沃塞洛托

沃塞洛托
临床资料
商品名（英语：Drug nomenclature）	Oxbryta
其他名称	GBT440, GBT-440
AHFS/Drugs.com	Monograph
MedlinePlus	a620011
核准状况	美 DailyMed: Voxelotor;
给药途径	口服
ATC码	B06AX03 (WHO) ;
法律规范状态
法律规范	欧：Rx-only; 美：处方药（℞-only）;
识别信息
	IUPAC命名法 2-Hydroxy-6-{[2-(1-isopropyl-1H-pyrazol-5-yl)-3-pyridinyl]methoxy}benzaldehyde; 2-羟基-6-{[2-(1-异丙基-1H-5-吡唑基)-3-吡啶基]甲氧基}苯甲醛;
CAS号	1446321-46-5
PubChem CID	71602803;
DrugBank	DB14975;
ChemSpider	37999268;
UNII	3ZO554A4Q8;
KEGG	D11330;
ChEMBL	ChEMBL4101807;
CompTox Dashboard（英语：CompTox Chemicals Dashboard） (EPA)	DTXSID801027954 ;
化学信息
化学式	C19H19N3O3
摩尔质量	337.38 g·mol−1
3D模型（JSmol（英语：JSmol））	交互式图像;
	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;

沃塞洛托（英语：Voxelotor）以商品名Oxbryta出售，是一种用于治疗镰刀型红血球疾病的药物。^[1]^[3]^[4]^[5]^[6]沃塞洛托是第一个血红蛋白氧亲和调节剂，^[7]已被证明具有通过增加镰刀型红血球疾病患者的血红蛋白水平和降低溶血指标来改善疾病的潜力。^[8]它在镰刀型红血球疾病患者和健康志愿者中具有安全性，没有任何剂量毒性。^[9]它由辉瑞的子公司 Global Blood Therapeutics 开发。^[10]

2019年11月，沃塞洛托在美国获得加速批准，用于治疗 12 岁及以上的镰刀型红血球疾病。^[11]^[12]2021年12月，沃塞洛托在美国获得加速批准，用于治疗4 - 11岁的镰刀型红血球疾病。^[13]

参考资料

^ ^1.0 ^1.1 Oxbryta- voxelotor tablet, film coated. DailyMed. 3 December 2019 [22 January 2020]. （原始内容存档于2021-01-04）.
^ Oxbryta EPAR. European Medicines Agency. 14 December 2021 [20 April 2022]. （原始内容存档于2023-03-01）. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
^ Voxelotor for Sickle Cell Disease. Global Blood Therapeutics. [2018-12-09]. （原始内容存档于2018-12-16）.
^ Voxelotor (Previously GBT440). Sickle Cell Anemia News. [2018-12-13]. （原始内容存档于2021-03-04）.
^ ASH 2017: The HbS Polymerization Inhibitor Voxelotor GBT440 Has Demonstrated Positive Initial Results in Adolescents With Sickle Cell Disease. PracticeUpdate. [16 December 2018]. （原始内容存档于2018-12-16）.
^ Adamson, Laurie. Voxelotor: A New Option for Young Patients With Sickle Cell Disease?. ASH Clinical News. 22 January 2018 [16 December 2018]. （原始内容存档于2018-12-16）.
^ Voxelotor. PubChem. [2018-12-09]. （原始内容存档于2020-08-10）.
^ Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, et al. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. The New England Journal of Medicine. August 2019, 381 (6): 509–519. PMID 31199090. doi:10.1056/NEJMoa1903212  .
^ Hutchaleelaha A, Patel M, Washington C, Siu V, Allen E, Oksenberg D, et al. Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease. British Journal of Clinical Pharmacology. June 2019, 85 (6): 1290–1302. PMC 6533444  . PMID 30743314. doi:10.1111/bcp.13896  .
^ 存档副本. [2023-02-27]. （原始内容存档于2023-02-21）.
^ FDA approves novel treatment to target abnormality in sickle cell disease. U.S. Food and Drug Administration (FDA). 25 November 2019 [25 November 2019]. （原始内容存档于25 November 2019）. 本文含有此来源中属于公有领域的内容。
^ Drug Approval Package: Oxbryta. U.S. Food and Drug Administration (FDA). 23 December 2019 [22 January 2020]. （原始内容存档于2020-11-02）.
^ FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years. U.S. Food and Drug Administration (FDA) (新闻稿). 17 December 2021 [17 December 2021]. （原始内容存档于2023-02-26）. 本文含有此来源中属于公有领域的内容。

外部链接

Voxelotor. Drug Information Portal. U.S. National Library of Medicine. [2023-02-27]. （原始内容存档于2022-12-07）.

[Oxbryta_label-1] 1.0 ^1.1 Oxbryta- voxelotor tablet, film coated. DailyMed. 3 December 2019 [22 January 2020]. （原始内容存档于2021-01-04）.

[Oxbryta_EPAR-2] Oxbryta EPAR. European Medicines Agency. 14 December 2021 [20 April 2022]. （原始内容存档于2023-03-01）. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.

[3] Voxelotor for Sickle Cell Disease. Global Blood Therapeutics. [2018-12-09]. （原始内容存档于2018-12-16）.

[4] Voxelotor (Previously GBT440). Sickle Cell Anemia News. [2018-12-13]. （原始内容存档于2021-03-04）.

[5] ASH 2017: The HbS Polymerization Inhibitor Voxelotor GBT440 Has Demonstrated Positive Initial Results in Adolescents With Sickle Cell Disease. PracticeUpdate. [16 December 2018]. （原始内容存档于2018-12-16）.

[6] Adamson, Laurie. Voxelotor: A New Option for Young Patients With Sickle Cell Disease?. ASH Clinical News. 22 January 2018 [16 December 2018]. （原始内容存档于2018-12-16）.

[7] Voxelotor. PubChem. [2018-12-09]. （原始内容存档于2020-08-10）.

[8] Vichinsky E, Hoppe CC, Ataga KI, Ware RE, Nduba V, El-Beshlawy A, et al. A Phase 3 Randomized Trial of Voxelotor in Sickle Cell Disease. The New England Journal of Medicine. August 2019, 381 (6): 509–519. PMID 31199090. doi:10.1056/NEJMoa1903212  .

[9] Hutchaleelaha A, Patel M, Washington C, Siu V, Allen E, Oksenberg D, et al. Pharmacokinetics and pharmacodynamics of voxelotor (GBT440) in healthy adults and patients with sickle cell disease. British Journal of Clinical Pharmacology. June 2019, 85 (6): 1290–1302. PMC 6533444  . PMID 30743314. doi:10.1111/bcp.13896  .

[10] 存档副本. [2023-02-27]. （原始内容存档于2023-02-21）.

[FDA_PR-11] FDA approves novel treatment to target abnormality in sickle cell disease. U.S. Food and Drug Administration (FDA). 25 November 2019 [25 November 2019]. （原始内容存档于25 November 2019）. 本文含有此来源中属于公有领域的内容。

[FDA_Approval-12] Drug Approval Package: Oxbryta. U.S. Food and Drug Administration (FDA). 23 December 2019 [22 January 2020]. （原始内容存档于2020-11-02）.

[FDA_PR_20211217-13] FDA approves drug to treat sickle cell disease in patients aged 4 up to 11 years. U.S. Food and Drug Administration (FDA) (新闻稿). 17 December 2021 [17 December 2021]. （原始内容存档于2023-02-26）. 本文含有此来源中属于公有领域的内容。

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