Kalydeco
ivacaftor
Table of contents
Overview
Kalydeco is a medicine which contains the active substance ivacaftor. It is used to treat cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs.
Kalydeco is used on its own to treat cystic fibrosis in patients aged 4 months and above who have one of the following mutations (changes) in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR): R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R.
Kalydeco is also used together with a medicine containing tezacaftor and ivacaftor to treat patients aged 6 years and above who have inherited the F508del mutation in the CFTR gene from both parents or who have inherited the F508del mutation plus one of the following mutations in CFTR: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272 26A→G, or 3849+10kbC→T.
Kalydeco is also used together with another medicine containing ivacaftor, tezacaftor and elexacaftor to treat patients aged 6 years and above who have at least one F508del mutation in the CFTR gene.
This product is no longer an orphan medicine. It was originally designated an orphan medicine on 8 July 2008. Kalydeco was withdrawn from the Community register of orphan medicinal products in July 2022 at the end of the 10-year period of market exclusivity.
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List item
Kalydeco : EPAR - Medicine overview (PDF/184.07 KB)
First published: 06/08/2012
Last updated: 26/01/2022
EMA/6151/2022 -
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List item
Kalydeco : EPAR - Risk-management-plan summary (PDF/142.78 KB)
First published: 03/02/2020
Last updated: 26/01/2022
Authorisation details
Product details | |
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Name |
Kalydeco
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Agency product number |
EMEA/H/C/002494
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Active substance |
ivacaftor
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International non-proprietary name (INN) or common name |
ivacaftor
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Therapeutic area (MeSH) |
Cystic Fibrosis
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Anatomical therapeutic chemical (ATC) code |
R07AX02
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Accelerated assessment |
This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment. |
Publication details | |
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Marketing-authorisation holder |
Vertex Pharmaceuticals (Ireland) Limited
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Revision |
35
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Date of issue of marketing authorisation valid throughout the European Union |
23/07/2012
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Contact address |
Unit 49, Block F2, Northwood Court, Santry |
Product information
27/04/2023 Kalydeco - EMEA/H/C/002494 - IB/0116/G
This medicine’s product information is available in all official EU languages.
Select ‘available languages’ to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.
Pharmacotherapeutic group
Therapeutic indication
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 (see sections 4.4 and 5.1).
- 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 F508del mutation in the CFTR gene (see section 5.1). Kalydeco granules are indicated for the treatment of infants aged at least 4 months, toddlers and children weighing 5 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 CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R (see sections 4.4 and 5.1).
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Assessment history
News
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 8-11 November 202112/11/2021
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26/03/2021
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18/09/2020
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24/07/2020
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30/04/2020
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 201918/10/2019
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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 15-18 October 201819/10/2018
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27/07/2018
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25/09/2015
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27/06/2014