VOXELOTOR

Details

Stereochemistry	ACHIRAL
Molecular Formula	C19H19N3O3
Molecular Weight	337.3725
Optical Activity	NONE
Defined Stereocenters	0 / 0
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

CC(C)N1N=CC=C1C2=NC=CC=C2COC3=C(C=O)C(O)=CC=C3

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	337.3725
Charge	0
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: https://www.ncbi.nlm.nih.gov/pubmed/27605087 | https://sicklecellanemianews.com/voxelotor-gbt440-for-sickle-cell-anemia/ | https://clinicaltrials.gov/ct2/results?cond=&term=voxelotor&cntry=&state=&city=&dist= | https://www.ncbi.nlm.nih.gov/pubmed/29352729

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

Targets

Primary Target	Pharmacology	Condition	Potency
Hemoglobin beta chain 1 Target ID: CHEMBL4331 Sources: https://www.gbt.com/pipeline/sickle-cell-disease/\|https://www.ncbi.nlm.nih.gov/pubmed/29352729\|https://www.ncbi.nlm.nih.gov/pubmed/27378309	Modulator 828

C_max

Value	Dose	Analyte	Population
2.21 μg/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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

Value	Dose	Analyte	Population
166 μg × h/mL EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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

T_1/2

Value	Dose	Analyte	Population
49.7 h EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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 EXPERIMENT https://pubmed.ncbi.nlm.nih.gov/30743314/	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

Dose	Population	Adverse 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: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/213137s000lbl.pdf	unhealthy, 12-59 years n = 88 Health Status: unhealthy Condition: sickle cell disease Age Group: 12-59 years Sex: M+F Population Size: 88 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/213137s000lbl.pdf	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: https://www.accessdata.fda.gov/drugsatfda_docs/label/2019/213137s000lbl.pdf
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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	unhealthy, 12-59 years n = 88 Health Status: unhealthy Condition: sickle cell disease Age Group: 12-59 years Sex: M+F Population Size: 88 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	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: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf
2800 mg single, oral Highest studied dose Dose: 2800 mg Route: oral Route: single Dose: 2800 mg Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	healthy n = 30 Health Status: healthy Population Size: 30 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf
1000 mg steady, oral Recommended Dose: 1000 mg Route: oral Route: steady Dose: 1000 mg Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	unhealthy Health Status: unhealthy Condition: sickle cell disease\|severe hepatic impairment Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf	Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf

AEs

Overview

CYP3A4	CYP2C9	CYP2D6	hERG
substrate 1737 inhibitor 1587	inhibitor 1767 inducer 389 substrate 1037	inhibitor 1852	inhibitor 1612

OverviewOther

Other Inhibitor	Other Substrate	Other Inducer
CYP1A2 1524 CYP2C19 1478 CYP2C8 911 BCRP 699 P-gp 1461 OATP1B1 788 OATP1B3 706 OCT2 647 OAT1 599 OAT3 642 MATE1 306 MATE2 263 BSEP 402	OATP1A2 62 OATP1B1 406 OATP1B3 350 BSEP 52 OAT1 326 OAT3 339 OCT2 355 MATE1 135 MATE2 96 CYP2C19 991 CYP2B6 664	CYP1A2 701 CYP2C19 293 CYP3A4/5 124 CYP2B6 547

Drug as perpetrator

Target	Modality	Activity	Clinical evidence
CYP2B6 1001	inducer 1573 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	likely
CYP2C8 965	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211	no [Ki 0.8 uM]	no (co-administration study) Comment: administered with rosiglitazone, no effect on rosiglitazone was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211
CYP2C19 1553	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211	no [Ki 11.1 uM]	no (co-administration study) Comment: administered with omeprazole, no effect on omeprazole was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211
CYP1A2 1692	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211	no [Ki 35 uM]	no (co-administration study) Comment: administered with caffeine, no effect on caffeine exposure was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211
CYP2C9 1819	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211	no [Ki 9.5 uM]	no (co-administration study) Comment: administered with warfarin, no effect on warfarin exposure was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211
CYP2D6 1891	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211	no [Ki 96.1 uM]	no (co-administration study) Comment: administered with metoprolol, no effect on metoprolol was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95;211
BCRP 773	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
BSEP 403	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
CYP3A4/5 335	inducer 1573 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
MATE1 308	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
MATE2 263	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OAT1 603	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OAT3 644	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OATP1B1 803	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OATP1B3 715	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OCT2 648	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
CYP1A2 1692	inducer 1573 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	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 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0
CYP2C19 1553	inducer 1573 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no	no (co-administration study) Comment: administered with omeprazole, no effect on omeprazole was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0
CYP2C9 1819	inducer 1573 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no	no (co-administration study) Comment: administered with warfarin, no effect on warfarin exposure was observed Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0
P-gp 1650	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	weak	no (co-administration study) Comment: AUC and Cmax of digoxin (a P-gp substrate) increased by only 12% and 17%. Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0
CYP3A4 1925	inhibitor 3277 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=106 Page: 106.0	yes	yes (co-administration study) Comment: midazolam exposure increased by 63% and the metabolite hydroxymidazolam increased by 75% Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=106 Page: 106.0

Drug as victim

Target	Modality	Activity
CYP3A4 1737	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=105 Page: 105.0	major
CYP2B6 664	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=105 Page: 105.0	minor
CYP2C19 991	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=105 Page: 105.0	minor
CYP2C9 1037	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=105 Page: 105.0	minor
BSEP 52	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
MATE1 135	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
MATE2 96	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OAT1 326	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OAT3 339	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OATP1A2 62	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OATP1B1 406	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OATP1B3 350	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no
OCT2 355	substrate 3367 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=95 Page: 95.0	no

Tox targets

Target	Modality	Activity	Metabolite	Clinical evidence
hERG 1647	inhibitor 1626 Sources: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2019/213137Orig1s000Multidiscipline.pdf#page=57 Page: 57.0

Sourcing

Vendor/Aggregator	ID	URL
001 Chemical	DY447663	https://www.001chemical.com/chem/1446321-46-5
AA Blocks	AA00ABL8	https://www.aablocks.com/goods/Goods/detail?id=AA00ABL8
Aaron Chemicals	AR00ACD0	http://www.aaronchem.com/1446321-46-5
AbMole Bioscience	M9778	http://www.abmole.com/products/voxelotor.html
Angel Pharmatech	AG303307	http://www.angelpharmatech.com/1446321-46-5.html
Ark Pharm	AK547400	http://www.arkpharminc.com/products/1446321-46-5.html
Ark Pharma Scientific Limited	H-022273	http://www.arkpharmtech.com/product/27327.html
BOC Sciences	1446321-46-5	https://www.bocsci.com/gbt-440-cas-1446321-46-5-item-474581.html
BioChemPartner	BCP20182	http://www.biochempartner.com/1446321-46-5-BCP20182
Chem Scene	CS-5317	http://www.chemscene.com/1446321-46-5.html
Chemspace	CSSB00025757729	https://chem-space.com/CSSB00025757729
DC Chemicals	DC10474	http://www.dcchemicals.com/product_show-GBT440.html
Debye Scientific	DA-44587	http://www.debyesci.com/cas_1446321-46-5.html
Excenen	EX-A2980	https://www.excenen.com/searchresultlist.php?id=1446321-46-5
Finetech	FT-0702904	http://www.finetechnology-ind.com/prod/detail/pub/1446321-46-5.html
Lab Network	LN01343613	https://labnetwork.com/frontend-app/p/#!/moleculedetails/LN01343613
MedChem Express	HY-18681	https://www.medchemexpress.com/GBT-440.html
Molcore	MC447663	https://www.molcore.com/product/1446321-46-5
Smolecule	S546872	http://www.smolecule.com/products/s546872
Synblock Inc	SB19656	http://www.synblock.com/product/1446321-46-5.html
THE BioTek	bt-286495	https://www.thebiotek.com/product/inhibitors/bt-286495
eNovation Chemicals	D677141	https://www.enovationchem.com/ProductDetails.asp?ProductID=D677141
eNovation Chemicals	D762780	https://www.enovationchem.com/ProductDetails.asp?ProductID=D762780
eNovation Chemicals	Y0998128	https://www.enovationchem.com/ProductDetails.asp?ProductID=Y0998128
eNovation Chemicals	Y1012667	https://www.enovationchem.com/ProductDetails.asp?ProductID=Y1012667

Substance Class	Chemical Created by `admin` on `Sat Dec 16 09:56:00 GMT 2023` Edited by `admin` on `Sat Dec 16 09:56:00 GMT 2023`
Record UNII	3ZO554A4Q8
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

VOXELOTOR

Official Name

English

VOXELOTOR [MI]

Common Name

English

VOXELOTOR [ORANGE BOOK]

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

Voxelotor [WHO-DD]

Common Name

English

GBT-440

Code

English

voxelotor [INN]

Common Name

English

Classification Tree Code System Code

NCI_THESAURUS

C78275