Drug: Kuvan (sapropterin dihydrochloride)
Approval Date: December 18, 2007
Scoop: Phenylketonuria (PKU) is a rare genetic disorder that causes mental retardation, smaller brain size, delayed speech and other neurological problems. This disease is a result of the enzyme phenylalanine hydroxylase (PAH) not functioning properly in the body. Kuvan works by increasing PAH enzyme activity in PKU patients with some residual PAH enzyme function. This then leads to an increased breakdown (metabolism) of phenylalanine (Phe), resulting in lower levels of Phe in the blood.