首个囊性纤维化的固定剂量复方制剂ORKAMBI(lumacaftor/ivacaftor)获得欧盟的上市批准

欧盟委员会已授权营销 ORKAMBI(lumacaftor/ivacaftor)，这是第一个从根本原因出发来治疗治疗囊性纤维化(CF)的药，用于年龄在12岁以及12岁以上患有F508del突变两种变体的患者。而这种情况，这类病人在欧洲，有大约12000人。Vertex现在开始跨欧洲联盟 (欧盟)，逐个国家申报审批流程。
贝尔法斯特女王大学的生物医学与牙科医学院院长Stuart Elborn，医学博士，还是一位临床3期TRAFFIC研究的领头人，说，'对于囊性纤维化患者，这种病是终身的战斗，再加上多次因肺部感染住院，会变得逐渐更加严重。到目前为止，患有F508del 突变的两个变体的人仅有的对症状和对疾病并发症的治疗， Lumacaftor和ivacaftor的组合代表在囊性纤维化治疗的一步改变，它指出来这些病人这一疾病的根本原因。通过这样做，它显示了非凡的意义和源源不断的利益.
今天的批准基于先前宣布的两个24周全球临床3期的研究数据，TRAFFIC和TRANSPORT，并附加临时24周数据从随后扩展研究，取得了相应的进展。年龄在12岁以及12岁以上患有F508del 突变两种变体的患者，已经接受了标准治疗。在TRAFFIC和TRANSPORT的研究，招收超过 1100名患者，那些有lumacaftor和 ivacaftor结合治疗经验显著改善肺功能。病人也经历了身体质量指数(BMI)的显著改善和减少肺急性加重期 (急性肺部感染) 包括那些需要住院和静脉抗生素的使用的患者。
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用于12岁及以上

用于6岁到11岁儿童

生产厂家：Vertex Pharmaceuticals (Europe) Limited
Orkambi 200 mg/125mg film coated tablets(ivacaftor and lumacaftor)
New cystic fibrosis combination drug not recommended for NHS use
Orkambi (lumacaftor/ivacaftor), a new gene-specific medication for cystic fibrosis, has been rejected by NICE on cost-effectiveness grounds.
NICE concluded that Orkambi, a combination of 200mg lumacaftor and 125mg ivacaftor, "has the potential to ease the treatment burden of cystic fibrosis by reducing the number of pulmonary exacerbations", but described the drug's cost-effectiveness as "considerably higher than what is normally considered a cost-effective use of NHS resources", and has not recommended it for use across the NHS.
In May, Orkambi was turned down by the Scottish Medicines Consortium, which said the drug's manufacturer, Vertex, had not provided "a sufficiently robust clinical and economic analysis" for use of the treatment within NHS Scotland.
Vertex has now submitted a patient access scheme for Orkambi in England and plans to propose a similar scheme for Scotland. Talks are also underway with a view to making Orkambi available to patients in Northern Ireland and Wales.
Orkambi is indicated for the treatment of cystic fibrosis in patients from the age of 12 years who are homozygous for the F508del mutation in the gene encoding the CFTR chloride channel.
F508del is the most common causative cystic fibrosis mutation. Lumacaftor, a CFTR corrector, increases the amount of CFTR at cell surfaces, and ivacaftor, a CFTR potentiator, enhances gating of the channels.