Kalydeco

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ivacaftor

Authorised
This medicine is authorised for use in the European Union.

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 Kalydeco was withdrawn from the Community register of orphan medicinal products in July 2022 at the end of the 10-year period of market exclusivity.

This EPAR was last updated on 28/04/2023

Authorisation details

Product details
Name
Kalydeco
Agency product number
EMEA/H/C/002494
Active substance
ivacaftor
International non-proprietary name (INN) or common name
ivacaftor
Therapeutic area (MeSH)
Cystic Fibrosis
Anatomical therapeutic chemical (ATC) code
R07AX02
Accelerated assessmentAccelerated 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
Marketing-authorisation holder
Vertex Pharmaceuticals (Ireland) Limited
Revision
35
Date of issue of marketing authorisation valid throughout the European Union
23/07/2012
Contact address

Unit 49, Block F2, Northwood Court, Santry
Dublin 9, D09 T665
Ireland

Product information

27/04/2023 Kalydeco - EMEA/H/C/002494 - IB/0116/G

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Product information documents contain:

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

Other respiratory system products

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 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 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).

 

Assessment history

Changes since initial authorisation of medicine

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