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Drugs. 2020 Feb;80(2):209-215. doi: 10.1007/s40265-020-01262-7.
Voxelotor (Oxbryta™) is a haemoglobin S polymerization inhibitor that has been developed for the treatment of sickle cell disease. In November 2019, voxelotor received its first global approval in the USA for the treatment of sickle cell disease in adults and paediatric patients aged ≥ 12 years. The drug was granted accelerated approval based on the results of the phase III HOPE trial. Phase III clinical development of voxelotor for sickle cell disease is ongoing worldwide. Voxelotor also has Orphan Drug designation and Priority Medicine status in Europe for the treatment of sickle cell disease. This article summarizes the milestones in the development of voxelotor leading to this first approval as a disease-modifying agent for sickle cell disease.
沃瑟罗特（Oxbryta™）是一种血红蛋白 S 聚合抑制剂，已被开发用于治疗镰状细胞病。2019 年 11 月，沃瑟罗特在美国获得全球首次批准，用于治疗成人和 12 岁及以上儿童的镰状细胞病。该药基于 III 期 HOPE 试验的结果获得加速批准。沃瑟罗特治疗镰状细胞病的全球 III 期临床开发正在进行中。沃瑟罗特在欧洲也被授予治疗镰状细胞病的孤儿药资格认定和优先药物地位。本文总结了沃瑟罗特的开发里程碑，最终该药作为镰状细胞病的疾病修正治疗药物获得批准。
