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Spinraza injection 12mg/5ml(Nusinersen 诺西那生钠注射液)
药店国别  
产地国家 德国 
处 方 药: 是 
所属类别 12毫克/5毫升/瓶 
包装规格 12毫克/5毫升/瓶 
计价单位: 瓶 
生产厂家中文参考译名:
百健公司
生产厂家英文名:
Biogen Inc
该药品相关信息网址1:
https://www.drugs.com/history/spinraza.html
该药品相关信息网址2:
该药品相关信息网址3:
原产地英文商品名:
SPINRAZA Injection 12mg/5ml/via
原产地英文药品名:
nusinersen
中文参考商品译名:
SPINRAZA鞘内注射剂 12毫克/5毫升/瓶
中文参考药品译名:
nusinersen
曾用名:
简介:

 

部份中文诺西那生钠处方资料(仅供参考)
英文名:nusinersen
商品名:Spinraza
中文名:诺西那生钠注射液
生产商:百健制药
药品简介
2017年6月4日,脊髓性肌萎缩症治疗药Spinraza(Nusinersen)在欧盟获准上市,用于5q脊髓性肌萎缩症(5q SMA)的治疗,该药物也成为在欧洲首个批准上市用于治疗该疾病的药物。
脊髓性肌萎缩症 (SMA)是指一类由于以脊髓前角细胞为主的变性导致肌无力和肌萎缩的遗传性疾病,大部分为隐性遗传。5q-SMA是一种常染色体隐性遗传疾病,是由5号染色体上的SMN1(运动神经元存活基因)突变所引起的,5q SMA是SMA的最常见类型,约占全部SMA病例的95%。
作用机理
Nusinersen是一种反义寡核苷酸(ASO),可通过结合至内含子7内含子的内含子剪接沉默位点(ISS-N1),增加生存运动神经元2(SMN2)信使核糖核酸(mRNA)转录本中外显子7包含的比例。SMN2前信使核糖核酸(pre-mRNA)。通过绑定,ASO取代了通常会抑制剪接的剪接因子。这些因素的置换导致外显子7保留在SMN2 mRNA中,因此,当产生SMN2 mRNA时,可以将其翻译为功能性全长SMN蛋白。
SMA是一种进行性神经肌肉疾病,由SMN1基因的5q染色体突变引起。位于SMN1附近的第二个基因SMN2负责少量SMN蛋白的产生。SMA是一种临床疾病,其疾病严重程度与SMN2基因拷贝数少和症状发作年龄小有关。
适应症
Spinraza适用于治疗5q脊髓性肌萎缩症。
用法与用量
Spinraza用于腰椎穿刺的鞘内使用。
推荐剂量为每次给药12毫克(5毫升)。
确诊后应尽早开始于0、14、28和63天服用4种负荷剂量的Spinraza治疗。此后每4个月给予一次维持剂量。
治疗时间
没有关于该药物长期疗效的信息。应定期检查是否需要继续治疗,并应根据患者的临床表现和对治疗的反应,个别考虑。
剂量遗漏或延迟
如果延迟或错过了负荷剂量,应尽快服用Spinraza,两次给药之间至少间隔14天,并以规定的频率继续给药。如果维持剂量延迟或错过,应尽快给予Spinraza,并每4个月继续给药。
特殊人群
肾功能不全
尚未对患有肾功能不全的患者研究Spinraza。肾功能不全患者的安全性和疗效尚未确定,应密切观察。
肝功能不全
尚未对患有肝功能不全的患者进行Spinraza的研究。肝中的Spinraza不会通过细胞色素P450酶系统代谢,因此对于肝功能不全的患者不太可能需要调整剂量。
给药方法
治疗应由具有执行腰椎穿刺经验的医疗保健专业人员进行。
使用脊髓麻醉针在1到3分钟内以鞘内推注方式施用Spinraza。不得在有感染或发炎迹象的皮肤区域内使用注射剂。建议在服用Spinraza之前,先清除相当于要注射的Spinraza体积的脑脊髓液(CSF)。
如患者的临床状况所指示,可能需要镇静剂来施用Spinraza。
可以考虑使用超声波(或其他成像技术)指导鞘内注射Spinraza,特别是在年轻患者和脊柱侧弯患者中。制备和使用Spinraza时应使用无菌技术;请参阅使用说明。
禁忌症
对活性物质或第6.1节中所列的任何赋形剂过敏。
保质期
4年
特殊的储存注意事项
存放在冰箱(2°C-8°C)中。
不要冻结。
将小瓶放在外部纸箱中,以避光。
如果没有可用的制冷,则Spinraza可能会保存在其原始纸箱中,并在30°C或低于30°C的光线下保护长达14天。
给药前,如有必要,可将未打开的Spinraza小瓶取出并放回冰箱。如果将其从原始纸箱中取出,则在不超过25°C的温度下,制冷的总合并时间不应超过30小时。
完整说明资料附件:
https://www.medicines.org.uk/emc/product/2715/smpc
---------------------------------------------------
SPINRAZA(Nusinersen)Approved in the European Union as First Treatment for Spinal Muscular Atrophy
The European Commission(EC) has granted a marketing authorization for SPINRAZA® (nusinersen) for the treatment of 5q spinal muscular atrophy (SMA), Biogen (BIIB) announced today.15q SMA is the most common form of the disease and represents approximately 95% of all SMA cases.2 SPINRAZA is the first approved treatment in the European Union (EU) for SMA, a leading genetic cause of death in infants that is marked by progressive, debilitating muscle weakness. SPINRAZA was reviewed under the European Medicines Agency’s(EMA) accelerated assessment program, intended to expedite access to patients with unmet medical needs.
“Today we join individuals and families affected by SMA across Europe in celebrating the approval of SPINRAZA. Based on the robust efficacy and safety profile demonstrated in the clinical trials, we believe SPINRAZA will have a meaningful impact on infants, children and adults living with this devastating disease,” said Michel Vounatsos, chief executive officer at Biogen. “As part of our mission to improve the lives of those affected by SMA, we remain steadfast in our commitment to work with healthcare professionals, advocacy groups and government agencies to ensure people who could benefit from SPINRAZA receive access to this important treatment as quickly as possible.”
The approval of SPINRAZA is primarily based on results from two pivotal multicenter, controlled studies, including end of study data from ENDEAR (infantile-onset SMA) and an interim analysis of CHERISH (later-onset SMA), both of which demonstrated the clinically meaningful efficacy and favorable benefit-risk profile of SPINRAZA. The approval was also supported by open-label data in pre-symptomatic and symptomatic individuals with, or likely to develop, Types 1, 2 and 3 SMA.
In the ENDEAR end of study analysis, a statistically significant greater percentage of patients achieved the definition of motor milestone responder in the SPINRAZA group (51%) compared to the sham-control group (0%)(p<0.0001). Some infants in the SPINRAZA group achieved motor milestones including full head control, ability to roll, sitting, and standing. Additionally, infants treated with SPINRAZA demonstrated a statistically significant reduction (47%) in the risk of death or permanent ventilation (p=0.0046).
In the CHERISH pre-specified interim analysis, there was a statistically significant and clinically meaningful improvement in motor function in children with later-onset SMA (most likely to develop Type 2 or Type 3) treated with SPINRAZA compared to untreated children.Improvements were measured by the Hammersmith Functional Motor Scale Expanded(HFMSE) and demonstrated a treatment difference of 5.9 points in the mean change from baseline to Month 15 in the HFMSE score (p=0.0000002). The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA. The Phase 3 end of study data were consis 

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