Clinical data | |
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Trade names | Oxbryta |
Other names | GBT440, GBT-440 |
AHFS/Drugs.com | Monograph |
MedlinePlus | a620011 |
License data | |
Routes of administration | By mouth |
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Chemical and physical data | |
Formula | C19H19N3O3 |
Molar mass | 337.379 g·mol−1 |
3D model (JSmol) | |
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Voxelotor, sold under the brand name Oxbryta, is a medication used for the treatment of sickle cell disease.[1][3][4][5][6] Voxelotor is the first hemoglobin oxygen-affinity modulator.[7] Voxelotor has been shown to have disease-modifying potential by increasing hemoglobin levels and decreasing hemolysis indicators in sickle cell patients.[8] It has a safe profile in sickle cell patients and healthy volunteers, without any dose-limiting toxicity.[9] It was developed by Global Blood Therapeutics, a subsidiary of Pfizer.[10]
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.[11][12] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[13] 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.[14]
Common side effects include headache, diarrhea, abdominal pain, nausea, fatigue, rash and pyrexia (fever).[11]
Voxelotor was granted accelerated approval by the U.S. Food and Drug Administration (FDA) in November 2019.[12][11][15] The FDA granted the application for voxelotor fast track designation and orphan drug designation.[11][16]
The approval of voxelotor was based on the results of a clinical trial with 274 participants with sickle cell disease.[11] The FDA granted the approval of Oxbryta to Global Blood Therapeutics.[11][12]
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.[17][18] The applicant for this medicinal product is Global Blood Therapeutics Netherlands B.V.[17][18] Voxelotor (Oxbryta) was approved for medical use in the European Union in February 2022.[2][19]