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Details

Stereochemistry ACHIRAL
Molecular Formula C19H19N3O3
Molecular Weight 337.3732
Optical Activity NONE
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of VOXELOTOR

SMILES

CC(C)n1c(ccn1)-c2c(cccn2)COc3cccc(c3C=O)O

InChI

InChIKey=FWCVZAQENIZVMY-UHFFFAOYSA-N
InChI=1S/C19H19N3O3/c1-13(2)22-16(8-10-21-22)19-14(5-4-9-20-19)12-25-18-7-3-6-17(24)15(18)11-23/h3-11,13,24H,12H2,1-2H3

HIDE SMILES / InChI

Molecular Formula C19H19N3O3
Molecular Weight 337.3732
Charge 0
Count
Stereochemistry ACHIRAL
Additional Stereochemistry No
Defined Stereocenters 0 / 0
E/Z Centers 0
Optical Activity NONE

GBT440 (previously GTx011) is a potent and direct drug for sickle cell treatment. In sickle cell anemia, abnormal hemoglobin molecules are formed, which causes problems for the flow of blood and oxygen through the body. GBT440 can selectively bind to hemoglobin, thereby increasing its affinity for oxygen. By inhibiting hemoglobin polymerization, it also prevents deformation of the red blood cells. GBT440, renamed Voxelotor, is thought to help prevent sickle cells blocking blood vessels, and therefore reduces pain (sickle cell crisis) experienced by patients. GBT440 is well absorbed following intravenous and oral administration, and quickly partitions into the red blood cell with a small part re‐distributed into the plasma. GBT440 was well tolerated in a randomized, placebo‐controlled, double blind, parallel group phase I/II study in healthy volunteers and sickle cell disease patients. Headache is the most reported adverse event related to the use of this drug, and no serious adverse events are known. A phase 3 clinical trial examining the efficacy and safety of the drug (compared to placebo) is planned to be completed in 2019. Voxelotor was also studied as a potential therapy for treatment of low oxygen levels in the blood of idiopathic pulmonary fibrosis patients, but this program was discontinued because of a lack of clinical benefits.

Approval Year

Cmax

Cmax

ValueDoseCo-administeredAnalytePopulation
2.21 μg/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
146 μg/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
136 μg/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
11.4 μg/mL
900 mg 1 times / day steady-state, oral
dose: 900 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
2.91 μg/mL
300 mg 1 times / day steady-state, oral
dose: 300 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
2.94 μg/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
688 μg/mL
900 mg 1 times / day steady-state, oral
dose: 900 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
225 μg/mL
300 mg 1 times / day steady-state, oral
dose: 300 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
AUC

AUC

ValueDoseCo-administeredAnalytePopulation
166 μg × h/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
10000 μg × h/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
15800 μg × h/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
215 μg × h/mL
900 mg 1 times / day steady-state, oral
dose: 900 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
56.6 μg × h/mL
300 mg 1 times / day steady-state, oral
dose: 300 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
134 μg × h/mL
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR plasma
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
14000 μg × h/mL
900 mg 1 times / day steady-state, oral
dose: 900 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
4950 μg × h/mL
300 mg 1 times / day steady-state, oral
dose: 300 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
T1/2

T1/2

ValueDoseCo-administeredAnalytePopulation
49.7 h
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: UNHEALTHY
age: ADULT
sex: FEMALE / MALE
food status: FASTED
61.1 h
1000 mg single, oral
dose: 1000 mg
route of administration: Oral
experiment type: SINGLE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
85.1 h
900 mg 1 times / day steady-state, oral
dose: 900 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
80.3 h
300 mg 1 times / day steady-state, oral
dose: 300 mg
route of administration: Oral
experiment type: STEADY-STATE
co-administered:
VOXELOTOR red blood cells
Homo sapiens
population: HEALTHY
age: ADULT
sex: MALE
food status: FASTED
Doses

Doses

DosePopulationAdverse events​
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Other AEs: Headache, Diarrhea...
Other AEs:
Headache (grade 1-2, 26%)
Diarrhea (grade 1-2, 19%)
Abdominal pain (grade 1-2, 19%)
Upper abdominal pain (grade 1-2, 19%)
Nausea (grade 1-2, 16%)
Fatigue (grade 1-2, 14%)
Rash (grade 1-2, 13%)
Urticaria (grade 1-2, 14%)
Generalized rash (grade 1-2, 10%)
Maculo-papular rash (grade 1-2, 14%)
Pruritic rash (grade 1-2, 14%)
Papular rash (grade 1-2, 14%)
Erythematous rash (grade 1-2, 14%)
Vesicular rash (grade 1-2, 14%)
Pyrexia (grade 1-2, 12%)
Diarrhea (grade 3, 1 patient)
Nausea (grade 3, 1 patient)
Rash (grade 3, 1 patient)
Generalized rash (grade 3, 3 patients)
Headache (serious, 1 patient)
Hypersensitivity (serious, 1 patient)
Pulmonary embolism (serious, 1 patient)
Headache (>1 patient)
Diarrhea (>1 patient)
Rash (>1 patient)
Vomiting (2.3%)
Sources:
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Disc. AE: Sickle cell anemia with crisis, Nausea...
Other AEs: Sickle cell anemia with crisis, Abdominal pain...
AEs leading to
discontinuation/dose reduction:
Sickle cell anemia with crisis (2.3%)
Nausea (1.1%)
Chest pain (1.1%)
Angina pectoris (1.1%)
Paresthesia (1.1%)
Pneumonia (1.1%)
Pulmonary embolism (1.1%)
Pulmonary sepsis (1.1%)
Abdominal pain (1.1%)
Other AEs:
Sickle cell anemia with crisis (19.3%)
Abdominal pain (1 patient)
Acute chest syndrome (5.7%)
Diarrhea (3.4%)
Rash (2.3%)
Sickle cell anemia with crisis (2.3%)
Generalized rash (2.3%)
Sources:
2800 mg single, oral
Highest studied dose
healthy
1000 mg steady, oral
Recommended
unhealthy
AEs

AEs

AESignificanceDosePopulation
Vomiting 2.3%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Diarrhea >1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Headache >1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Rash >1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Generalized rash grade 1-2, 10%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pyrexia grade 1-2, 12%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Rash grade 1-2, 13%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Erythematous rash grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Fatigue grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Maculo-papular rash grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Papular rash grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pruritic rash grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Urticaria grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Vesicular rash grade 1-2, 14%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Nausea grade 1-2, 16%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Abdominal pain grade 1-2, 19%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Diarrhea grade 1-2, 19%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Upper abdominal pain grade 1-2, 19%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Headache grade 1-2, 26%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Diarrhea grade 3, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Nausea grade 3, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Rash grade 3, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Generalized rash grade 3, 3 patients
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Headache serious, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Hypersensitivity serious, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pulmonary embolism serious, 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Abdominal pain 1 patient
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Abdominal pain 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Angina pectoris 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Chest pain 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Nausea 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Paresthesia 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pneumonia 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pulmonary embolism 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Pulmonary sepsis 1.1%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Sickle cell anemia with crisis 19.3%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Generalized rash 2.3%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Rash 2.3%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Sickle cell anemia with crisis 2.3%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Sickle cell anemia with crisis 2.3%
Disc. AE
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Diarrhea 3.4%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
Acute chest syndrome 5.7%
1500 mg 1 times / day steady, oral
Recommended
Dose: 1500 mg, 1 times / day
Route: oral
Route: steady
Dose: 1500 mg, 1 times / day
Sources:
unhealthy, 12-59 years
Health Status: unhealthy
Age Group: 12-59 years
Sex: M+F
Sources:
OverviewDrug as perpetrator​

Drug as perpetrator​

TargetModalityActivityMetaboliteClinical evidence
likely
no [Ki 0.8 uM]
no (co-administration study)
Comment: administered with rosiglitazone, no effect on rosiglitazone was observed
Page: 95;211
no [Ki 11.1 uM]
no (co-administration study)
Comment: administered with omeprazole, no effect on omeprazole was observed
Page: 95;211
no [Ki 35 uM]
no (co-administration study)
Comment: administered with caffeine, no effect on caffeine exposure was observed
Page: 95;211
no [Ki 9.5 uM]
no (co-administration study)
Comment: administered with warfarin, no effect on warfarin exposure was observed
Page: 95;211
no [Ki 96.1 uM]
no (co-administration study)
Comment: administered with metoprolol, no effect on metoprolol was observed
Page: 95;211
no
no
no
no
no
no
no
no
no
no
no
no (co-administration study)
Comment: administered with caffeine, no effect on caffeine exposure was observed; did not cause concentration-dependent increases in mRNA levels
Page: 95
no
no (co-administration study)
Comment: administered with omeprazole, no effect on omeprazole was observed
Page: 95
no
no (co-administration study)
Comment: administered with warfarin, no effect on warfarin exposure was observed
Page: 95
weak
no (co-administration study)
Comment: AUC and Cmax of digoxin (a P-gp substrate) increased by only 12% and 17%.
Page: 95
yes
yes (co-administration study)
Comment: midazolam exposure increased by 63% and the metabolite hydroxymidazolam increased by 75%
Page: 106
Drug as victim

Drug as victim

TargetModalityActivityMetaboliteClinical evidence
major
minor
minor
minor
no
no
no
no
no
no
no
no
no
Tox targets

Tox targets

Substance Class Chemical
Created
by admin
on Sat Jun 26 13:08:36 UTC 2021
Edited
by admin
on Sat Jun 26 13:08:36 UTC 2021
Record UNII
3ZO554A4Q8
Record Status Validated (UNII)
Record Version
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Name Type Language
VOXELOTOR
INN  
USAN   INN  
Official Name English
VOXELOTOR [ORANGE BOOK]
Common Name English
VOXELOTOR [WHO-DD]
Common Name English
VOXELOTOR [USAN]
Common Name English
OXBRYTA
Brand Name English
BENZALDEHYDE, 2-HYDROXY-6-((2-(1-(1-METHYLETHYL)-1H-PYRAZOL-5-YL)-3-PYRIDINYL)METHOXY)-
Systematic Name English
GTX-011
Code English
2-HYDROXY-6-((2-(1-ISOPROPYL-1H-PYRAZOL-5-YL)PYRIDIN-3-YL)METHOXY) BENZALDEHYDE
Systematic Name English
GBT440
Code English
GBT-440
Code English
VOXELOTOR [INN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 499715
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
NCI_THESAURUS C78275
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
EU-Orphan Drug EU/3/16/1769
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
Code System Code Type Description
DRUG CENTRAL
5356
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
NCI_THESAURUS
C152089
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
CAS
1446321-46-5
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
FDA UNII
3ZO554A4Q8
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
LACTMED
Voxelotor
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
INN
10454
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
PUBCHEM
71602803
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
DRUG BANK
DB14975
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
RXCUI
2265678
Created by admin on Sat Jun 26 13:08:36 UTC 2021 , Edited by admin on Sat Jun 26 13:08:36 UTC 2021
PRIMARY
Related Record Type Details
CUMULATIVE EXCRETION
URINE
EXCRETED UNCHANGED
FECAL
METABOLIC ENZYME -> SUBSTRATE
In vitro metabolism studies involving recombinant enzymes indicated that CYP3A4, CYP1A1, CYP3A5, CYP2C9, CYP2C19 and CYP2B6 were responsible for the oxidative metabolism of voxelotor.
METABOLIC ENZYME -> SUBSTRATE
In vitro metabolism studies involving recombinant enzymes indicated that CYP3A4, CYP1A1, CYP3A5, CYP2C9, CYP2C19 and CYP2B6 were responsible for the oxidative metabolism of voxelotor.
METABOLIC ENZYME -> SUBSTRATE
In vitro metabolism studies involving recombinant enzymes indicated that CYP3A4, CYP1A1, CYP3A5, CYP2C9, CYP2C19 and CYP2B6 were responsible for the oxidative metabolism of voxelotor.
METABOLIC ENZYME -> SUBSTRATE
MAJOR
METABOLIC ENZYME -> SUBSTRATE
In vitro metabolism studies involving recombinant enzymes indicated that CYP3A4, CYP1A1, CYP3A5, CYP2C9, CYP2C19 and CYP2B6 were responsible for the oxidative metabolism of voxelotor.
METABOLIC ENZYME -> INHIBITOR
BINDER->LIGAND
BINDING
METABOLIC ENZYME -> SUBSTRATE
In vitro metabolism studies involving recombinant enzymes indicated that CYP3A4, CYP1A1, CYP3A5, CYP2C9, CYP2C19 and CYP2B6 were responsible for the oxidative metabolism of voxelotor.
CUMULATIVE EXCRETION
FECAL
TARGET->INHIBITOR OF AGGREGATION
BINDS DEOXYHEMOGLOBIN S WITH 1:1 STOICHIOMETRY; BLOOD:PLASMA RATIO 15:1
EXCRETED UNCHANGED
URINE
Related Record Type Details
METABOLITE -> PARENT
FECAL; URINE
METABOLITE -> PARENT
FECAL; URINE
METABOLITE -> PARENT
FECAL; URINE
METABOLITE -> PARENT
FECAL; URINE
Related Record Type Details
ACTIVE MOIETY
Official Title: A Phase I Randomised, Placebo-controlled, Double-blind, Single and Multiple Ascending Dose Study of the Tolerability and Pharmacokinetics of GBT440 in Healthy Subjects and Patients With Sickle Cell Disease Purpose : The purpose of this study is to assess the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of GBT440 compared with placebo in healthy subjects and subjects with sickle cell disease (SCD).
Name Property Type Amount Referenced Substance Defining Parameters References
Tmax PHARMACOKINETIC
Volume of Distribution PHARMACOKINETIC
Volume of Distribution PHARMACOKINETIC
Biological Half-life PHARMACOKINETIC