Ravicti Approved for Urea Cycle Disorders
FRIDAY, Feb. 1 -- Ravicti (glycerol phenylbutyrate) has been approved by the U.S. Food and Drug Administration to treat certain urea cycle disorders (UCDs) in people 2 years and older.
These inherited conditions involve a lack of enzymes that help the body remove ammonia from the blood. In people with UCDs, ammonia buildup can lead to coma, brain damage or death, the FDA said Friday in a news release.
Ravicti is a liquid taken three times daily with meals. It's been approved for people whose disorders cannot be managed by a protein-restricted diet or amino acid supplements alone, the agency said.
Ravicti was compared to another UCD drug in a clinical study involving 44 adults. The most common side effects of the new drug were diarrhea, flatulence and headache.
Ravicti is marketed by Hyperion Therapeutics, based in South San Francisco, Calif.
The U.S. National Library of Medicine has more about UCDs.
Posted: February 2013