Evrysdi治疗脊髓性肌萎缩症(SMA),CHMP持积极意见

2021-02-27 Allan MedSci原创

SMA是导致婴儿死亡的主要遗传原因,而5q SMA是该疾病的最常见形式。

罗氏公司近日宣布,欧洲药品管理局(EMA)人用药品委员会(CHMP)已建议批准Evrysdi™(risdiplam)用于治疗5q脊髓性肌萎缩症(SMA)2个月及以上患者,这些患者患有1型、2型或3型或一到四个SMN2副本的SMA。

SMA是导致婴儿死亡的主要遗传原因,而5q SMA是该疾病的最常见形式。SMA会导致进行性肌肉无力和萎缩。目前,大量的医疗需求未得到满足。控制肌肉的运动神经里的某种蛋白质(运动神经元存活因子,SMN)出了问题,使得神经讯号的传递受到阻碍,造成肌肉的收缩失常。控制这种蛋白质形成的基因,称为SMN1,当这种基因出现问题时,就会产生SMA。

CHMP建议是基于FIREFISH临床研究得出的数据。在FIREFISH中,根据Bayley量表的总运动量表评估,接受Evrysdi治疗12个月的婴儿中有29%(12/41)能够在无支撑的情况下坐立至少五秒钟。此外,93%的患者存活。

 

原始出处:

https://www.firstwordpharma.com/node/1804490?tsid=4

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    2021-08-19 chendoc252
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    2021-03-01 科研科研科研

    Chmp支持治疗脊髓性肌肉

    0

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    2021-03-01 医生2394
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    2021-03-01 江川靖瑶
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