Voxelotor

Voxelotor, also known as GBT-440, sold under the brand name Oxbryta, is a drug for the treatment of sickle cell disease.[1][2][3][4] Developed by Global Blood Therapeutics, voxelotor is the first hemoglobin oxygen-affinity modulator.[5] Voxelotor has been shown to have disease modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.[6] It has a safe profile in sickle cell patients and healthy volunteers, without any dose limiting toxicity.[7]

Voxelotor
Clinical data
Trade namesOxbryta
Other namesGBT440, GBT-440
License data
Routes of
administration		By mouth
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
Chemical and physical data
FormulaC19H19N3O3
Molar mass337.379 g·mol−1
3D model (JSmol)

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.[8]

Side effects

Common side effects for people taking voxelotor were headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).[8]

History

Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in November 2019, and further clinical trials are required to verify and describe Oxbryta's clinical benefit.[8][9] The FDA granted the application for voxelotor fast track designation and orphan drug designation.[8][10]

The approval of voxelotor was based on the results of a clinical trial with 274 patients with sickle cell disease.[8]

The FDA granted the approval of Oxbryta to Global Blood Therapeutics.[8]

References
  1. "Voxelotor for Sickle Cell Disease". Global Blood Therapeutics. Retrieved 9 December 2018.
  2. "Voxelotor (Previously GBT440)". Sickle Cell Anemia News. Retrieved 13 December 2018.
  3. "ASH 2017: The HbS Polymerization Inhibitor Voxelotor GBT440 Has Demonstrated Positive Initial Results in Adolescents With Sickle Cell Disease". PracticeUpdate. Retrieved 16 December 2018.
  4. Adamson, Laurie (22 January 2018). "Voxelotor: A New Option for Young Patients With Sickle Cell Disease?". ASH Clinical News. Retrieved 16 December 2018.
  5. Pubchem. "Voxelotor". pubchem.ncbi.nlm.nih.gov. Retrieved 9 December 2018.
  6. Vichinsky, Elliott; Hoppe, Carolyn C.; Ataga, Kenneth I.; Ware, Russell E.; Nduba, Videlis; El-Beshlawy, Amal; Hassab, Hoda; Achebe, Maureen M.; Alkindi, Salam; Brown, R. Clark; Diuguid, David L.; Telfer, Paul; Tsitsikas, Dimitris A.; Elghandour, Ashraf; Gordeuk, Victor R.; Kanter, Julie; Abboud, Miguel R.; Lehrer-Graiwer, Joshua; Tonda, Margaret; Intondi, Allison; Tong, Barbara; Howard, Jo (8 August 2019). "A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease". New England Journal of Medicine. 381 (6): 509–519. doi:10.1056/NEJMoa1903212. PMID 31199090.
  7. Hutchaleelaha, Athiwat; Patel, Mira; Washington, Carla; Siu, Vincent; Allen, Elizabeth; Oksenberg, Donna; Gretler, Daniel D.; Mant, Timothy; Lehrer‐Graiwer, Josh (31 March 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.
  8. "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.
  9. "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.
  10. "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.


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