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欧盟委员会批准Vertex Pharmaceuticals的Alyftrek（Deutivacaftor/Tezacaftor/Vanzacaftor）用于6岁及以上囊性纤维化患者，且囊性纤维化跨膜电导调节基因中至少有一个非I类突变
2025-07-01 19:54
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- ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene, making it the broadest label for this medicine in the world.
- In head-to-head clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior on ppFEV1 and superior compared to KAFTRIO (ivacaftor/tezacaftor/elexacaftor) at reducing sweat chloride, demonstrating greater improvement in CFTR function.
- Approximately 31,000 people with CF in the EU are now eligible for this new highly effective modulator therapy.
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