Drug Name |
Voxelotor |
Drug ID |
BADD_D02411 |
Description |
Voxelotor is a novel hemoglobin S polymerization inhibitor for the treatment of sickle cell disease. This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India. Sickle cell disease can lead to excruciating pain, stroke, infection, and various other complications arising from the blockage of blood vessels.[T734]
Voxelotor was granted accelerated FDA approval on November 25 2019, as it likely to be a promising treatment for the 100,000 individuals in the U.S. suffering from the disease, in addition to 20 million others worldwide.[L10403] It was developed by Global Blood Therapeutics, Inc.[L10403] and is unique from other drugs used to treat sickle cell anemia, such as [hydroxyurea], [L-glutamine], and [crizanlizumab][A188135,A188138] due to its novel mechanism of action. |
Indications and Usage |
Voxelotor is indicated to treat sickle cell disease in both adult and pediatric patients aged 4 years and older.[L10397] |
Marketing Status |
approved; investigational |
ATC Code |
B06AX03 |
DrugBank ID |
DB14975
|
KEGG ID |
D11330
|
MeSH ID |
C000628792
|
PubChem ID |
71602803
|
TTD Drug ID |
D09RII
|
NDC Product Code |
72786-101; 72786-102; 59116-5720; 66406-0333; 66406-0442; 72786-111; 66406-0301; 11014-0463 |
UNII |
3ZO554A4Q8
|
Synonyms |
voxelotor | 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)-3-pyridinyl)methoxy)benzaldehyde | Oxbryta | GBT440 |