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 2 years 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.

This EPAR was last updated on 08/11/2018

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
Additional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.

Orphan

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 (Europe) Ltd
Revision
15
Date of issue of marketing authorisation valid throughout the European Union
22/07/2012
Contact address
2 Kingdom Street
London W2 6BD
United Kingdom

Product information

09/10/2018 Kalydeco - EMEA/H/C/002494 - II/0063/G

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

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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 2 years and older and weighing 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.

Assessment history

Changes since initial authorisation of medicine

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