also be necessary.