.Pharmacolibrary.Drugs.ATC.A.A16AX07

Information

Sapropterin dihydrochloride is a synthetic form of tetrahydrobiopterin (BH4), a natural cofactor for the enzyme phenylalanine hydroxylase. It is used as an adjunct treatment for phenylketonuria (PKU) in both pediatric and adult patients who have been shown to be responsive to this therapy. Sapropterin is an FDA- and EMA-approved medication.

Pharmacokinetics

Pharmacokinetic parameters following a single oral dose (10 mg/kg) in healthy adult volunteers.

References

1. Muntau, AC, et al., & Rogoff, D (2017). Efficacy, safety and population pharmacokinetics of sapropterin in PKU patients <4 years: results from the SPARK open-label, multicentre, randomized phase IIIb trial. Orphanet journal of rare diseases 12(1) 47–None. DOI:10.1186/s13023-017-0600-x PUBMED:https://pubmed.ncbi.nlm.nih.gov/28274234

2. Feillet, F, et al., & Foehr, E (2008). Pharmacokinetics of sapropterin in patients with phenylketonuria. Clinical pharmacokinetics 47(12) 817–825. DOI:10.2165/0003088-200847120-00006 PUBMED:https://pubmed.ncbi.nlm.nih.gov/19026037

3. Sanford, M, & Keating, GM (2009). Sapropterin: a review of its use in the treatment of primary hyperphenylalaninaemia. Drugs 69(4) 461–476. DOI:10.2165/00003495-200969040-00006 PUBMED:https://pubmed.ncbi.nlm.nih.gov/19323589

Revisions

• 06/2025 initial generated model