Voxelotor Explained

Tradename:Oxbryta
Dailymedid:Voxelotor
Routes Of Administration:By mouth
Atc Prefix:B06
Atc Suffix:AX03
Legal Us:Rx-only
Legal Eu:Rx-only
Cas Number:1446321-46-5
Pubchem:71602803
Drugbank:DB14975
Chemspiderid:37999268
Unii:3ZO554A4Q8
Kegg:D11330
Chembl:4101807
Synonyms:GBT440, GBT-440
Iupac Name:2-Hydroxy-6-benzaldehyde
C:19
H:19
N:3
O:3
Smiles:CC(C)N1C(=CC=N1)C2=C(C=CC=N2)COC3=CC=CC(=C3C=O)O
Stdinchi: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
Stdinchikey:FWCVZAQENIZVMY-UHFFFAOYSA-N

Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease.[1] [2] [3] [4] [5] 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] It was developed by Global Blood Therapeutics, a subsidiary of Pfizer.[9]

In November 2019, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those twelve years of age and older.[10] [11] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[12] In December 2021, voxelotor received accelerated approval in the United States for the treatment of sickle cell disease for those aged four to eleven years.[13]

Side effects

Common side effects include headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).

History

Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in November 2019.[14] The FDA granted the application for voxelotor fast track designation and orphan drug designation.[15]

The approval of voxelotor was based on the results of a clinical trial with 274 participants with sickle cell disease. The FDA granted the approval of Oxbryta to Global Blood Therapeutics.

Society and culture

Legal status

On 16 December 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Oxbryta, intended for the treatment of hemolytic anemia due to sickle cell disease. The applicant for this medicinal product is Global Blood Therapeutics Netherlands B.V.[16] [17] Voxelotor (Oxbryta) was approved for medical use in the European Union in February 2022.[18] [19]

External links

Notes and References

  1. Web site: Oxbryta- voxelotor tablet, film coated . DailyMed . 3 December 2019 . 22 January 2020.
  2. Web site: Voxelotor for Sickle Cell Disease . Global Blood Therapeutics. 9 December 2018.
  3. Web site: Voxelotor (Previously GBT440). Sickle Cell Anemia News. 13 December 2018.
  4. Web site: ASH 2017: The HbS Polymerization Inhibitor Voxelotor GBT440 Has Demonstrated Positive Initial Results in Adolescents With Sickle Cell Disease . PracticeUpdate . 16 December 2018 .
  5. Web site: Adamson . Laurie . Voxelotor: A New Option for Young Patients With Sickle Cell Disease? . ASH Clinical News . 16 December 2018 . 22 January 2018.
  6. Web site: Voxelotor. PubChem. 9 December 2018.
  7. Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, Hassab H, Achebe MM, Alkindi S, Brown RC, Diuguid DL, Telfer P, Tsitsikas DA, Elghandour A, Gordeuk VR, Kanter J, Abboud MR, Lehrer-Graiwer J, Tonda M, Intondi A, Tong B, Howard J . 6 . A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease . The New England Journal of Medicine . 381 . 6 . 509–519 . August 2019 . 31199090 . 10.1056/NEJMoa1903212 . free .
  8. Hutchaleelaha A, Patel M, Washington C, Siu V, Allen E, Oksenberg D, Gretler DD, Mant T, Lehrer-Graiwer J . 6 . Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease . British Journal of Clinical Pharmacology . 85 . 6 . 1290–1302 . June 2019 . 30743314 . 10.1111/bcp.13896 . 6533444 . free .
  9. Pfizer Completes Acquisition of Global Blood Therapeutics . 5 October 2022 .
  10. Web site: FDA approves novel treatment to target abnormality in sickle cell disease . U.S. Food and Drug Administration (FDA) . 25 November 2019 . https://web.archive.org/web/20191125220853/https://www.fda.gov/news-events/press-announcements/fda-approves-novel-treatment-target-abnormality-sickle-cell-disease . 25 November 2019 . live . 25 November 2019.
  11. Web site: Drug Approval Package: Oxbryta . U.S. Food and Drug Administration (FDA) . 23 December 2019 . 22 January 2020.
  12. Web site: New Drug Therapy Approvals 2019 . U.S. Food and Drug Administration . 31 December 2019 . 15 September 2020.
  13. FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years . U.S. Food and Drug Administration (FDA) . 17 December 2021 . 17 December 2021.
  14. Web site: Drug Trials Snapshots: Oxbryta . U.S. Food and Drug Administration (FDA) . 11 December 2019 . https://web.archive.org/web/20191215205251/https://www.fda.gov/drugs/resources-information-approved-drugs/drug-trials-snapshots-oxbryta . 15 December 2019 . live . 15 December 2019.
  15. Web site: Voxelotor Orphan Drug Designation . U.S. Food and Drug Administration (FDA) . 25 November 2019 . https://web.archive.org/web/20191126060431/https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=499715 . 26 November 2019 . live . 25 November 2019.
  16. Web site: Oxbryta: Pending EC decision . European Medicines Agency . 17 December 2021 . 18 December 2021. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  17. New treatment for sickle cell disease . European Medicines Agency (RMA) . 17 December 2021 . 18 December 2021.
  18. Web site: Oxbryta EPAR . European Medicines Agency . 14 December 2021 . 20 April 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  19. Web site: Oxbryta Product information . Union Register of medicinal products . 3 March 2023.

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