Table of contents

  • Brand Names
  • Chemistry
  • Pharmacologic Category
  • Mechanism of Action
  • Therapeutic Use
  • Pregnancy and Lactation Implications
  • Contraindications
  • Warnings and Precautions
  • Adverse Reactions
  • Caution and personalized dose adjustment in patients with the following genotypes
  • Drug Interactions
  • Dosage
  • Pharmacokinetics and Pharmacodynamics
  • Special Considerations

Brand Names


Austria: Kuvan; Czech Republic: Kuvan, Tetrahydrobiopterin; Bulgaria: Kuvan; Denmark: Kuvan; Estonia: Kuvan; France: Kuvan; Germany: Kuvan; Greece: Kuvan; Hungary: Kuvan; Ireland: Kuvan; Italy: Kuvan; Latvia: Kuvan; Lithuania: Kuvan; Luxembourg: Kuvan; Malta: Kuvan; Netherlands: Kuvan; Poland: Kuvan; Portugal: Kuvan; Romania: Kuvan; Slovakia: Kuvan; Slovenia: Kuvan; Spain: Kuvan; Sweden: Kuvan; UK: Kuvan.

North America

USA: Kuvan.


Japan: Biopten.

Drug combinations


Sapropterin Dihydrochloride: C~9~H~15~N~5~O~3~ 2HCl. Mw: 314.17. (1) 4(1H)-Pteridinone, 2-amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-, dihydrochloride, (6R)-; (2)(6R)-2-Amino-6-[(1R,2S)-1,2-dihydroxypropyl]-5,6,7,8-tetrahydro-4(1H)-pteridinone dihydrochloride. CAS-69056-38-8; CAS-62989-33-7 (sapropterin)(2005).

Pharmacologic Category

Other Miscellaneous Therapeutic Agents. Enzyme Cofactor. (ATC-Code: A16AX07).

Mechanism of action

A synthetic form of cofactor BH~4~ (tetrahydrobiopterin) for enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates phenylalanine to form tyrosine. BH~4~ activates residual PAH enzyme, improving normal phenylalanine metabolism and decreasing phenylalanine levels in sapropterin responders.

Therapeutic use

Adjunct to dietary management in treatment of BH~4~ responsive phenylketonuria (PKU).

Pregnancy and lactiation implications

Teratogenic effects not observed in animals. There are no adequate studies in pregnant women. Excretion in breast milk unknown (not recommended in nursing women).

Unlabeled use


None reported to date.

Warnings and precautions

Do not administer to patients with allergy to any component of the formulation. Patients with mild-to-moderate allergy (e.g. rash) should be evaluated for risk vs benefit prior to continuing therapy. Not studied in hepatic impairment (hepatic damage associated with impaired phenylalanine metabolism). Low levels of phenylalanine associated with catabolism and protein breakdown. Not studied in renal impairment (use with caution). Folic acid antagonists may decrease BH~4~ levels via dihydropteridine reductase enzyme inhibition. In sapropterin clinical studies for a non-PKU use, seizure, over-stimulation, and irritability reported in patients also receiving levodopa (use caution). Use with caution with medications that affect nitric oxide-mediated vasorelaxation, including PDE-5 inhibitors (risk of hypotension). Response to sapropterin treatment established through treatment (cannot be predetermined by lab testing). Patients whose phenylalanine levels do not decrease after treatment at 20 mg/kg/day for 1 month are considered nonresponders.



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