Details
| Stereochemistry | ACHIRAL |
| Molecular Formula | C10H11O2.Na |
| Molecular Weight | 186.1829 |
| Optical Activity | NONE |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Charge | 0 |
SHOW SMILES / InChI
SMILES
[Na+].[O-]C(=O)CCCC1=CC=CC=C1
InChI
InChIKey=VPZRWNZGLKXFOE-UHFFFAOYSA-M
InChI=1S/C10H12O2.Na/c11-10(12)8-4-7-9-5-2-1-3-6-9;/h1-3,5-6H,4,7-8H2,(H,11,12);/q;+1/p-1
| Molecular Formula | Na |
| Molecular Weight | 22.98976928 |
| Charge | 1 |
| Count |
|
| Stereochemistry | ACHIRAL |
| Additional Stereochemistry | No |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Optical Activity | NONE |
| Molecular Formula | C10H11O2 |
| Molecular Weight | 163.1931 |
| Charge | -1 |
| Count |
|
| Stereochemistry | ACHIRAL |
| Additional Stereochemistry | No |
| Defined Stereocenters | 0 / 0 |
| E/Z Centers | 0 |
| Optical Activity | NONE |
Sodium phenylbutyrate is a salt of an aromatic fatty acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. It is used as adjunctive therapy for the management of chronic urea cycle disorders due to deficiencies in carbamylphosphate (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase. It is indicated in all neonatal- onset efficiency presenting within the first 28 days of life. Also indicated in patients with late-onset, presenting after the first month of life with a history of hyperammonemic encephalopathy. Sodium phenylbutyrate is a pro-drug and is rapidly metabolized to phenylacetate. Phenylacetate is a metabolically active compound that conjugates with glutamine via acetylation to form phenylacetylglutamine. The kidneys then excrete Phenylacetylglutamine. PBA (phenylbutyric acid) is absorbed from the intestine and converted by way of β-oxidation to the active moiety, phenylacetic acid (PAA). PAA is conjugated with glutamine in the liver and kidney by way of N-acyl coenzyme A-l-glutamine N-acyltransferase to form phenylacetylglutamine (PAGN). Like urea, PAGN incorporates two waste nitrogens and is excreted in the urine. On a molar basis, it is comparable to urea (each containing two moles of nitrogen). Therefore, phenylacetylglutamine provides an alternate vehicle for waste nitrogen excretion.
CNS Activity
Sources: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Scientific_Discussion/human/000219/WC500024748.pdf
Curator's Comment: It was argued that high concentrations are unlikely during therapeutic use of sodium phenylbutyrate (PB) because of poor transfer across the adult blood-brain barrier. The implications of these findings with respect to human foetal brain are unknown.
Approval Year
Targets
| Primary Target | Pharmacology | Condition | Potency |
|---|---|---|---|
Target ID: GO:0007050 Sources: https://www.ncbi.nlm.nih.gov/pubmed/19024104 |
|||
| 6.34 µM [Ki] | |||
Target ID: CHEMBL2366039 Sources: https://www.ncbi.nlm.nih.gov/pubmed/20512995 |
|||
Target ID: Nitrogen |
Conditions
| Condition | Modality | Targets | Highest Phase | Product |
|---|---|---|---|---|
| Palliative | BUPHENYL Approved UseBUPHENYL® is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy. It is important that the diagnosis be made early and treatment initiated immediately to improve survival. Any episode of acute hyperammonemia should be treated as a lifethreatening emergency. Launch Date1996 |
|||
| Palliative | BUPHENYL Approved UseBUPHENYL® is indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). It is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy. It is important that the diagnosis be made early and treatment initiated immediately to improve survival. Any episode of acute hyperammonemia should be treated as a lifethreatening emergency. Launch Date1996 |
Cmax
| Value | Dose | Co-administered | Analyte | Population |
|---|---|---|---|---|
457.9 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
9 g 1 times / day multiple, oral dose: 9 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
949.9 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
18 g 1 times / day multiple, oral dose: 18 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
1679.8 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
27 g 1 times / day multiple, oral dose: 27 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
1878.3 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
36 g 1 times / day multiple, oral dose: 36 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
1669.4 μM EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
45 g 1 times / day multiple, oral dose: 45 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
218 μg/mL |
5 mg single, oral dose: 5 mg route of administration: Oral experiment type: SINGLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: HEALTHY age: ADULT sex: UNKNOWN food status: FASTED |
AUC
| Value | Dose | Co-administered | Analyte | Population |
|---|---|---|---|---|
925.5 μM × h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
9 g 1 times / day multiple, oral dose: 9 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
2170.6 μM × h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
18 g 1 times / day multiple, oral dose: 18 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
3778.6 μM × h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
27 g 1 times / day multiple, oral dose: 27 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
4687.4 μM × h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
36 g 1 times / day multiple, oral dose: 36 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
4941.4 μM × h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
45 g 1 times / day multiple, oral dose: 45 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
T1/2
| Value | Dose | Co-administered | Analyte | Population |
|---|---|---|---|---|
0.76 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
9 g 1 times / day multiple, oral dose: 9 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
0.61 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
18 g 1 times / day multiple, oral dose: 18 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
0.58 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
27 g 1 times / day multiple, oral dose: 27 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
0.94 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
36 g 1 times / day multiple, oral dose: 36 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
1.16 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/11489804 |
45 g 1 times / day multiple, oral dose: 45 g route of administration: Oral experiment type: MULTIPLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: FED |
|
0.77 h |
5 mg single, oral dose: 5 mg route of administration: Oral experiment type: SINGLE co-administered: |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: HEALTHY age: ADULT sex: UNKNOWN food status: FASTED |
Funbound
| Value | Dose | Co-administered | Analyte | Population |
|---|---|---|---|---|
0.3% EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/8946671 |
unknown, unknown |
SODIUM PHENYLBUTYRATE plasma | Homo sapiens population: UNKNOWN age: UNKNOWN sex: UNKNOWN food status: UNKNOWN |
Doses
| Dose | Population | Adverse events |
|---|---|---|
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
DLT: Nausea, Vomiting... Dose limiting toxicities: Nausea (grade 3, 2 patients) Sources: Vomiting (grade 3, 1 patient) Gait instability (grade 3, 2 patients) Lightheadedness (grade 3, 2 patients) Confusion (grade 3, 1 patient) |
15 g 1 times / day steady, oral MTD Dose: 15 g, 1 times / day Route: oral Route: steady Dose: 15 g, 1 times / day Sources: |
unhealthy, 38-61 years Health Status: unhealthy Age Group: 38-61 years Sex: M+F Sources: |
Other AEs: Nausea, Gait instability... Other AEs: Nausea (grade 1, 2 patients) Sources: Gait instability (grade 1, 1 patient) Syncope vasovagal (grade 2, 2 patients) Diarrhea (grade 1, 1 patient) |
27 g 1 times / day steady, oral MTD Dose: 27 g, 1 times / day Route: oral Route: steady Dose: 27 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
DLT: Fatigue... Dose limiting toxicities: Fatigue (grade 3, 1 patient) Sources: |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
DLT: Fatigue, Somnolence... Dose limiting toxicities: Fatigue (grade 3, 2 patients) Sources: Somnolence (grade 3, 2 patients) Anemia (grade 3, 1 patient) Neutropenia (grade 3, 1 patient) |
45 g single, intravenous Highest studied dose Dose: 45 g Route: intravenous Route: single Dose: 45 g Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
|
45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
DLT: Fatigue, Central nervous system toxicity... Other AEs: Nausea, Vomiting... Dose limiting toxicities: Fatigue (grade 3-4, 1 patient) Other AEs:Central nervous system toxicity (grade 3-4, 1 patient) Nausea (grade 3-4, 1 patient) Sources: Vomiting (grade 3-4, 1 patient) Edema (grade 3-4, 1 patient) |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
DLT: Nausea, Vomiting... Dose limiting toxicities: Nausea (grade 3-4, 1 patient) Sources: Vomiting (grade 3-4, 1 patient) Hypocalcemia (grade 3-4, 1 patient) |
150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
DLT: Fatigue... Other AEs: Anemia, Light-headed... Dose limiting toxicities: Fatigue (grade 2, 2 patients) Other AEs:Anemia (grade 3, 1 patient) Sources: Light-headed (grade 2, 2 patients) Edema (grade 2, 2 patients) Nausea (grade 2, 1 patient) Vomiting (grade 2, 1 patient) Constipation (grade 1, 2 patients) Fever (grade 1, 1 patient) |
AEs
| AE | Significance | Dose | Population |
|---|---|---|---|
| Confusion | grade 3, 1 patient DLT |
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
| Vomiting | grade 3, 1 patient DLT |
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
| Gait instability | grade 3, 2 patients DLT |
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
| Lightheadedness | grade 3, 2 patients DLT |
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
| Nausea | grade 3, 2 patients DLT |
18 g 1 times / day steady, oral Dose: 18 g, 1 times / day Route: oral Route: steady Dose: 18 g, 1 times / day Sources: |
unhealthy, 31-73 years Health Status: unhealthy Age Group: 31-73 years Sex: M+F Sources: |
| Diarrhea | grade 1, 1 patient | 15 g 1 times / day steady, oral MTD Dose: 15 g, 1 times / day Route: oral Route: steady Dose: 15 g, 1 times / day Sources: |
unhealthy, 38-61 years Health Status: unhealthy Age Group: 38-61 years Sex: M+F Sources: |
| Gait instability | grade 1, 1 patient | 15 g 1 times / day steady, oral MTD Dose: 15 g, 1 times / day Route: oral Route: steady Dose: 15 g, 1 times / day Sources: |
unhealthy, 38-61 years Health Status: unhealthy Age Group: 38-61 years Sex: M+F Sources: |
| Nausea | grade 1, 2 patients | 15 g 1 times / day steady, oral MTD Dose: 15 g, 1 times / day Route: oral Route: steady Dose: 15 g, 1 times / day Sources: |
unhealthy, 38-61 years Health Status: unhealthy Age Group: 38-61 years Sex: M+F Sources: |
| Syncope vasovagal | grade 2, 2 patients | 15 g 1 times / day steady, oral MTD Dose: 15 g, 1 times / day Route: oral Route: steady Dose: 15 g, 1 times / day Sources: |
unhealthy, 38-61 years Health Status: unhealthy Age Group: 38-61 years Sex: M+F Sources: |
| Fatigue | grade 3, 1 patient DLT |
27 g 1 times / day steady, oral MTD Dose: 27 g, 1 times / day Route: oral Route: steady Dose: 27 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
| Anemia | grade 3, 1 patient DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
| Neutropenia | grade 3, 1 patient DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
| Fatigue | grade 3, 2 patients DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
| Somnolence | grade 3, 2 patients DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 51.4 years (range: 29.7–71.1 years) Health Status: unhealthy Age Group: 51.4 years (range: 29.7–71.1 years) Sex: M+F Sources: |
| Edema | grade 3-4, 1 patient | 45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Nausea | grade 3-4, 1 patient | 45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Vomiting | grade 3-4, 1 patient | 45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Central nervous system toxicity | grade 3-4, 1 patient DLT, Disc. AE |
45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Fatigue | grade 3-4, 1 patient DLT, Disc. AE |
45 g 1 times / day steady, oral Highest studied dose Dose: 45 g, 1 times / day Route: oral Route: steady Dose: 45 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Hypocalcemia | grade 3-4, 1 patient DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Nausea | grade 3-4, 1 patient DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Vomiting | grade 3-4, 1 patient DLT |
36 g 1 times / day steady, oral Dose: 36 g, 1 times / day Route: oral Route: steady Dose: 36 g, 1 times / day Sources: |
unhealthy, 57 years (range: 26–75 years) Health Status: unhealthy Age Group: 57 years (range: 26–75 years) Sex: M+F Sources: |
| Fever | grade 1, 1 patient | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Constipation | grade 1, 2 patients | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Nausea | grade 2, 1 patient | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Vomiting | grade 2, 1 patient | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Edema | grade 2, 2 patients | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Light-headed | grade 2, 2 patients | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Fatigue | grade 2, 2 patients DLT, Disc. AE |
150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
| Anemia | grade 3, 1 patient | 150 mg/kg 3 times / day steady, oral MTD Dose: 150 mg/kg, 3 times / day Route: oral Route: steady Dose: 150 mg/kg, 3 times / day Sources: |
unhealthy, 57 years(range: 25–71 years) Health Status: unhealthy Age Group: 57 years(range: 25–71 years) Sex: M+F Sources: |
PubMed
| Title | Date | PubMed |
|---|---|---|
| Safety and efficacy of glycerol phenylbutyrate for management of urea cycle disorders in patients aged 2months to 2years. | 2017-11 |
|
| Glycerol phenylbutyrate (Ravicti) for urea cycle disorders. | 2014-08-18 |
|
| Sodium phenylbutyrate reverses lysosomal dysfunction and decreases amyloid-β42 in an in vitro-model of inclusion-body myositis. | 2014-05 |
|
| Ammonia control and neurocognitive outcome among urea cycle disorder patients treated with glycerol phenylbutyrate. | 2013-06 |
|
| Developing a new formulation of sodium phenylbutyrate. | 2012-12 |
|
| Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis. | 2010-06 |
Patents
Sample Use Guides
Tablets is indicated for children weighing more than 20 kg and for adults. The usual total daily dose of Tablets and Powder for patients with urea cycle disorders is 450–600 mg/kg/day in patients weighing less than 20 kg, or 9.9–13.0 g/m2/day in larger patients. The tablets and powder are to be taken in equally divided amounts with each meal or feeding (i.e., three to six times per day). Powder is indicated for oral use (via mouth, gastrostomy, or nasogastric tube) only.
Route of Administration:
Oral
In Vitro Use Guide
Sources: https://www.ncbi.nlm.nih.gov/pubmed/24472798
Curator's Comment: Sodium phenylbutyrate (NaPB) treatment reverses lysosomal dysfunction in an in vitro model of inclusion-body myositis, involving cultured human muscle fibers. NaPB treatment improved lysosomal activity, decreased Aβ42 and its oligomers, decreased γ-secretase activity, and virtually prevented muscle-fiber vacuolization
Unknown
| Substance Class |
Chemical
Created
by
admin
on
Edited
Mon Mar 31 18:12:32 GMT 2025
by
admin
on
Mon Mar 31 18:12:32 GMT 2025
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| Record UNII |
NT6K61736T
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| Record Status |
Validated (UNII)
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| Record Version |
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Brand Name | English | ||
|
Brand Name | English |
| Classification Tree | Code System | Code | ||
|---|---|---|---|---|
|
FDA ORPHAN DRUG |
110598
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
EMA ASSESSMENT REPORTS |
AMMONAPS (AUTHORIZED: ORNITHINE CARBAMOYLTRANSFERASE DEFICIENCY DISEASE)
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
235806
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
68192
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
EU-Orphan Drug |
EU/3/11/948
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
588117
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
EMA ASSESSMENT REPORTS |
PHEBURANE (AUTHORIZED: CARBAMOYL-PHOSPHATE SYNTHASE I DEFICIENCY DISEASE)
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
NCI_THESAURUS |
C492
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
767720
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
77893
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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||
|
NCI_THESAURUS |
C1946
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
FDA ORPHAN DRUG |
293309
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
FDA ORPHAN DRUG |
372112
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
FDA ORPHAN DRUG |
439714
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
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|
EMA ASSESSMENT REPORTS |
AMMONAPS (AUTHORIZED: CITRULLINEMIA)
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
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|
WHO-VATC |
QA16AX03
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
FDA ORPHAN DRUG |
236907
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
FDA ORPHAN DRUG |
354011
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
EMA ASSESSMENT REPORTS |
AMMONAPS (AUTHORIZED: CARBAMOYL-PHOSPHATE SYNTHASE I DEFICIENCY DISEASE
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
||
|
WHO-ATC |
A16AX03
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
| Code System | Code | Type | Description | ||
|---|---|---|---|---|---|
|
5258
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
m11917
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
75316
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
657802
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
1614512
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
NT6K61736T
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
1716-12-7
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
CHEMBL1469
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
100000091834
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
NT6K61736T
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
SODIUM PHENYLBUTYRATE
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
DTXSID7040948
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
HH-22
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
214837
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | RxNorm | ||
|
DB06819
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
C1440
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY | |||
|
SUB12586MIG
Created by
admin on Mon Mar 31 18:12:32 GMT 2025 , Edited by admin on Mon Mar 31 18:12:32 GMT 2025
|
PRIMARY |
| Related Record | Type | Details | ||
|---|---|---|---|---|
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PARENT -> SALT/SOLVATE |
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| Related Record | Type | Details | ||
|---|---|---|---|---|
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IMPURITY -> PARENT |
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| Related Record | Type | Details | ||
|---|---|---|---|---|
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ACTIVE MOIETY |
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