Voxelotor

Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease.[1][2][3][4][5] Developed by Global Blood Therapeutics, voxelotor is the first hemoglobin oxygen-affinity modulator.[6] Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.[7] It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity.[8]

Voxelotor

Clinical data
Trade names	Oxbryta
Other names	GBT440, GBT-440
AHFS/Drugs.com	Monograph
MedlinePlus	a620011
License data	US DailyMed: Voxelotor US FDA: Oxbryta
Routes of administration	By mouth
ATC code	None
Legal status
Legal status	US: ℞-only
Identifiers
IUPAC name 2-Hydroxy-6-{[2-(1-isopropyl-1H-pyrazol-5-yl)-3-pyridinyl]methoxy}benzaldehyde
CAS Number	1446321-46-5
PubChem CID	71602803
DrugBank	DB14975
ChemSpider	37999268
UNII	3ZO554A4Q8
KEGG	D11330
ChEMBL	ChEMBL4101807
Chemical and physical data
Formula	C19H19N3O3
Molar mass	337.379 g·mol^−1
3D model (JSmol)	Interactive image
SMILES CC(C)N1C(=CC=N1)C2=C(C=CC=N2)COC3=CC=CC(=C3C=O)O
InChI 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 Key:FWCVZAQENIZVMY-UHFFFAOYSA-N

In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease (SCD) for those 12 years of age and older.[9][10] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[11]

Side effects

Common side effects for people taking voxelotor were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).[9]

History

Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in November 2019,[10] and further clinical trials are required to verify and describe Oxbryta's clinical benefit.[9][12] The FDA granted the application for voxelotor fast track designation and orphan drug designation.[9][13]

The approval of voxelotor was based on the results of a clinical trial with 274 participants with sickle cell disease.[9]

The FDA granted the approval of Oxbryta to Global Blood Therapeutics.[9][10]

References

1. "Oxbryta- voxelotor tablet, film coated". DailyMed. 3 December 2019. Retrieved 22 January 2020.
2. "Voxelotor for Sickle Cell Disease". Global Blood Therapeutics. Retrieved 9 December 2018.
3. "Voxelotor (Previously GBT440)". Sickle Cell Anemia News. Retrieved 13 December 2018.
4. "ASH 2017: The HbS Polymerization Inhibitor Voxelotor GBT440 Has Demonstrated Positive Initial Results in Adolescents With Sickle Cell Disease". PracticeUpdate. Retrieved 16 December 2018.
5. Adamson, Laurie (22 January 2018). "Voxelotor: A New Option for Young Patients With Sickle Cell Disease?". ASH Clinical News. Retrieved 16 December 2018.
6. "Voxelotor". PubChem. Retrieved 9 December 2018.
7. Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, et al. (August 2019). "A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease". The New England Journal of Medicine. 381 (6): 509–519. doi:10.1056/NEJMoa1903212. PMID 31199090.
8. Hutchaleelaha A, Patel M, Washington C, Siu V, Allen E, Oksenberg D, et al. (June 2019). "Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease". British Journal of Clinical Pharmacology. 85 (6): 1290–1302. doi:10.1111/bcp.13896. PMC 6533444. PMID 30743314.
9. "FDA approves novel treatment to target abnormality in sickle cell disease". U.S. Food and Drug Administration (FDA). 25 November 2019. Archived from the original on 25 November 2019. Retrieved 25 November 2019. This article incorporates text from this source, which is in the public domain.
10. "Drug Approval Package: Oxbryta". U.S. Food and Drug Administration (FDA). 23 December 2019. Retrieved 22 January 2020.
11. "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.
12. "Drug Trials Snapshots: Oxbryta". U.S. Food and Drug Administration (FDA). 11 December 2019. Archived from the original on 15 December 2019. Retrieved 15 December 2019. This article incorporates text from this source, which is in the public domain.
13. "Voxelotor Orphan Drug Designation". U.S. Food and Drug Administration (FDA). 25 November 2019. Archived from the original on 26 November 2019. Retrieved 25 November 2019. This article incorporates text from this source, which is in the public domain.

External links

• "Voxelotor". Drug Information Portal. U.S. National Library of Medicine.