Sapropterin, also known as Kuvan, is a synthetic form of the naturally occurring compound tetrahydrobiopterin (BH4). It is primarily used to treat a specific type of metabolic disorder known as Phenylketonuria (PKU) in both adults and children, including neonates. Sapropterin works by enhancing the activity of the enzyme phenylalanine hydroxylase (PAH), which helps in the breakdown of phenylalanine, an amino acid that can cause severe neurological damage when accumulated in high levels in the blood.
