The Medication(s) listed below have been approved by the Food & Drug Administration (FDA) as orphan products for treatment of this condition. There may be other products available
that are not considered orphan products.
Sapropterin (Brand name: Kuvan)
- Manufactured by
Biomarin Pharmaceutical Inc.
FDA-approved indication: December 2014 approved to reduce blood phenylalanine (Phe) levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). Kuvan is to be used in conjunction with a Phe-restricted diet.
National Library of Medicine Drug Information PortalMedline Plus Health Information
Pegvaliase (Brand name: Palynziq)
- Manufactured by
BioMarin Pharmaceutical
FDA-approved indication: May 2018 approved to reduce blood phenylalanine concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
National Library of Medicine Drug Information Portal