| Systematic (IUPAC) name | |
|---|---|
| Sodium 4-phenylbutanoate | |
| Clinical data | |
| AHFS/Drugs.com | Consumer Drug Information |
| Licence data | EMA:Link, US FDA:link |
| Pregnancy cat. | not to be used |
| Legal status | ? |
| Pharmacokinetic data | |
| Metabolism | to phenylacetic acid |
| Half-life | 0.8 hours |
| Excretion | 80% as phenylacetylglutamine |
| Identifiers | |
| CAS number | 1716-12-7 |
| ATC code | A16AX03 |
| PubChem | CID 9815454 |
| ChemSpider | 5068 |
| ChEMBL | CHEMBL1746 |
| Chemical data | |
| Formula | C10H11NaO2 |
| Mol. mass | 186.2 g/mol |
| SMILES | eMolecules & PubChem |
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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]
On July 5, 2011, Colorado researchers, Dr. Curt Freed and Wenbo Zhou, have discovered a drug that stops the progression of Parkinson's disease in mice and is now being tested on humans. They have found that the drug phenylbutyrate turns on a gene, called DJ-1, that can protect dopamine neurons in Parkinson's disease. Parkinson's disease is caused by dying midbrain dopamine neurons.[2]
Phenylbutyrate is a prodrug. In the human body it is metabolized by beta-oxidation to phenylacetate.
Phenylacetate conjugates 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.
PBA has been associated with longer lifespans in Drosophila.[3]
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