沃塞洛托

沃塞洛托
臨床資料
商品名（英语：Drug nomenclature）	Oxbryta
其他名稱	GBT440, GBT-440
AHFS/Drugs.com	Monograph
MedlinePlus	a620011
核准狀況	美 DailyMed: Voxelotor;
给药途径	口服
ATC碼	B06AX03 (WHO) ;
法律規範狀態
法律規範	歐：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-羥基-6-{[2-(1-異丙基-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（英語：Voxelotor）以商品名Oxbryta出售，是一種用於治療鐮刀型紅血球疾病的藥物。^[1]^[3]^[4]^[5]^[6]沃塞洛托是第一個血紅蛋白氧親和調節劑，^[7]已被證明具有通過增加鐮刀型紅血球疾病患者的血紅蛋白水平和降低溶血指標來改善疾病的潛力。^[8]它在鐮刀型紅血球疾病患者和健康志願者中具有安全性，沒有任何劑量毒性。^[9]它由輝瑞的子公司 Global Blood Therapeutics 開發。^[10]

2019年11月，沃塞洛托在美国获得加速批准，用于治疗 12 岁及以上的鐮刀型紅血球疾病。^[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岁的鐮刀型紅血球疾病。^[13]

參考資料

^ ^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）. 本文含有此來源中屬於公有领域的内容。
^ 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）. 本文含有此來源中屬於公有领域的内容。

外部連結

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）. 本文含有此來源中屬於公有领域的内容。

[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）. 本文含有此來源中屬於公有领域的内容。

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