FDA批准口服性脊髓性肌萎缩症(SMA)药物Evrysdi

2020-08-09 Allan MedSci原创

近日,FDA批准了Roche及其合作伙伴PTC Therapeutics的Evrysdi(risdiplam),使之成为首个针对两个月及两个月以上所有类型脊髓性肌萎缩症(SMA)患者的口服治疗药物。

近日,FDA批准了Roche及其合作伙伴PTC Therapeutics的Evrysdi(risdiplam),使之成为首个针对两个月及两个月以上所有类型脊髓性肌萎缩症(SMA)患者的口服治疗药物。Evrysdi(risdiplam)是一款SMN2拼接修饰剂,也称为RG7916,将由罗氏Genentech部门在美国销售。

FDA药物评估和研究中心的神经科学办公室主任Billy Dunn说道:“Evrysdi是首款治疗该疾病的口服药物,它为SMA患者提供了重要的治疗选择”。Biogen和Ionis Pharmaceuticals的Spinraza(nusinersen)需每四个月输注到椎管中,已于2016年获得美国监管机构的批准,而诺华的基因疗法Zolgensma(onasemnogene abeparvovec-xioi)去年已被批准用于SMA患者。

FDA关于Evrysdi的决定是基于FIREFISH和SUNFISH研究的结果。今年1月,罗氏(Roche)报告说,已针对1至7个月的1型SMA婴儿进行了FIREFISH试验。研究的第1部分评估了几种Evrysdi剂量,并确定第2部分的治疗剂量为0.2 mg / kg。该研究的第1部分结果表明,按照贝利婴幼儿发育量表第三版(BSID-III)的测量,接受治疗的婴儿中有41%能够获得至少五秒钟的无支撑坐姿的能力。此外,90%的婴儿在没有永久通气的情况下还活着,并且至少达到了15个月大;而81%的婴儿在经过至少23个月的治疗并达到28个月或更大的年龄后没有进行永久通气。

最近,罗氏(Roche)报告了FIREFISH第2部分的数据,该部分也达到了其主要目标,根据BSID-III的评估,到12个月时,有29%的婴儿能够在没有支撑的情况下能够保持坐姿5秒钟。

 

原始出处:

https://www.firstwordpharma.com/node/1747563?tsid=4

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    2021-07-09 chendoc252
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    2020-11-29 bugit
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    2020-08-11 江川靖瑶
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    2020-08-09 长毛小米

    学习

    0

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    2020-08-09 12543f43m99暂无昵称

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