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. Cystic fibrosis affects the cells that produce mucus and digestive juices. As a result, these secretions become thick and cause blockage. Build-up of thick and sticky secretions in the lungs causes inflammation and long-term infection. In the gut, blockage of the tubes from the pancreas slows down the digestion of food and causes poor growth.

Kalydeco is used on its own to treat cystic fibrosis in patients aged 1 year and above who have one of nine mutations (changes) in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR). The mutations are: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R (also known as gating mutations).

Kalydeco is also used on its own to treat patients with cystic fibrosis aged 18 years and above who have the R117H mutation in the CFTR gene.

Kalydeco is also used together with another cystic fibrosis medicine containing the active substances tezacaftor and ivacaftor to treat patients with cystic fibrosis aged 12 years and above who have a mutation called F508del in the CFTR gene. The two medicines are used in patients who have inherited the F508del mutation from both parents and therefore have the mutation in both copies of the CFTR gene. They are also used in patients who have inherited the F508del mutation from one parent and also have 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 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. Further information on the orphan designation.

This EPAR was last updated on 14/10/2019

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
19
Date of issue of marketing authorisation valid throughout the European Union
22/07/2012
Contact address

28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland

Product information

26/04/2019 Kalydeco - EMEA/H/C/002494 - II/0076

Contents

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Pharmacotherapeutic group

Other respiratory system procucts

Therapeutic indication

Kalydeco tablets are indicated for the treatment of patients with cystic fibrosis (CF) aged 6 years and older and weighing 25 kg or more who have one of the following gating (class III) mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

Kalydeco tablets are also indicated for the treatment of patients with cystic fibrosis (CF) aged 18 years and older who have an R117H mutation in the CFTR gene.

Kalydeco tablets are also indicated in a combination regimen with tezacaftor 100 mg/ivacaftor 150 mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older 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.

Kalydeco granules are indicated for the treatment of children with cystic fibrosis (CF) aged  12 months and older and weighing 7 kg to less than 25 kg who have 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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