CHMP对Sarepta的DMD治疗持负面看法

2018-05-05 MedSci MedSci原创

Sarepta表示,在欧洲批准的杜氏肌营养不良疗法可能会有延迟,这表明这表明欧洲药品管理局人用药品委员会(CHMP)的投票结果是否定的。"根据与CHMP代表的讨论,我们了解到CHMP没有得出eteplirsen对于51号外显子患者无效的结论,而是Sarepta尚未达到有条件批准的监管门槛,部分原因是由于在研究中使用外部控制作为比较对象。"Sarepta总裁兼首席执行官Doug Ingram说。Sa


Sarepta表示,在欧洲批准的杜氏肌营养不良疗法可能会有延迟,这表明这表明欧洲药品管理局人用药品委员会(CHMP)的投票结果是否定的。

"根据与CHMP代表的讨论,我们了解到CHMP没有得出eteplirsen对于51号外显子患者无效的结论,而是Sarepta尚未达到有条件批准的监管门槛,部分原因是由于在研究中使用外部控制作为比较对象。"Sarepta总裁兼首席执行官Doug Ingram说。

Sarepta现在计划提交复审申请,并要求召集由DMD和神经肌肉专家组成的科学咨询小组(SAG),以"提供专家指导和洞察力,尤其是外部控制的有效性和减缓DMD患者肺部衰退的重要性。"他补充说。

Exondys 51通过产生功能性肌营养不良蛋白来解决DMD的根本原因,并且临床研究已经证明了广泛可靠的安全性和耐受性以及该药物的功效。

该注射针对患有确定的肌营养不良蛋白基因突变的患者适用于51外显子跳跃,其影响约13%的DMD群体。

2016年,美国监管机构发布了有条件批准的治疗后,认为Sarepta提交的数据表明肌营养不良蛋白的产量增加,有可能预测一些DMD患者的肌营养不良蛋白基因突变可承受外显子51跳跃。

但它当时也强调临床效益尚未确定,因此要求该公司进行进一步的临床试验以将有条件批准转换为完整临床试验。


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    2018-11-18 gaoxiaoe
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    2018-05-07 医生2394
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    2018-05-07 紫砂壶
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    2018-05-05 明月清辉

    谢谢分享.学习了

    0

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    2018-05-05 1ddf0692m34(暂无匿称)

    学习了.长知识

    0

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