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Details

Stereochemistry ABSOLUTE
Molecular Formula C23H36N2O4
Molecular Weight 404.5429
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of ELIGLUSTAT

SMILES

CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)C2=CC=C3OCCOC3=C2

InChI

InChIKey=FJZZPCZKBUKGGU-AUSIDOKSSA-N
InChI=1S/C23H36N2O4/c1-2-3-4-5-6-9-22(26)24-19(17-25-12-7-8-13-25)23(27)18-10-11-20-21(16-18)29-15-14-28-20/h10-11,16,19,23,27H,2-9,12-15,17H2,1H3,(H,24,26)/t19-,23-/m1/s1

HIDE SMILES / InChI

Molecular Formula C23H36N2O4
Molecular Weight 404.5429
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 2 / 2
E/Z Centers 0
Optical Activity UNSPECIFIED

Description
Curator's Comment: description was created based on several sources, including http://www.ncbi.nlm.nih.gov/pubmed/?term=PMC3715929

Eliglustat, marketed by Genzyme as CERDELGA, is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease who are CYP2D6 extensive metabolizers, intermediate metabolizers, or poor metabolizers (PMs) as detected by an FDA-cleared test.

CNS Activity

Curator's Comment: Eliglustat, does not cross the blood brain barrier due to recognition by the multidrug transporter pgp-1. Thus, there is no significant distribution of the drug into the brain.

Originator

Curator's Comment: In pre-clinical studies, the molecule was developed at the Michigan University. James A. Shayman is an inventor on patents covering the composition of matter, synthesis, and uses of eliglustat tartrate and related compounds. These patents are held by the University of Michigan were licensed to Genzyme/Sanofi Corporation.

Approval Year

Targets

Targets

Primary TargetPharmacologyConditionPotency
Target ID: Q16739
Gene ID: 7357.0
Gene Symbol: UGCG
Target Organism: Homo sapiens (Human)
Conditions

Conditions

ConditionModalityTargetsHighest PhaseProduct
Secondary
CERDELGA

Approved Use

Indicated for the long-term treatment of adult patients with Gaucher disease type 1

Launch Date

2014
Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
158 ng/mL
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
265 ng/mL
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
303 ng/mL
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
4.83 ng/mL
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
130 ng/mL
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
10.5 ng/mL
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
1852 ng/mL
30 mg/kg single, oral
dose: 30 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
13.3 ng/mL
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
10.4 ng/mL
1 mg/kg single, oral
dose: 1 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
267 ng/mL
10 mg/kg single, oral
dose: 10 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
2.7 ng/mL
0.3 mg/kg single, oral
dose: 0.3 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
88.3 ng/mL
300 mg single, oral
dose: 300 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
91.1 ng/mL
5 mg/kg single, oral
dose: 5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
79.1 ng/mL
300 mg single, oral
dose: 300 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: HIGH-FAT
12.1 ng/mL
84 mg 2 times / day multiple, oral
dose: 84 mg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
ELIGLUSTAT TARTRATE plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
1000 ng × h/mL
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
1042 ng × h/mL
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
1776 ng × h/mL
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
21.7 ng × h/mL
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
556 ng × h/mL
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
62.1 ng × h/mL
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
10528 ng × h/mL
30 mg/kg single, oral
dose: 30 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
112 ng × h/mL
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
151 ng × h/mL
1 mg/kg single, oral
dose: 1 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
1562 ng × h/mL
10 mg/kg single, oral
dose: 10 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
28.1 ng × h/mL
0.3 mg/kg single, oral
dose: 0.3 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
623 ng × h/mL
300 mg single, oral
dose: 300 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
692 ng × h/mL
5 mg/kg single, oral
dose: 5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
696 ng × h/mL
300 mg single, oral
dose: 300 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: HIGH-FAT
76.3 ng × h/mL
84 mg 2 times / day multiple, oral
dose: 84 mg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
ELIGLUSTAT TARTRATE plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
5.77 h
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
5.64 h
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
5.44 h
350 mg 2 times / day steady-state, oral
dose: 350 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
4.32 h
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.19 h
200 mg 2 times / day steady-state, oral
dose: 200 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
7.28 h
50 mg 2 times / day steady-state, oral
dose: 50 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: FEMALE
food status: FASTED
6.8 h
30 mg/kg single, oral
dose: 30 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.4 h
2 mg/kg single, oral
dose: 2 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.5 h
1 mg/kg single, oral
dose: 1 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.1 h
10 mg/kg single, oral
dose: 10 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
4.8 h
0.3 mg/kg single, oral
dose: 0.3 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.2 h
5 mg/kg single, oral
dose: 5 mg/kg
route of administration: Oral
experiment type: SINGLE
co-administered:
ELIGLUSTAT TARTRATE blood
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
6.5 h
84 mg 2 times / day multiple, oral
dose: 84 mg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
ELIGLUSTAT TARTRATE plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
Funbound

Funbound

ValueDoseCo-administeredAnalytePopulation
17%
84 mg 2 times / day multiple, oral
dose: 84 mg
route of administration: Oral
experiment type: MULTIPLE
co-administered:
ELIGLUSTAT TARTRATE plasma
Homo sapiens
population: UNKNOWN
age: UNKNOWN
sex: UNKNOWN
food status: UNKNOWN
Doses

Doses

DosePopulationAdverse events​
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
DLT: Bradycardia, Dizziness...
Other AEs: Dizziness, Nausea...
Dose limiting toxicities:
Bradycardia (grade 2, 1.3%)
Dizziness (grade 3, 1.3%)
Dysequilibrium (grade 2, 1.3%)
Hypotension (grade 2, 1.3%)
Vomiting (grade 2, 1.3%)
Other AEs:
Dizziness (grade 2, 1.3%)
Nausea (grade 2, 1.3%)
Sources:
20 mg/kg 1 times / day single, oral
MTD
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 20 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Other AEs: Nausea...
200 mg 2 times / day multiple, oral
MTD
Dose: 200 mg, 2 times / day
Route: oral
Route: multiple
Dose: 200 mg, 2 times / day
Sources:
healthy, mean age 28 years
n = 24
Health Status: healthy
Age Group: mean age 28 years
Sex: M+F
Population Size: 24
Sources:
Other AEs: Nausea...
Other AEs:
Nausea (grade 1-2, 12.5%)
Sources:
350 mg 2 times / day multiple, oral
Studied dose
Dose: 350 mg, 2 times / day
Route: oral
Route: multiple
Dose: 350 mg, 2 times / day
Sources:
healthy, mean age 28 years
n = 24
Health Status: healthy
Age Group: mean age 28 years
Sex: M+F
Population Size: 24
Sources:
Other AEs: Vomiting, Nausea...
Other AEs:
Vomiting (16.7%)
Nausea (grade 1-2, 29.2%)
Sources:
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Other AEs: Atrioventricular block second degree, Atrioventricular block...
Other AEs:
Atrioventricular block second degree (1%)
Atrioventricular block (<1%)
Atrioventricular block first degree (<1%)
Sinoatrial block (<1%)
Supraventricular tachycardia (1%)
Arrhythmia supraventricular (<1%)
Atrial tachycardia (<1%)
Ventricular tachycardia (1%)
Ventricular extrasystoles (<1%)
Tachycardia (<1%)
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Other AEs: Nasopharyngitis, Upper respiratory tract infection...
Other AEs:
Nasopharyngitis (13%)
Upper respiratory tract infection (11%)
Influenza (6%)
Sinusitis (6%)
Infection urinary tract (6%)
Bronchitis (3%)
Gastroenteritis (3%)
Viral infection (3%)
Gastroenteritis viral (2%)
Ear infection (2%)
Oral herpes (2%)
Pharyngitis (2%)
Pneumonia (2%)
Diarrhoea (10%)
Abdominal pain upper (8%)
Nausea (8%)
Dyspepsia (7%)
Abdominal pain (6%)
Constipation (6%)
Gastrooesophageal reflux disease (5%)
Vomiting (4%)
Abdominal distension (3%)
Dysphagia (2%)
Flatulence (2%)
Toothache (2%)
Gastritis (2%)
Dry mouth (2%)
Headache (17%)
Dizziness (10%)
Paraesthesia (2%)
Neuropathy peripheral (2%)
Syncope (2%)
Dysgeusia (2%)
Somnolence (2%)
Arthralgia (14%)
Back pain (9%)
Pain in extremity (8%)
Bone pain (5%)
Myalgia (3%)
Musculoskeletal pain (3%)
Fatigue (7%)
Asthenia (4%)
Oedema peripheral (4%)
Chest pain (3%)
Pyrexia (3%)
Influenza like illness (2%)
Cough (6%)
Epistaxis (4%)
Oropharyngeal pain (4%)
Nasal congestion (2%)
Throat irritation (2%)
Blood creatine phosphokinase increased (5%)
Weight decreased (2%)
ALT increased (2%)
Rash (2%)
Pruritus (2%)
Dermatitis contact (2%)
Dry skin (2%)
Laceration (2%)
Contusion (2%)
Palpitations (5%)
Dysmenorrhoea (2%)
Metrorrhagia (2%)
Splenomegaly (2%)
Anxiety (2%)
Hypertension (2%)
Flushing (2%)
Haematuria (2%)
Hepatomegaly (2%)
Hypersensitivity (2%)
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
AEs

AEs

AESignificanceDosePopulation
Dizziness grade 2, 1.3%
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Nausea grade 2, 1.3%
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Bradycardia grade 2, 1.3%
DLT
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Dysequilibrium grade 2, 1.3%
DLT
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Hypotension grade 2, 1.3%
DLT
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Vomiting grade 2, 1.3%
DLT
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Dizziness grade 3, 1.3%
DLT
30 mg/kg 1 times / day single, oral
Highest studied dose
Dose: 30 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 30 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Nausea
20 mg/kg 1 times / day single, oral
MTD
Dose: 20 mg/kg, 1 times / day
Route: oral
Route: single
Dose: 20 mg/kg, 1 times / day
Sources:
healthy, mean age 26 years
n = 74
Health Status: healthy
Age Group: mean age 26 years
Sex: M
Population Size: 74
Sources:
Nausea grade 1-2, 12.5%
200 mg 2 times / day multiple, oral
MTD
Dose: 200 mg, 2 times / day
Route: oral
Route: multiple
Dose: 200 mg, 2 times / day
Sources:
healthy, mean age 28 years
n = 24
Health Status: healthy
Age Group: mean age 28 years
Sex: M+F
Population Size: 24
Sources:
Vomiting 16.7%
350 mg 2 times / day multiple, oral
Studied dose
Dose: 350 mg, 2 times / day
Route: oral
Route: multiple
Dose: 350 mg, 2 times / day
Sources:
healthy, mean age 28 years
n = 24
Health Status: healthy
Age Group: mean age 28 years
Sex: M+F
Population Size: 24
Sources:
Nausea grade 1-2, 29.2%
350 mg 2 times / day multiple, oral
Studied dose
Dose: 350 mg, 2 times / day
Route: oral
Route: multiple
Dose: 350 mg, 2 times / day
Sources:
healthy, mean age 28 years
n = 24
Health Status: healthy
Age Group: mean age 28 years
Sex: M+F
Population Size: 24
Sources:
Atrioventricular block second degree 1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Supraventricular tachycardia 1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Ventricular tachycardia 1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Arrhythmia supraventricular <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Atrial tachycardia <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Atrioventricular block first degree <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Atrioventricular block <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Sinoatrial block <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Tachycardia <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Ventricular extrasystoles <1%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.135
Diarrhoea 10%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dizziness 10%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Upper respiratory tract infection 11%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Nasopharyngitis 13%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Arthralgia 14%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Headache 17%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
ALT increased 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Anxiety 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Contusion 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dermatitis contact 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dry mouth 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dry skin 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dysgeusia 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dysmenorrhoea 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dysphagia 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Ear infection 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Flatulence 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Flushing 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Gastritis 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Gastroenteritis viral 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Haematuria 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Hepatomegaly 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Hypersensitivity 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Hypertension 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Influenza like illness 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Laceration 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Metrorrhagia 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Nasal congestion 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Neuropathy peripheral 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Oral herpes 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Paraesthesia 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Pharyngitis 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Pneumonia 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Pruritus 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Rash 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Somnolence 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Splenomegaly 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Syncope 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Throat irritation 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Toothache 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Weight decreased 2%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Abdominal distension 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Bronchitis 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Chest pain 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Gastroenteritis 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Musculoskeletal pain 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Myalgia 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Pyrexia 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Viral infection 3%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Asthenia 4%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Epistaxis 4%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Oedema peripheral 4%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Oropharyngeal pain 4%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Vomiting 4%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Blood creatine phosphokinase increased 5%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Bone pain 5%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Gastrooesophageal reflux disease 5%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Palpitations 5%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Abdominal pain 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Constipation 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Cough 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Infection urinary tract 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Influenza 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Sinusitis 6%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Dyspepsia 7%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Fatigue 7%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Abdominal pain upper 8%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Nausea 8%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Pain in extremity 8%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
Back pain 9%
100 mg 2 times / day multiple, oral (mean)
Studied dose
Dose: 100 mg, 2 times / day
Route: oral
Route: multiple
Dose: 100 mg, 2 times / day
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
unhealthy, mean age 37.1 years
n = 393
Health Status: unhealthy
Condition: Gaucher disease type 1
Age Group: mean age 37.1 years
Sex: M+F
Population Size: 393
Sources: Page: nda/2014/205494Orig1s000MedR.pdf - p.140
OverviewDrug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
no
no
no
no
no
no
no
no
no
no
no
no
no
no
no
yes [IC50 22 uM]
yes [Ki 27 uM]
yes [Ki 5.82 uM]
yes (co-administration study)
Comment: Following multiple doses of eliglustat 150 mg BID, systemic exposures (AUC and Cmax) to metoprolol increased compared to metoprolol administration alone. In EMs, AUC and Cmax increased by 132% and 72%
Page: 108.0
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
major
yes (co-administration study)
Comment: Following multiple doses of CERDELGA 127 mg twice daily (1.5 times the recommended dosage), metoprolol (a CYP2D6 substrate) mean Cmax and AUC increased by 1.7-fold and 2.3- fold in CYP2D6 EMs, respectively, and by 1.2-fold and 1.6-fold in IMs, respectively
Page: 11.0
minor
yes (co-administration study)
Comment: As enzyme substrate: In healthy subjects, co-administration with a strong CYP2D6 and a weak CYP3A inhibitor paroxetine and a strong CYP3A inhibitor ketoconazole increased steady state eliglustat AUC
Page: 11.0
no
no
no
yes
Tox targets

Tox targets

PubMed

PubMed

TitleDatePubMed
Improvement in hematological, visceral, and skeletal manifestations of Gaucher disease type 1 with oral eliglustat tartrate (Genz-112638) treatment: 2-year results of a phase 2 study.
2010 Nov 18
Property-based design of a glucosylceramide synthase inhibitor that reduces glucosylceramide in the brain.
2012 Feb
Eliglustat, an investigational oral therapy for Gaucher disease type 1: Phase 2 trial results after 4 years of treatment.
2014 Dec
Eliglustat: first global approval.
2014 Oct
Patents

Patents

Sample Use Guides

CYP2D6 extensive metabolizers (Ems) or intermediate metabolizers (IMs): 84 mg orally twice daily (2.2); CYP2D6 poor metabolizers (PMs): 84 mg orally once daily (2.2); Swallow capsules whole, do not crush, dissolve or open capsules (2.3); Avoid eating grapefruit or drinking grapefruit juice
Route of Administration: Oral
Studies of eliglustat potency and efficacy in cell systems showed the reduction in GL-1 in a human microsome assay and in human K562 erythropoietic cells with an IC50 of around 20 nM. In murine and canine cells, potency was a lower with IC50 values of around 60 nM and 80 nM respectively.
Substance Class Chemical
Created
by admin
on Fri Dec 15 18:50:24 GMT 2023
Edited
by admin
on Fri Dec 15 18:50:24 GMT 2023
Record UNII
DR40J4WA67
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ELIGLUSTAT
DASH   INN   MI   USAN   VANDF   WHO-DD  
USAN   INN  
Official Name English
GENZ 99067
Code English
N-((1R,2R)-2-(2,3-DIHYDRO-1,4-BENZODIOXIN-6-YL)-2-HYDROXY-1-(PYRROLIDIN-1- YLMETHYL)ETHYL)OCTANAMIDE
Systematic Name English
GENZ-99067
Code English
ELIGLUSTAT [VANDF]
Common Name English
OCTANAMIDE, N-((1R,2R)-2-(2,3-DIHYDRO-1,4-BENZODIOXIN-6-YL)-2-HYDROXY-1-(1- PYRROLIDINYLMETHYL)ETHYL)-
Systematic Name English
Eliglustat [WHO-DD]
Common Name English
ELIGLUSTAT [USAN]
Common Name English
ELIGLUSTAT [MI]
Common Name English
eliglustat [INN]
Common Name English
Classification Tree Code System Code
WHO-ATC A16AX10
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
FDA ORPHAN DRUG 265408
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
NCI_THESAURUS C471
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
WHO-VATC QA16AX10
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
NDF-RT N0000175783
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
Code System Code Type Description
PUBCHEM
23652731
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
RXCUI
1547220
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY RxNorm
IUPHAR
7536
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
EVMPD
SUB129474
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
NDF-RT
N0000182137
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY Cytochrome P450 2D6 Inhibitors [MoA]
DRUG CENTRAL
4834
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
EPA CompTox
DTXSID20964175
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
USAN
UU-08
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
DAILYMED
DR40J4WA67
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
NDF-RT
N0000185503
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY P-Glycoprotein Inhibitors [MoA]
FDA UNII
DR40J4WA67
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
CAS
491833-29-5
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
ChEMBL
CHEMBL2110588
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
NCI_THESAURUS
C96282
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
LACTMED
Eliglustat
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
SMS_ID
100000155412
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
MERCK INDEX
m4869
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY Merck Index
WIKIPEDIA
Eliglustat
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
INN
9325
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
CHEBI
82752
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
DRUG BANK
DB09039
Created by admin on Fri Dec 15 18:50:24 GMT 2023 , Edited by admin on Fri Dec 15 18:50:24 GMT 2023
PRIMARY
Related Record Type Details
SALT/SOLVATE -> PARENT
METABOLIC ENZYME -> INHIBITOR
Eliglustat also exhibited time-dependent inhibition (TDI) of CYP2D6.
Ki
EXCRETED UNCHANGED
Mass balance study (GZGD02107) indicated that about 42% of the radioactive dose was recovered in urine and 51% in feces from healthy subjects who are EMs. Less than 1% total radioactivity of unchanged eliglustat was found in urine and feces, suggesting that metabolism is the primary elimination pathway for eliglustat.
FECAL; URINE
METABOLIC ENZYME -> SUBSTRATE
BINDER->LIGAND
BINDING
TRANSPORTER -> INHIBITOR
IC50
TRANSPORTER -> SUBSTRATE
METABOLIC ENZYME -> SUBSTRATE
MAJOR
TARGET -> INHIBITOR
IC50
METABOLIC ENZYME -> INHIBITOR
Ki
Related Record Type Details
METABOLITE INACTIVE -> PARENT
PLASMA; URINE
METABOLITE -> PARENT
PLASMA; URINE
Related Record Type Details
ACTIVE MOIETY
Name Property Type Amount Referenced Substance Defining Parameters References
Volume of Distribution PHARMACOKINETIC INTRAVENOUS ADMINISTRATION

in CYP2D6 extensive metabolizers (EMs)

Tmax PHARMACOKINETIC MULTIPLE DOSES

in CYP2D6 extensive metabolizers (EMs)

Biological Half-life PHARMACOKINETIC in CYP2D6 extensive metabolizers (EMs)