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Details

Stereochemistry ACHIRAL
Molecular Formula C19H19N3O3
Molecular Weight 337.3725
Optical Activity UNSPECIFIED
Defined Stereocenters 0 / 0
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of VOXELOTOR

SMILES

CC(C)N1N=CC=C1C2=C(COC3=CC=CC(O)=C3C=O)C=CC=N2

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

Description

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

Targets

Primary TargetPharmacologyConditionPotency
Name Type Language
VOXELOTOR
INN  
USAN   INN  
Official Name English
VOXELOTOR [WHO-DD]
Common Name English
VOXELOTOR [USAN]
Common 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
Common Name English
GBT-440
Common Name English
VOXELOTOR [INN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 499715
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
NCI_THESAURUS C78275
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
EU-Orphan Drug EU/3/16/1769
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
Code System Code Type Description
NCI_THESAURUS
C152089
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
PRIMARY
CAS
1446321-46-5
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
PRIMARY
INN
10454
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
PRIMARY
PUBCHEM
71602803
Created by admin on Mon Oct 21 19:54:56 UTC 2019 , Edited by admin on Mon Oct 21 19:54:56 UTC 2019
PRIMARY