Synonyms: 4-phenylbutanoic acid | Ammonaps® | Buphenyl® | gamma-phenylbutyric acid | triButyrate
phenylbutyrate is an approved drug (FDA (1996), EMA (1999))
Compound class:
Synthetic organic
Comment: The salt, sodium phenylbutyrate (PubChem CID 5258) is an inhibitor of histone deacetylase (HDAC) activity [2-3].
Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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No information available. |
Mechanism Of Action and Pharmacodynamic Effects |
In patients with liver enzyme deficiency hyperammonemia can develop due to impaired waste nitrogen removal. Sodium phenylbutyrate is converted to phenylacetate (active metabolite) which acts as an ammonia sink and chemical chaperone and prevents waste nitrogen from entering the urea cycle, thus preventing hyperammonemia. Sodium phenylbutyrate also acts via HDACs as a transcriptional regulator to alter chromatin structure [2]. This latter action is reported to provide neuroprotective effects [1], which may help reduce the progressive brain damage which is a major pathological symptom of metabolic disorders such as maple syrup urine disease, proteinuric diseases and other urea cycle disorders. In 5q spinal muscular atrophy, sodium phenylbutyrate increases transcription of the SMN2 (survival of motor neuron 2) gene via inhibition of histone deacetylase activity thus effecting elevated levels of functional SMN2 protein [2]. |