法规知识库 · EMA 集中审评
Kalydeco
ivacaftor · EMEA/H/C/002494 · 已授权
获批上市许可
2012-07-23
欧委会决定日
2025-04-04
持有人 / 申请人
Vertex Pharmaceuticals (Ireland) Limited
终止日期字段
无
从产品到官方文件
文件记录
34
官方索引 34
EPAR / 审评
32
assessment / overview 等
产品信息
2
PI / presentations
其他
0
RMP / PIP 等
ATC：R07AX02
治疗领域：Cystic Fibrosis
药理分类：Other respiratory system products
修订号：46 · 最新影响 PI 的程序：WS2551
适应症（原文）：Kalydeco tablets are indicated: - As monotherapy for the treatment of adults, adolescents, and children aged 6 years and older and weighing 25 kg or more with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. - In a combination regimen with tezacaftor/ivacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, and 3849+10kbC→T. - In a combination regimen with ivacaftor/tezacaftor/elexacaftor tablets for the treatment of adults, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one non-Class I mutation in the CFTR gene. Kalydeco granules are indicated: - As monotherapy for the treatment of infants aged at least 1 month, toddlers and children weighing 3 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or one of the following gating (Class III) mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. - In a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one non-Class I mutation in the CFTR gene.
关键日期时间轴
来自 EMA medicines JSON 日期字段；缺失字段不展示，不补推结论。
2025-04-04 · 欧委会决定
2012-07-23 · 获批上市许可
2012-05-24 · CHMP 意见通过
说明书、EPAR 与其他文件
仅展示 EMA 官方 document_url；本站不下载或镜像 PDF。
EPAR / 审评资料
- 打开官方文件 ↗
Kalydeco : EPAR - Procedural steps taken and scientific information after authorisation
procedural-steps-after · 2025-08-04
- 打开官方文件 ↗
Kalydeco : EPAR - Statement indicating compliance with the agreed completed paediatric investigation plan
pip-compliance · 2025-08-04 · EMADOC-1700519818-2240068
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Kalydeco : EPAR - Procedural steps taken and scientific information after authorisation (archive)
procedural-steps-after · 2025-08-04
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Kalydeco-H-C-002494-WS-2551 : EPAR - Assessment report - Variation
variation-report · 2025-07-02 · EMA/96436/2025
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Kalydeco : EPAR - Risk management plan
rmp · 2025-07-02
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Kalydeco : EPAR - Medicine overview
overview · 2025-07-02
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Kalydeco-H-C-002494-X-0115-G : EPAR - Assessment report - Variation
variation-report · 2024-05-16 · EMA/110324/2024
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Kalydeco-H-C-PSUSA-00009204-202301 : EPAR - Scientific conclusions and grounds for the variation to the terms of the marketing authorisation
scientific-conclusion · 2024-03-05 · EMA/97030/2024
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Kalydeco-H-C-002494-X-0114-G : EPAR - Assessment Report - Extension
variation-report · 2024-03-04 · EMA/443706/2023
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Kalydeco : Orphan maintenance assessment report (post-authorisation) (II-0089)
orphan-maintenance-report-post · 2022-08-10 · EMA/OD/0000042076
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Kalydeco : Orphan maintenance assessment report (post-authorisation) (II-0085)
orphan-maintenance-report-post · 2022-08-10 · EMA/OD/0000036247
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Kalydeco : Orphan maintenance assessment report (post-authorisation) (II-0063)
orphan-maintenance-report-post · 2022-08-10 · EMA/716663/2018
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Kalydeco-H-C-2494-II-0096 : EPAR - Assessment Report - Variation
variation-report · 2022-01-26 · EMA/714293/2021
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Kalydeco-H-C-2494-II-0089: EPAR - Assessment Report - Variation
variation-report · 2021-05-12 · EMA/206661/2021
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Kalydeco-H-C-2494-X-83-G: EPAR - Assessment Report - Variation
variation-report · 2021-01-22 · EMA/628136/2020
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Kalydeco-H-C-2494-II-0027 : EPAR - Assessment Report - Variation
variation-report · 2020-12-16 · EMA/670896/2020 Corr.1
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Kalydeco-H-C-2494-X-0034-G : EPAR - Assessment Report - Extension
variation-report · 2020-12-16 · EMA/733450/2015 Corr.1
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Kalydeco-H-C-2494-P46-021.1 : EPAR - Assessment Report
variation-report · 2020-12-16 · EMA/534823/2015 Corr.1
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Kalydeco-H-C-2494-P46-0022 : EPAR - Assessment Report
variation-report · 2020-12-16 · EMA/CHMP/136348/2015 Corr.1
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Kalydeco-H-C-2494-II-0009 : EPAR - Assessment Report - Variation
variation-report · 2020-12-16 · EMA/CHMP/360053/2014 Corr.1
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Kalydeco-H-C-2494-II-0086 : EPAR - Assessment Report - Variation
variation-report · 2020-12-09 · EMA/530990/2020
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Kalydeco-H-C-2494-II-0085 : EPAR - Assessment Report - Variation
variation-report · 2020-10-26 · EMA/563387/2020
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Kalydeco-H-C-2494-II-0082 : EPAR - Assessment Report - Variation
variation-report · 2020-09-14 · EMA/297262/2020
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Kalydeco-H-C-2494-P46-029 : EPAR - Assessment Report
variation-report · 2020-06-22 · EMA/273575/2020
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Kalydeco-H-C-2494-P46-028 : EPAR - Assessment Report
variation-report · 2020-06-12 · EMA/694594/2019
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Kalydeco-H-C-2494-X-0075-G : EPAR - Assessment Report - Extension
variation-report · 2020-02-03 · EMA/CHMP/613823/2019
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Kalydeco-H-C-2494-P46-026 and Symkevi-H-C-4682-P46-005 : EPAR - Assessment Report
variation-report · 2019-11-14 · EMA/CHMP/401041/2019
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Kalydeco-H-C-2494-P46-0027 and Symkevi-H-C-4682-P46-006 : EPAR - Assessment Report
variation-report · 2019-10-14 · EMA/CHMP/411016/2019
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Kalydeco-H-C-2494-II-0069 : EPAR - Assessment Report - Variation
variation-report · 2019-01-03 · EMA/CHMP/799967/2018
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Kalydeco-H-C-2494-II-0063-G : EPAR - Assessment Report - Variation
variation-report · 2018-10-30 · EMA/636932/2018
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Kalydeco-H-C-2494-PSUV-0008 : EPAR- Scientific conclusions and grounds recommending the variation to the terms of the marketing authorisation
scientific-conclusion · 2014-04-07 · EMA/55354/2014
- 打开官方文件 ↗
Kalydeco : EPAR - Public assessment report
assessment-report · 2012-08-06 · EMA/473279/2012
数据来自 EMA 官方 JSON；最终以 EMA / 欧委会文件为准。本页不自动给出安全或非安全结论。