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 Portal Medline Plus Health Information

  Learn more about Phenylketonuria

• 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

  Learn more about Phenylketonuria