Orkambi

RSS

lumacaftor / ivacaftor

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

Overview

Orkambi is a medicine used to treat cystic fibrosis in patients aged 2 years and above. Cystic fibrosis is an inherited disease that has severe effects on the lungs and the digestive system. It affects the cells that produce mucus and digestive juices. In cystic fibrosis, the 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.

Orkambi is used in patients who have a genetic mutation (change) called the F508del mutation. This mutation affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR) which is involved in regulating the production of mucus and digestive juices. Orkambi is used in patients who have inherited the mutation from both parents.

Orkambi contains the active substances lumacaftor and ivacaftor.

This EPAR was last updated on 29/07/2019

Authorisation details

Product details
Name
Orkambi
Agency product number
EMEA/H/C/003954
Active substance
  • Lumacaftor
  • ivacaftor
International non-proprietary name (INN) or common name
lumacaftor / ivacaftor
Therapeutic area (MeSH)
Cystic Fibrosis
Anatomical therapeutic chemical (ATC) code
R07AX30
Additional monitoringAdditional 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.

Publication details
Marketing-authorisation holder
Vertex Pharmaceuticals (Ireland) Limited
Revision
18
Date of issue of marketing authorisation valid throughout the European Union
18/11/2015
Contact address

28-32 Pembroke Street Upper
Dublin 2
D02 EK84
Ireland

Product information

28/05/2019 Orkambi - EMEA/H/C/003954 - IB/0048

Contents

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Pharmacotherapeutic group

OTHER RESPIRATORY SYSTEM PRODUCTS

Therapeutic indication

Orkambi tablets are indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who are homozygous for the F508del mutation in the CFTR gene.

Orkambi granules are indicated for the treatment of cystic fibrosis (CF) in children aged 2 years and older who are homozygous for the F508del mutation in the CFTR gene.

Assessment history

Changes since initial authorisation of medicine

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