Voxelotor

Voxelotor
Names
Trade namesOxbryta
Other namesGBT440, GBT-440
IUPAC name
  • 2-Hydroxy-6-{[2-(1-isopropyl-1H-pyrazol-5-yl)-3-pyridinyl]methoxy}benzaldehyde
Clinical data
Drug classHemoglobin S polymerization inhibitor[1]
Main usesSickle cell disease[1]
Side effectsHeadache, abdominal pain, rash, fever, tiredness[1]
Routes of
use		By mouth
Typical dose1.5 gm OD[1]
External links
AHFS/Drugs.comMonograph
US NLMVoxelotor
MedlinePlusa620011
Legal
License data
Legal status
Chemical and physical data
FormulaC19H19N3O3
Molar mass337.379 g·mol−1
3D model (JSmol)
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

Voxelotor, sold under the brand name Oxbryta, is a medication used to treat sickle cell disease.[1] Evidence shows that it improves hemoglobin levels; however, overall benefits are unclear as of 2021.[1] It is taken by mouth.[1]

Common side effects include headache, abdominal pain, rash, fever, and tiredness.[1] Severe side effects may include allergic reactions.[1] Use in pregnancy is of unclear safety.[2] It is believed to work by improving the ability of abnormal hemoglobin to carry oxygen.[3]

Voxelotor was approved for medical use in the United States in 2019.[1] In Europe it was granted orphan designation in 2016.[3] In the United States it costs about 10,100 USD per month as of 2021.[4]

Medical uses

It is used to treat sickle cell disease.[5] Evidence shows that it improves hemoglobin values; however, overall benefits are unclear as of 2021.[1]

Dosage

The typical dose is 1.5 grams taken once per day; though doses of 1 gram to 2.5 grams per day may be used.[1]

Side effects

Common side effects for people taking voxelotor were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).[6]

History

Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in 2019,[7] and further clinical trials are required to verify and describe its clinical benefit.[6][8] The FDA granted the application for voxelotor fast track designation and orphan drug designation.[6][9] FDA considers it to be a first-in-class medication.[10] The approval of voxelotor was based on the results of a clinical trial with 274 participants with sickle cell disease.[6] The FDA granted the approval to Global Blood Therapeutics.[6][7]

In Europe it was granted orphan designation in 2016.[3] Developed by Global Blood Therapeutics, voxelotor is the first hemoglobin oxygen-affinity modulator.[11]

References

  1. 1 2 3 4 5 6 7 8 9 10 11 12 "Voxelotor Monograph for Professionals". Drugs.com. Retrieved 16 September 2021.
  2. ↑ "Voxelotor (Oxbryta) Use During Pregnancy". Drugs.com. Archived from the original on 21 September 2020. Retrieved 16 September 2021.
  3. 1 2 3 "EU/3/16/1769: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)". Archived from the original on 22 September 2021. Retrieved 16 September 2021.
  4. ↑ "Oxbryta Prices, Coupons & Savings Tips - GoodRx". GoodRx. Archived from the original on 26 January 2021. Retrieved 16 September 2021.
  5. ↑ "Oxbryta- voxelotor tablet, film coated". DailyMed. 3 December 2019. Archived from the original on 4 January 2021. Retrieved 22 January 2020.
  6. 1 2 3 4 5 "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. Public Domain This article incorporates text from this source, which is in the public domain.
  7. 1 2 "Drug Approval Package: Oxbryta". U.S. Food and Drug Administration (FDA). 23 December 2019. Archived from the original on 2 November 2020. Retrieved 22 January 2020.
  8. ↑ "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. Public Domain This article incorporates text from this source, which is in the public domain.
  9. ↑ "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. Public Domain This article incorporates text from this source, which is in the public domain.
  10. ↑ "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Archived from the original on 16 September 2020. Retrieved 15 September 2020.
  11. ↑ "Voxelotor". PubChem. Archived from the original on 10 August 2020. Retrieved 9 December 2018.
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