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Sodium phenylbutyrate



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 Orphan Europe (France) as Ammonaps®.

Additional recommended knowledge

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

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

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.


 

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]

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.
 
This article is licensed under the GNU Free Documentation License. It uses material from the Wikipedia article "Sodium_phenylbutyrate". A list of authors is available in Wikipedia.
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