Sodium phenylbutyrate
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Sodium phenylbutyrate
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Systematic (IUPAC) name | |
Sodium 4-phenylbutanoate | |
Identifiers | |
CAS number | 1716-12-7 |
ATC code | A16AX03 |
PubChem | 15585 |
Chemical data | |
Formula | C10H11O2Na |
Mol. weight | 186.2 g/mol |
Pharmacokinetic data | |
Bioavailability | ? |
Metabolism | to phenylacetate |
Half life | 0.8 hours |
Excretion | 80% as phenylacetylglutamine |
Therapeutic considerations | |
Licence data | |
Pregnancy cat. |
not to be used |
Legal status | |
Routes | ? |
Sodium phenylbutyrate is an orphan drug, marketed by Ucyclyd Pharma (Hunt Valley, USA) under the trade name Buphenyl® and by Orphan Europe (France) as Ammonaps®. Phenylbutyrate is a prodrug. In the human body it is metabolized by beta-oxidation to phenylacetate. Phenylacetate conjugate with glutamine to phenylacetylglutamine, that is eliminated with the urine.
Sodium phenylbutyrate is also under investigation for the treatment of some sickle-cell disorders (Blood Products Plasma Expanders and Haemostatics) and for use as a potential differentiation-inducing agent in malignant glioma and acute myeloid leukaemia.
[edit] References
1. Batshaw ML, et al. Alternative pathway therapy for urea cycle disorders: twenty years later. J Pediatr 2001; 138 (suppl 1): S46-S55. (PubMed id:11148549) Correction. ibid. 2002; 140: 490.
2. Kang HL, Benzer S, Min KT.; Life extension in Drosophila by feeding a drug.; Proc Natl Acad Sci U S A. 2002 Jan 22; 99(2):838-43. Epub 2002 Jan 15.; PMID: 11792861