Kuvan - 2007 FDA approvals

Drug: Kuvan (sapropterin dihydrochloride)
Indication: Phenylketonuria
Company: BioMarin
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.

Kuvan - 2007 FDA approvals

Suggested Articles

Karen DeSalvo's appointment is the latest in a string of hires that deepen the tech giant’s investment in and focus on healthcare.

Spring Bank’s hepatitis B drug, given in tandem with Gilead’s Vemlidy, tamped down on chronic disease in 23% of patients in a phase 2 study.

Verseau will use the money to start testing its macrophage checkpoint modulators in humans.