FDA approves Ravicti (Hyperion Therapeutics ) for Urea Cycle Disorders

On 31 January the FDA approved Ravicti (glycerol phenylbutyrate) from Hyperion Therapeutics, for the chronic management of some Urea Cycle Disorders in patients ages 2 years and older.

Urea Cycle Disorders, or UCDs, are a collection of inherited metabolic disorders in which affected patients suffer from high levels of systemic ammonia, a potent neurotoxin. Ammonia is produced via normal protein ingestion; the body normally detoxifies it by converting it through a series of enzymatic steps to urea, which is excreted in urine. Throughout their lives, UCD patients may experience recurrent hyperammonemic crises in which ammonia levels rise, and can develop complications ranging from nausea, vomiting and headache to coma and death.