Zolgensma美国FDA使用说明

通用名：Onasemnogene abeparvovec-xioi Suspension for intravenous infusion

商品名：Zolgensma

生产厂家：AveXis Inc.

美国上市日期：2019-05-24

适应症：

Zolgensma（onasemnogene abeparvovec-xioi）是一种基于腺相关病毒载体的基因治疗，用于治疗2岁以下患有脊髓性肌萎缩症（SMA）的儿童患者，其存活运动神经元1（SMN1）基因具有双等位基因突变。

使用限制：

（1）重复给药的安全性和有效性尚未确定。

（2）使用Zolgensma治疗晚期SMA患者（例如四肢完全瘫痪，永久性呼吸机依赖）尚未确定。

用法用量：

Zolgensma仅用于单剂量静脉输注。

（1）Zolgensma的推荐剂量为每kg体重给予1.1×10^14载体基因组。

（2）静脉输注Zolgensma超过60分钟。

（3）从给予Zolgensma的前一天起，每天给予全身皮质类固醇激素（等效于口服泼尼松龙1mg/kg）总共30天。在30天结束时，通过临床检查和实验室检查评估肝功。对于无明显异常的患者，在随后的28天将皮质类固醇逐渐减量。若持续肝功异常，继续给予全身皮质类固醇（等效于口服泼尼松龙1mg/kg/天）直至检测无明显异常，然后在随后的28天将皮质类固醇逐渐减量。若使用等效于口服泼尼松龙1mg/kg/天的剂量未获得足够缓解，需请专家会诊。

剂型规格：

（1）Zolgensma是一种静脉输注混悬液，单剂量瓶。

（2）Zolgensma以包含2至9个小瓶的试剂盒提供，作为2个小瓶填充体积（5.5mL或8.3mL）的组合。 所有样品瓶的标称浓度为2.0×10^13载体基因组（vg）/ mL。每瓶Zolgensma含有不少于5.5 mL或8.3 mL的可萃取体积。

禁忌症：无。

注意事项：

（1）血小板减少症：使用Zolgensma之前监测血小板计数。给药后第一个月每周监测一次，第二个月和第三个月每两周监测一次，直至血小板计数回到基线水平。

（2）肌钙蛋白-I升高：在输注Zolgensma之前监测肌钙蛋白-I，并且在第一个月每周监测一次，然后在第二个月和第三个月每月一次，直到肌钙蛋白-I恢复到基线水平。

不良反应：

最常见不良反应（发生率≥5%）为转氨酶升高和呕吐。

特殊人群用药：

儿科使用：不推荐在早产儿达到足月胎龄之前使用Zolgensma，因为伴随皮质类固醇治疗可能会对神经发育产生不利影响。延迟Zolgensma输注，直至达到足月孕龄。

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

· Liver enzymes could become elevated and cause acute serious liver injury in children who receive ZOLGENSMA.

· Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

· Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

· Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.

· Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

What do I need to know about vaccinations and ZOLGENSMA?

· Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.

· Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for up to 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.