Kalydeco

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ivacaftor

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

Overview

Kalydeco is a medicine 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 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 12 years and above who have inherited the F508del mutation from both parents or have inherited the F508del mutation plus another mutation called a ‘minimal function mutation’.

Kalydeco contains the active substance ivacaftor.

Cystic fibrosis is rare, and Kalydeco was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 8 July 2008.

This EPAR was last updated on 22/01/2021

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.

OrphanOrphan

This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Vertex Pharmaceuticals (Ireland) Limited
Revision
27
Date of issue of marketing authorisation valid throughout the European Union
23/07/2012
Contact address

28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland

Product information

25/11/2020 Kalydeco - EMEA/H/C/002494 - X/0083/G

Contents

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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.
  • 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 and adolescents aged 12 years and older with cystic fibrosis (CF) who are homozygous for the F508del mutation in the CFTR gene or heterozygous for F508del and have a minimal function (MF) mutation in the CFTR gene.

Assessment history

Changes since initial authorisation of medicine

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