Kalydeco - withdrawal of application for variation to marketing authorisation
Application withdrawn
The application for a change to this medicine's authorisation has been withdrawn
ivacaftor
Post-authorisationHuman
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 1 month and above who weigh at least 3 kg and who have certain mutations (changes) in the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR).
- together with a medicine containing tezacaftor/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 from one parent and have certain other mutations in the CFTR gene;
- together with another medicine containing ivacaftor/tezacaftor/elexacaftor to treat patients aged 2 years and above who have at least one non-class I mutation in the CFTR gene.
Kalydeco contains the active substance ivacaftor. It is available as tablets and as granules to be taken by mouth and has been authorised in the EU since July 2012.
The company applied to extend the use of Kalydeco to children aged 1 year and older with cystic fibrosis who have at least one non-class I mutation in the CFTR gene, in combination with ivacaftor/ tezacaftor/elexacaftor.
Cystic fibrosis is caused by mutations in the CFTR gene. This gene makes the CFTR protein, which works on the surface of cells to regulate the production of mucus in the lungs and digestive juices in the gut. The mutations reduce the number of CFTR proteins on the cell surface or affect the way the protein works, resulting in mucus and digestive fluids being too thick. This in turn leads to blockages, inflammation, increased risk of lung infections and poor digestion and growth.
The active substance in Kalydeco, ivacaftor, improves the activity of the defective CFTR protein. This makes mucus and digestive juices less thick, thereby helping to relieve symptoms of the disease.
Available data on the effectiveness and safety of ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor in older children and adults were presented to support the medicine use in younger children. The company also provided clinical data from a study in children aged between 1 and 2 years with cystic fibrosis, which looked at how these active substances are absorbed, modified and removed from the body, as well as its effects and safety.
The application was withdrawn after the European Medicines Agency had evaluated the initial information from the company and had prepared questions for the company. The company had not responded to the questions at the time of the withdrawal.
As the Agency was still evaluating the initial information from the company, it had not yet made any recommendations.
In its letter notifying the Agency of the withdrawal, the company stated that it withdrew its application due to the Agency’s preliminary feedback that additional information would be required to support this extension of indication.
The company informed the Agency that there are no consequences for patients in clinical trials using Kalydeco.
If your child is in a clinical trial and you need more information about their treatment, speak with their clinical trial doctor.
There are no consequences on the use of Kalydeco in its authorised uses.