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Details

Stereochemistry ACHIRAL
Molecular Formula C33H38O6
Molecular Weight 530.6512
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of GLYCEROL PHENYLBUTYRATE

SMILES

O=C(CCCC1=CC=CC=C1)OCC(COC(=O)CCCC2=CC=CC=C2)OC(=O)CCCC3=CC=CC=C3

InChI

InChIKey=ZSDBFLMJVAGKOU-UHFFFAOYSA-N
InChI=1S/C33H38O6/c34-31(22-10-19-27-13-4-1-5-14-27)37-25-30(39-33(36)24-12-21-29-17-8-3-9-18-29)26-38-32(35)23-11-20-28-15-6-2-7-16-28/h1-9,13-18,30H,10-12,19-26H2

HIDE SMILES / InChI

Molecular Formula C33H38O6
Molecular Weight 530.6512
Charge 0
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

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

TargetsConditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Palliative
BUPHENYL

Approved Use

BUPHENYL® 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 Date

8.3073597E11
Palliative
BUPHENYL

Approved Use

BUPHENYL® 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 Date

8.3073597E11
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
949.9 μM
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
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
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
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
457.9 μM
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
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

AUC

ValueDoseCo-administeredAnalytePopulation
2170.6 μM × h
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
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
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
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
925.5 μM × h
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
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
0.61 h
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
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
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
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
0.76 h
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
0.77 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

Funbound

ValueDoseCo-administeredAnalytePopulation
0.3%
unknown, unknown
SODIUM PHENYLBUTYRATE plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse 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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
Sources:
DLT: Nausea, Vomiting...
Dose limiting toxicities:
Nausea (grade 3, 2 patients)
Vomiting (grade 3, 1 patient)
Gait instability (grade 3, 2 patients)
Lightheadedness (grade 3, 2 patients)
Confusion (grade 3, 1 patient)
Sources:
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
n = 4
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 38-61 years
Sex: M+F
Population Size: 4
Sources:
Other AEs: Nausea, Gait instability...
Other AEs:
Nausea (grade 1, 2 patients)
Gait instability (grade 1, 1 patient)
Syncope vasovagal (grade 2, 2 patients)
Diarrhea (grade 1, 1 patient)
Sources:
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)
n = 7
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 7
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)
n = 4
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 4
Sources:
DLT: Fatigue, Somnolence...
Dose limiting toxicities:
Fatigue (grade 3, 2 patients)
Somnolence (grade 3, 2 patients)
Anemia (grade 3, 1 patient)
Neutropenia (grade 3, 1 patient)
Sources:
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
Sources:
DLT: Fatigue, Central nervous system toxicity...
Other AEs: Nausea, Vomiting...
Dose limiting toxicities:
Fatigue (grade 3-4, 1 patient)
Central nervous system toxicity (grade 3-4, 1 patient)
Other AEs:
Nausea (grade 3-4, 1 patient)
Vomiting (grade 3-4, 1 patient)
Edema (grade 3-4, 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, 57 years (range: 26–75 years)
n = 7
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 7
Sources:
DLT: Nausea, Vomiting...
Dose limiting toxicities:
Nausea (grade 3-4, 1 patient)
Vomiting (grade 3-4, 1 patient)
Hypocalcemia (grade 3-4, 1 patient)
Sources:
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
Sources:
DLT: Fatigue...
Other AEs: Anemia, Light-headed...
Dose limiting toxicities:
Fatigue (grade 2, 2 patients)
Other AEs:
Anemia (grade 3, 1 patient)
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)
Sources:
AEs

AEs

AESignificanceDosePopulation
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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
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
n = 11
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 31-73 years
Sex: M+F
Population Size: 11
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
n = 4
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 38-61 years
Sex: M+F
Population Size: 4
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
n = 4
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 38-61 years
Sex: M+F
Population Size: 4
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
n = 4
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 38-61 years
Sex: M+F
Population Size: 4
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
n = 4
Health Status: unhealthy
Condition: Huntington’s Disease
Age Group: 38-61 years
Sex: M+F
Population Size: 4
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)
n = 7
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 7
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)
n = 4
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Condition: recurrent malignant gliomas
Age Group: 51.4 years (range: 29.7–71.1 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 4
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 4
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)
n = 7
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 7
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)
n = 7
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 7
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)
n = 7
Health Status: unhealthy
Age Group: 57 years (range: 26–75 years)
Sex: M+F
Population Size: 7
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
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)
n = 3
Health Status: unhealthy
Age Group: 57 years(range: 25–71 years)
Sex: M+F
Population Size: 3
Sources:
Sourcing

Sourcing

Vendor/AggregatorIDURL
PubMed

PubMed

TitleDatePubMed
Pharmacology and safety of glycerol phenylbutyrate in healthy adults and adults with cirrhosis.
2010 Jun
Developing a new formulation of sodium phenylbutyrate.
2012 Dec
Sodium phenylbutyrate reverses lysosomal dysfunction and decreases amyloid-β42 in an in vitro-model of inclusion-body myositis.
2014 May
2017 Apr
Patents

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
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 Sat Dec 16 01:35:18 UTC 2023
Edited
by admin
on Sat Dec 16 01:35:18 UTC 2023
Record UNII
ZH6F1VCV7B
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
GLYCEROL PHENYLBUTYRATE
INN   ORANGE BOOK   USAN   VANDF   WHO-DD  
INN   USAN  
Official Name English
GLYCEROL PHENYLBUTYRATE [VANDF]
Common Name English
GLYCEROL PHENYLBUTYRATE [WHO-DD]
Common Name English
GLYCEROL PHENYLBUTYRATE [USAN]
Common Name English
GLYCEROL PHENYLBUTYRATE [MI]
Common Name English
HPN-100
Code English
TRI(4-PHENYLBUTYRYL)GLYCEROL
Systematic Name English
glycerol phenylbutyrate [INN]
Common Name English
BENZENEBUTANOIC ACID, 1,2,3-PROPANETRIYL ESTER
Common Name English
GLYCEROL PHENYLBUTYRATE [ORANGE BOOK]
Common Name English
GLYCERYL TRI (4-PHENYLBUTYRATE)
Systematic Name English
Propane-1,2,3-triyl tris(4-phenylbutanoate)
Systematic Name English
HPN100
Code English
RAVICTI
Brand Name English
GT4P
Common Name English
BENZENEBUTANOIC ACID, 1,1',1''-(1,2,3-PROPANETRIYL) ESTER
Common Name English
GLYCERYL TRI(4-PHENYLBUTYRATE)
Systematic Name English
GLYCERYL TRI-(4-PHENYLBUTYRATE)
Systematic Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 204205
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
WHO-VATC QA16AX09
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
FDA ORPHAN DRUG 203505
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
EU-Orphan Drug EU/3/10/739
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
WHO-ATC A16AX09
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
EMA ASSESSMENT REPORTS RAVICTI (AUTHORIZED: UREA CYCLE DISORDERS, INBORN)
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
FDA ORPHAN DRUG 269308
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
Code System Code Type Description
MERCK INDEX
m11918
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
PUBCHEM
10482134
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
EVMPD
SUB96043
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
INN
9517
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
EPA CompTox
DTXSID40210005
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
RXCUI
1368451
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY RxNorm
DRUG BANK
DB08909
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
USAN
YY-32
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
CAS
611168-24-2
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
ChEMBL
CHEMBL2105745
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
WIKIPEDIA
Glycerol phenylbutyrate
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
MESH
C570223
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
SMS_ID
100000141661
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
DRUG CENTRAL
4745
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
DAILYMED
ZH6F1VCV7B
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
NCI_THESAURUS
C170031
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
FDA UNII
ZH6F1VCV7B
Created by admin on Sat Dec 16 01:35:18 UTC 2023 , Edited by admin on Sat Dec 16 01:35:18 UTC 2023
PRIMARY
Related Record Type Details
METABOLITE ACTIVE -> PRODRUG
Related Record Type Details
ACTIVE MOIETY