Orkambi:欧盟批准的2-5岁儿童囊性纤维化治疗药物

2019-01-26 MedSci MedSci原创

欧盟委员会批准囊性纤维化(CF)药物Orkambi用于治疗2-5岁的儿童。此次批准使Orkambi成为欧洲第一个治疗CF儿童患者的药物,目前这一类患者人数约为1500。

欧盟委员会批准囊性纤维化(CF)药物Orkambi用于治疗2-5岁的儿童。此次批准使Orkambi成为欧洲第一个治疗CF儿童患者的药物,目前这一类患者人数约为1500。

Orkambi是lumacaftor和ivacaftor的组合,它们均靶向CF患者体内缺乏的CFTR蛋白发挥治疗作用。

囊性纤维化会导致寿命减短,平均死亡年龄为20多岁。儿童患者的早期治疗就是希望可以延长生命,但是该公司尚未出具这种长期数据。本次儿童的临床试验则是侧重于通过汗液氯化物水平变化测量安全性和有效性。

该批准基于第3阶段数据,其中Orkambi在60名年龄为2-5岁的患者中一般耐受24周。安全性也与6岁及以上患者的安全性一致。

英国有大约10000名CF患者,是世界上拥有第二大CF患者的国家。对于Vertex公司来说这是一个很大的市场,但它没有提供进一步的折扣。据了解,Vertex已经向NICE提供了近90%的折扣,将Orkambi的价格从每位患者约100000英镑降至每位患者约14000英镑。

原始出处:

http://www.pmlive.com/pharma_news/vertex_gains_eu_approval_for_earlier_orkambi_use_1275863

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    2019-08-20 wolongzxh
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