美国为Dravet综合征提供了新的治疗方案

2018-08-26 MedSci MedSci原创

美国监管机构已批准Biocodex的Diacomit用于治疗与Dravet综合征(DS)相关的癫痫发作,这是一种罕见且难以治疗的癫痫形式。该决定允许医生给服用氯巴嗪的两岁以上的病人开这种药。批准是基于两项多中心安慰剂对照试验,STICLO France和STICLO Italy,这两项试验的主要疗效终点都是应答率,定义为在全身性阵挛或强直性阵挛发作频率(每30天)下降超过50%的患者数量。在STI



美国监管机构已批准Biocodex的Diacomit用于治疗与Dravet综合征(DS)相关的癫痫发作,这是一种罕见且难以治疗的癫痫形式。

该决定允许医生给服用氯巴嗪的两岁以上的病人开这种药。

批准是基于两项多中心安慰剂对照试验,STICLO France和STICLO Italy,这两项试验的主要疗效终点都是应答率,定义为在全身性阵挛或强直性阵挛发作频率(每30天)下降超过50%的患者数量。

在STICLO France,接受Diacomit(stiripentol)的患者的应答率为71%,而接受安慰剂的患者为5%。

在STICLO Italy,接受该药物治疗的患者的应答率为67%,而接受安慰剂的患者为9.1%。
最常见的不良反应发生在至少10%的Diacomit治疗患者中且比安慰剂更频繁,包括嗜睡(67%),食欲下降(45%),激动(27%),共济失调(27%), 体重下降(27%),肌张力减退(24%),恶心(15%),震颤(15%),构音障碍(12%)和失眠(12%)。

Dravet综合征,也被称为严重的婴儿期肌阵挛性癫痫(SMEI),是一种灾难性的早期癫痫综合征,据认为在美国大约有2000到8000名患者出现这种症状。


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