Sodium phenylbutyrate

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Sodium phenylbutyrate
Systematic (IUPAC) name
Sodium 4-phenylbutanoate
Identifiers
CAS number 1716-12-7
ATC code A16AX03
PubChem 15585
Chemical data
Formula C10H11NaO2 
Mol. mass 186.2 g/mol
Pharmacokinetic data
Bioavailability  ?
Metabolism to phenylacetate
Half life 0.8 hours
Excretion 80% as phenylacetylglutamine
Therapeutic considerations
Licence data

EU, US
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 Swedish Orphan International (Sweden) as Ammonaps.

It has been used to treat urea cycle disorders.[1]

Scanadinavian Formulas, Inc. Sellersville, PA supplies Sodium Phenylbutyrate worldwide for clinical trials.

[edit] Metabolism

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.

Nitrogen elimination
Nitrogen elimination

[edit] Research

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.

PBA has been associated with longer lifespans in Drosophila.[2]

[edit] References

  1. ^ Batshaw ML, MacArthur RB, Tuchman M (2001). "Alternative pathway therapy for urea cycle disorders: twenty years later". J. Pediatr. 138 (1 Suppl): S46–54; discussion S54–5. PMID 11148549. 
  2. ^ Kang HL, Benzer S, Min KT (2002). "Life extension in Drosophila by feeding a drug". Proc. Natl. Acad. Sci. U.S.A. 99 (2): 838–43. doi:10.1073/pnas.022631999. PMID 11792861. 
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