glycerol phenylbutyrate

Glycerol phenylbutyrate is a nitrogen-binding agent used for the chronic management of urea cycle disorders (UCDs) in adults and pediatric patients aged 2 years and older who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. It is a prodrug that, after oral administration, is hydrolyzed in the gastrointestinal tract to release phenylbutyrate (PBA), which undergoes beta-oxidation to form phenylacetate (PAA). PAA conjugates with glutamine in the liver and kidneys to form phenylacetylglutamine (PAGN), which is excreted by the kidneys. This process provides an alternative pathway for waste nitrogen excretion, thereby reducing toxic ammonia accumulation associated with UCDs. Glycerol phenylbutyrate does not treat acute hyperammonemia but helps prevent its occurrence as part of long-term management[1][4][8].

Brand names

Ravicti

Other names

glycerol phenylbutyrate