Phenylbutanoic acid
Phenylbutanoic acid is a small molecule pharmaceutical. It is currently being investigated in clinical studies.
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New Drug Application (NDA)
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Abbreviated New Drug Application (ANDA)
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FDA
EMA
Brand Name | Status | Last Update |
|---|---|---|
| ravicti | New Drug Application | 2021-09-13 |
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Clinical
Clinical Trials
68 clinical trials
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Indications Phases 4
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Inborn urea cycle disorders | D056806 | — | E72.2 | 1 | 3 | 3 | 3 | 2 | 12 |
| Insulin resistance | D007333 | HP_0000855 | E88.819 | — | — | — | 1 | 1 | 2 |
Indications Phases 3
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Amyotrophic lateral sclerosis | D000690 | HP_0007354 | G12.21 | 1 | 4 | 3 | — | 1 | 7 |
| Neurodegenerative diseases | D019636 | EFO_0005772 | G31.9 | — | 1 | 1 | — | — | 2 |
| Inborn genetic diseases | D030342 | EFO_0000508 | — | — | 1 | 1 | — | — | 1 |
| Intrahepatic cholestasis | D002780 | — | — | — | 1 | 1 | — | — | 1 |
| Maple syrup urine disease | D008375 | — | E71.0 | — | 1 | 1 | — | — | 1 |
| Hemophilia b | D002836 | — | D67 | — | 1 | 1 | — | — | 1 |
| Progressive supranuclear palsy | D013494 | EFO_0002512 | G23.1 | — | — | 1 | — | — | 1 |
Indications Phases 2
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Leukemia | D007938 | — | C95 | 2 | 2 | — | — | — | 4 |
| Lymphoma | D008223 | — | C85.9 | 2 | 2 | — | — | — | 4 |
| Myelodysplastic syndromes | D009190 | — | D46 | 2 | 1 | — | — | — | 3 |
| Cystic fibrosis | D003550 | EFO_0000390 | E84 | 3 | 3 | — | — | — | 3 |
| Pulmonary tuberculosis | D014397 | EFO_1000049 | A15 | — | 2 | — | — | — | 2 |
| Hepatic encephalopathy | D006501 | — | K72.91 | 1 | 1 | — | — | — | 2 |
| Spinal muscular atrophies of childhood | D014897 | Orphanet_83419 | G12.0 | 2 | 2 | — | — | — | 2 |
| Inborn errors amino acid metabolism | D000592 | — | E72.9 | — | 2 | — | — | — | 2 |
| Brain neoplasms | D001932 | EFO_0003833 | C71 | 1 | 1 | — | — | — | 2 |
| Prostatic neoplasms | D011471 | — | C61 | — | 1 | — | — | — | 1 |
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Indications Phases 1
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Neoplasms | D009369 | — | C80 | 2 | — | — | — | — | 2 |
| Myelodysplastic-myeloproliferative diseases | D054437 | — | — | 2 | — | — | — | — | 2 |
| Color vision defects | D003117 | — | H53.5 | 1 | — | — | — | — | 1 |
| Parkinson disease | D010300 | EFO_0002508 | G20 | 1 | — | — | — | — | 1 |
| Healthy volunteers/patients | — | — | — | 1 | — | — | — | — | 1 |
| Inclusion body myositis | D018979 | EFO_0007323 | G72.41 | 1 | — | — | — | — | 1 |
| Neuroblastoma | D009447 | EFO_0000621 | — | 1 | — | — | — | — | 1 |
| Epilepsy | D004827 | EFO_0000474 | G40.9 | 1 | — | — | — | — | 1 |
| Hyperammonemia | D022124 | HP_0001987 | E72.20 | 1 | — | — | — | — | 1 |
Indications Without Phase
Indication | MeSH | Ontology | ICD-10 | Ph 1 | Ph 2 | Ph 3 | Ph 4 | Other | Total |
|---|---|---|---|---|---|---|---|---|---|
| Obesity | D009765 | EFO_0001073 | E66.9 | — | — | — | — | 1 | 1 |
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
| Drug common name | Phenylbutanoic acid |
| INN | — |
| Description | 4-phenylbutyric acid is a monocarboxylic acid the structure of which is that of butyric acid substituted with a phenyl group at C-4. It is a histone deacetylase inhibitor that displays anticancer activity. It inhibits cell proliferation, invasion and migration and induces apoptosis in glioma cells. It also inhibits protein isoprenylation, depletes plasma glutamine, increases production of foetal haemoglobin through transcriptional activation of the gamma-globin gene and affects hPPARgamma activation. It has a role as an EC 3.5.1.98 (histone deacetylase) inhibitor, an antineoplastic agent, an apoptosis inducer and a prodrug. It is functionally related to a butyric acid. It is a conjugate acid of a 4-phenylbutyrate. |
| Classification | Small molecule |
| Drug class | — |
| Image (chem structure or protein) |  |
| Structure (InChI/SMILES or Protein Sequence) | O=C(O)CCCc1ccccc1 |
Identifiers
| PDB | — |
| CAS-ID | 1821-12-1 |
| RxCUI | — |
| ChEMBL ID | CHEMBL1469 |
| ChEBI ID | 41500 |
| PubChem CID | 4775 |
| DrugBank | — |
| UNII ID | — |
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Safety
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1,096 adverse events reported
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