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Sapropterin dihydrochloride

Tetrahydrobiopterin
Tetrahydrobiopterin structure.png
Clinical data
License data
Pregnancy
category
  • US: C (Risk not ruled out)
Routes of
administration
By mouth
ATC code
Legal status
Legal status
Pharmacokinetic data
Biological half-life 4 hours (healthy adults)
6–7 hours (PKU patients)
Identifiers
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
ChEBI
ChEMBL
Chemical and physical data
Formula C9H15N5O3
Molar mass 241.25 g/mol
3D model (Jmol)
 NYesY (what is this?)  

Tetrahydrobiopterin (BH4, THB), also known as sapropterin, is a naturally occurring essential cofactor of the three aromatic amino acid hydroxylase enzymes, used in the degradation of amino acid phenylalanine and in the biosynthesis of the neurotransmitters serotonin (5-hydroxytryptamine, 5-HT), melatonin, dopamine, norepinephrine (noradrenaline), epinephrine (adrenaline), and is a cofactor for the production of nitric oxide (NO) by the nitric oxide synthases. Chemically, its structure is that of a reduced pteridine derivative.

Sapropterin dihydrochloride may help lower phenylalanine levels in some people with phenylketonuria. It is FDA approved for this use along with dietary measures. Most people however, have little or no benefit.

The most common adverse effects, observed in more than 10% of patients, include headache and a running or obstructed nose. Diarrhea and vomiting are also relatively common, seen in at least 1% of patients.

No interaction studies have been conducted. Because of its mechanism, tetrahydrobiopterin might interact with dihydrofolate reductase inhibitors like methotrexate and trimethoprim, and NO-enhancing drugs like nitroglycerin, molsidomine, minoxidil, and PDE5 inhibitors. Combination of tetrahydrobiopterin with levodopa can lead to increased excitability.


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