FDA批准赛诺菲的Enjaymo治疗冷凝集素病

2022-02-05 Allan MedSci原创

FDA本周五宣布,它已批准赛诺菲(Sanofi)的 Enjaymo(sutimlimab-jome)以减少成人冷凝集素病(CAD)溶血导致的红细胞输血需求。

冷凝集素病(cold agglutinin disease,CAD)是一种自身免疫性溶血性贫血(autoimmune hemolytic anemia, AIHA)。冷凝集素是以3℃-4℃为最适温度的凝集性自身抗体,可引起身体低温部位出现与红细胞凝集相关的临床症状和溶血性贫血。具体而言,冷凝集素是可在低于正常核心体温时识别红细胞上抗原的抗体,这些抗体可引起红细胞凝集和血管外溶血,从而导致贫血,通常没有血红蛋白尿。

图1. 经典补体途径的持续 C1 激活使患者处于慢性溶血状态

FDA本周五宣布,它已批准赛诺菲(Sanofi)的 补体 C1s 抑制剂 Enjaymo(sutimlimab-jome)以减少成人冷凝集素病(CAD)溶血导致的红细胞输血需求 (图1)。

该批准是基于单臂 III 期 CARDINAL 试验的最新结果,该试验纳入了 24 名最近接受过输血的 CAD 患者。所有参与者都接受了长达六个月的 Enjaymo 输液,并且可以选择在试验的第二部分继续治疗。结果显示,54% 的患者符合复合主要终点的标准。反应定义为血红蛋白 (Hb) 增加≥2 g/dL。

FDA 指出,Enjaymo 的常见副作用是呼吸道感染、病毒感染、腹泻、消化不良、咳嗽、关节痛、关节炎以及小腿和手部肿胀。

去年,赛诺菲报告了 Enjaymo 第二次 III 期试验的积极数据。CADENZA 研究的 A 部分招募了 42 名在前六个月内没有输过血的 CAD 患者,他们被随机分配到 Enjaymo 或安慰剂组。在主要复合终点上,Enjaymo 组 73% 的患者实现了反应,而安慰剂组为 15%。反应被定义为在直到第 26 周的特定时间点,Hb 从基线改善≥1.5 g/dL,并且从第 5 周到第 26 周没有输血需求。

赛诺菲表示,预计未来几周将在美国上市的 Enjaymo 的标价为每瓶 1800 美元。赛诺菲负责人 Bill Sibold 指出,“Enjaymo 是唯一获批的抑制 CAD 中红细胞破坏的治疗方法”。

 

原始出处:

https://firstwordpharma.com/story/5493697

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    2022-02-05 ms2000001692188884

    0

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    2022-02-05 仁术2021

    不错学习了

    0

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