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近日，美国食品和药物管理局今天批准Ultomiris (ravulizumab)注射液治疗成人突发夜间血红蛋白尿症(PNH)，这是一种罕见的危及生命的血液病。
“Ultomiris的批准将改变PNH患者的治疗方式，”医学博士Richard Pazdur说他是FDA肿瘤卓越中心主任和FDA药物评估和研究中心血液和肿瘤产品办公室代理主任。“在获得批准之前，唯一获得批准的PNH疗法需要每两周进行一次治疗，这对患者及其家人来说可能是一种负担。”Ultomiris使用了一种新的配方，因此患者每8周只需要接受一次治疗，而不会影响疗效。
批准日期：2018年12月21日 公司：Alexion Pharmaceuticals, Inc.
ULTOMIRIS(ravulizumab-cwvz)注射液，静脉使用
美国初步批准:2018年
警告:严重脑膜炎球菌感染使用ULTOMIRIS治疗的患者发生了危及生命的脑膜炎球菌感染/败血症，如果没有及早发现和治疗，可能会迅速危及生命或致命。
•遵守当前针对补充不足的脑膜炎球菌疫苗住院患者的免疫接种实践咨询委员会(ACIP)建议。
•使用脑膜炎球菌疫苗免疫患者至少2周，比第一次使用ULTOMIRIS要早，除非延迟使用ULTOMIRIS治疗的风险大于发生ameningococcal感染的风险。(见关于脑膜炎球菌风险管理的额外指导的警告和预防措施感染。)接种疫苗可以降低但不能消除脑膜炎球菌感染的风险。
•监测患者脑膜炎球菌感染的早期症状，怀疑感染时立即评估。
ULTOMIRIS只有通过aRisk评估和缓解策略(REMS)下的受限程序才能获得。根据ultomiris REMS，处方医生必须参加该项目。
作用机制
Ravulizumab-cwvz是一种终末补体抑制剂，特异性结合复合物蛋白C5具有高亲和力，从而抑制其与C5a的裂解促炎性黄曲霉毒素)和C5b(终末补体复合物的起始亚基[C5b-9])，并阻止终末补体的生成复杂的C5b9。ULTOMIRIS抑制终末互补介导的PNH患者血管内溶血。
适应症和用法
ULTOMIRIS是一种补体抑制剂，用于治疗发作性夜间血红蛋白尿(PNH)的成人患者。
剂量和管理
只作为静脉输液使用。
体重依赖型剂量给药方案:
体重范围(kg) 负荷剂量(毫克) 维修剂量(mg)
大于或等于40到小于60 2400 3000
大于或等于60超过100 2700 3300
大于或等于100 3000 3600
重要的准备和管理指示见完整的处方信息。
剂型和强度
注射:单剂量瓶300mg/30mL(10mg/mL)。
禁忌症
未解决的NeisseriaMeningitidis感染患者禁用ULTOMIRIS。
警告和预防措施
给有其他全身感染的病人使用ULTOMIRIS时要小心。
不良反应
最常见的不良反应(>10%)是上呼吸系统感染和头痛。
报告疑似不良反应，联系阿历克西翁制药公司(电话:1-844-259-6783)或食品药品管理局(电话:1- 800-fdaa-1088或www.fda.gov/medwatch)。
包装提供/存储和处理
ULTOMIRIS (ravulizumab-cwvz)注射液为透明至半透明，微白无色素防腐剂，每盒300mg/30mL(10mg/mL)一剂。NDC 25682-022-01。
ULTOMIRIS瓶冷藏储存在2°C - 8°C(36°F - 46°F)在原来的纸箱保护。不冻结。不动摇。有关乌托膜稀释液的稳定性及贮存情况，请参阅。
Ultomiris Approved for Paroxysmal Nocturnal Hemoglobinuria
The Food and Drug Administration (FDA) has approved Ultomiris (ravulizumab-cwvz; Alexion) injection for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
“Prior to this approval, the only approved therapy for PNH required treatment every 2 weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every 8 weeks, without compromising efficacy," stated Richard Pazdur, MD, director of FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research.
Ultomiris, a long-acting complement inhibitor, prevents hemolysis by specifically binding to the complement protein C5, inhibiting its cleavage to C5a and C5b, and thus preventing the generation of the terminal complement complex C5b9.
The safety and efficacy of Ultomiris in patients with PNH was assessed in 2 open-label, randomized, active-controlled, non-inferiority Phase 3 studies. In PNH Study 301, patients naïve to complement inhibitor treatment (N=246) were randomized to receive Ultomiris or eculizumab (Soliris; current standard of care). Results showed Ultomiris was noninferior to eculizumab as evidenced by a similar incidence of hemolysis and transfusion avoidance.
In PNH Study 302 (N=195), patients with PNH who were clinically stable after having been treated with eculizumab for at least 6 months were randomized to either Ultomiris or to continue eculizumab. The data showed similar results between the Ultomiris and eculizumab groups on clinical measures including hemolysis and transfusion avoidance.
Headache and upper respiratory tract infection were common adverse reactions associated with Ultomiris. A Boxed Warning in the prescribing information describes the risk of life-threatening meningococcal infections and sepsis. Patients should be immunized with meningococcal vaccines at least 2 weeks prior to receiving the first dose of Ultomiris, unless the risks of delaying treatment outweigh the risks of developing an infection.
Ultomiris will be available as a 300mg/30mL (10mg/mL) strength preservative-free solution in single-dose vials.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761108s000lbl.pdf
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