诺和诺德向美国食品药品管理局递交镰状细胞病药物etavopivat上市申请：三期试验血管闭塞危象减少27%，适用12岁及以上患者
诺和诺德 11 月 13 日宣布，已向美国食品药品管理局递交口服药物 etavopivat 的新药上市申请，并向欧洲药品管理局递交上市许可申请，用于治疗镰状细胞病。
申请针对 12 岁及以上患者。食品药品管理局预计将在 2027 年作出决定。
在三期 HIBISCUS 试验中，接受 etavopivat 治疗的患者血管闭塞危象较安慰剂组减少 27%，首次血管闭塞危象出现的时间较安慰剂组推迟约 4 个月。
试验第 24 周时，治疗组有 48.7% 的患者达到血红蛋白应答，安慰剂组为 7.2%。
该药此前已获得食品药品管理局的快速通道资格、罕见儿科疾病资格与孤儿药资格。
Novo Nordisk Submits Etavopivat to the Food and Drug Administration for Sickle Cell Disease: the Phase 3 Trial Cut Vaso Occlusive Crises by 27 Percent, for Patients Aged 12 and Older
Novo Nordisk announced on November 13 that it has submitted a New Drug Application to the U.S. Food and Drug Administration for the oral drug etavopivat, along with a marketing authorization application to the European Medicines Agency, for the treatment of sickle cell disease.
The applications cover patients aged 12 and older. A Food and Drug Administration decision is expected in 2027.
In the Phase 3 HIBISCUS trial, patients treated with etavopivat had 27 percent fewer vaso-occlusive crises than those on placebo, and the time to a first vaso-occlusive crisis was delayed by about four months compared with placebo.
At week 24 of the trial, 48.7 percent of patients in the treatment group achieved a hemoglobin response, compared with 7.2 percent on placebo.
The drug has previously received Fast Track, Rare Pediatric Disease and Orphan Drug designations from the Food and Drug Administration.
来源：诺和诺德
发布：GSM·美国（发布链接）
图片来源：图：血红蛋白SC病（镰状细胞病的一种）患者血液涂片显微图像，可见靶形红细胞与镰状细胞。Spicy，CC BY-SA 4.0，经 Wikimedia Commons。 / Photo: Microscopic blood film in hemoglobin SC disease, a form of sickle cell disease, showing target and sickle cells. By Spicy, CC BY-SA 4.0, via Wikimedia Commons.
GSM Global · 本篇英文版 English edition of this story
