Ultomiris 100mg/mL高浓度制剂获美国FDA批准，用于治疗PNH和aHUS大幅缩短治疗时间
Ultomiris（ravulizumab-cwvz）100mg/mL制剂：
（1）治疗阵发性夜间血红蛋白尿（PNH）成人患者；
（2）治疗非典型溶血性尿毒综合征（aHUS）成人和儿童（≥1个月）患者，以抑制补体介导的血栓性微血管病（TMA）。
Ultomiris 100mg/mL制剂代表了aHUS和PNH患者临床治疗经验方面的一个进步，因为与Ultomiris 10mg/mL制剂相比，Ultomiris 100mg/mL制剂可减少约60%的平均年输液时间，同时具有可比的安全性和有效性。使用Ultomiris 100mg/mL制剂，大多数患者每年接受治疗的时间不超过6小时。
目前，Ultomiris 100mg/mL制剂也正在接受欧盟和日本监管机构的审查。就在最近，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）发布了建议批准Ultomiris 100mg/mL制剂的积极意见，预计这款产品将在11月获得批准。
Alexion公司计划在几天内将Ultomiris 100mg/mL制剂推向市场，并制定全面的培训计划。Ultomiris 10mg/mL制剂将继续供应至2021年年中。届时，在与美国FDA沟通后，Alexion公司将把Ultomiris 10mg/mL制剂撤出市场。过渡期旨在提供无缝转换，而不会中断患者的输液计划。
PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.
Atypical HUS can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Affecting both adults and children, atypical HUS patients can present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit.
The prognosis of both aHUS and PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.[1]
references:
[1]retrieved 12 Oct. 2020 from: https://www.businesswire.com/news/home/20201012005090/en/
（1）治疗阵发性夜间血红蛋白尿（PNH）成人患者；
（2）治疗非典型溶血性尿毒综合征（aHUS）成人和儿童（≥1个月）患者，以抑制补体介导的血栓性微血管病（TMA）。
Ultomiris 100mg/mL制剂代表了aHUS和PNH患者临床治疗经验方面的一个进步，因为与Ultomiris 10mg/mL制剂相比，Ultomiris 100mg/mL制剂可减少约60%的平均年输液时间，同时具有可比的安全性和有效性。使用Ultomiris 100mg/mL制剂，大多数患者每年接受治疗的时间不超过6小时。
目前，Ultomiris 100mg/mL制剂也正在接受欧盟和日本监管机构的审查。就在最近，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）发布了建议批准Ultomiris 100mg/mL制剂的积极意见，预计这款产品将在11月获得批准。
Alexion公司计划在几天内将Ultomiris 100mg/mL制剂推向市场，并制定全面的培训计划。Ultomiris 10mg/mL制剂将继续供应至2021年年中。届时，在与美国FDA沟通后，Alexion公司将把Ultomiris 10mg/mL制剂撤出市场。过渡期旨在提供无缝转换，而不会中断患者的输液计划。
PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death.
Atypical HUS can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Affecting both adults and children, atypical HUS patients can present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit.
The prognosis of both aHUS and PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.[1]
references:
[1]retrieved 12 Oct. 2020 from: https://www.businesswire.com/news/home/20201012005090/en/
