Kalydeco
Authorised
This medicine is authorised for use in the European Union
ivacaftor
MedicineHumanAuthorised
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 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 whose disease is due to at least one F508del mutation in the CFTR gene.
More information on the specific CTFR mutations for the different treatments is available in the package leaflet.
Kalydeco contains the active substance ivacaftor.
Kalydeco can only be obtained with a prescription. It should only be prescribed by a doctor with experience in the treatment of cystic fibrosis, and only in patients with certain confirmed CTFR mutations.
Kalydeco is available as tablets and as granules in a sachet, both of which come in different strengths.
The dose and formulation depend on the patient’s age and body weight.
The dose of Kalydeco may need to be adjusted if the patient is also taking a type of medicine called a ‘moderate or strong CYP3A inhibitor’, such as certain antibiotics or medicines for fungal infections, and
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, which 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.
Kalydeco on its own
Kalydeco was shown to be effective at improving lung function in 4 main studies involving patients with cystic fibrosis who had various mutations. The main measure of effectiveness in these studies was based on improvements in patients’ FEV1. FEV1 is the maximum amount of air a person can breathe out in one second and is a measure of how well the lungs work. In the studies, Kalydeco was compared with placebo (a dummy treatment).
Two of the studies involved 219 patients with cystic fibrosis who had the G551D mutation. One of the studies was in patients aged over 12 years, while the other was in patients aged between 6 and 11 years. After 24 weeks of treatment, patients aged 12 years and older who took Kalydeco had an average improvement in FEV1 of 10.6 percentage points more than those who took placebo. Similar results were seen in patients aged between 6 and 11 years, where Kalydeco treatment led to an improvement of 12.5 percentage points more than treatment with placebo.
The third study involved 39 patients over 6 years of age with cystic fibrosis due to several mutations other than G551D. After 8 weeks of treatment, patients who took Kalydeco had an average improvement in FEV1 of 10.7 percentage points more than those who took placebo.
The fourth study involved 69 patients aged 6 years and above with cystic fibrosis who had the R117H mutation. When analysing the subset of patients aged 18 years and above alone, an average improvement in FEV1 of around 5 percentage points was seen in patients who took Kalydeco compared with patients who took placebo. However, no difference was seen between placebo and Kalydeco for children aged 6 years and above. The study also looked at changes in the level of chloride in patients’ sweat. In all age groups, patients who took Kalydeco had a decrease in sweat chloride level compared with those who took placebo. Patients with cystic fibrosis have high levels of chloride in sweat due to CFTR not working properly and a decrease in sweat chloride can indicate that the medicine is having an effect.
Another study investigated Kalydeco granules in 34 patients aged between 2 and 5 years of age who had cystic fibrosis due to a G551D or S549N mutation. The study found that Kalydeco granules resulted in increased bodyweight and a decrease in sweat chloride. Patients with cystic fibrosis have low bodyweight due to problems with digestion of food.
Positive results were also shown with Kalydeco granules in a study involving 6 children aged 4 months to less than 6 months, 11 children aged 6 months to less than 12 months and 19 children aged 12 months to less than 24 months.
Kalydeco in combination with tezacaftor / ivacaftor
Kalydeco taken together with tezacaftor plus ivacaftor was shown to be effective at improving lung function in two main studies of patients with cystic fibrosis aged 12 years and above and one study of patients from 6 up to 12 years.
The first study involved 510 patients with cystic fibrosis who inherited the F508del mutation from both parents. Kalydeco taken with tezacaftor / ivacaftor was compared with placebo. After 24 weeks of treatment, patients who took the medicines had an average increase in FEV1 of 3.4 percentage points compared with a reduction of 0.6 percentage points in patients who took placebo.
The second study involved 248 patients with cystic fibrosis who inherited the F508del mutation from one parent and who also have another CFTR mutation. Kalydeco taken with tezacaftor / ivacaftor was compared with Kalydeco taken alone and with placebo. Lung function was measured after 4 and 8 weeks of treatment. Patients who took Kalydeco and tezacaftor / ivacaftor had an average increase in FEV1 of 6.5 percentage points compared with an increase of 4.4 percentage points in patients who took Kalydeco alone and a reduction of 0.3 percentage points in patients who took placebo.
The study of patients aged from 6 up to 12 years involved 69 patients who had the F508del mutation from both parents or from one parent together with another mutation. The study looked at a measure of lung disease called the lung clearance index (LCI). After 8 weeks of treatment, patients who took Kalydeco together with tezacaftor / ivacaftor had a moderate decrease in LCI, which can indicate that the medicine is having an effect.
The fourth study involved 66 children aged 6 to 11 years with either an F508del mutation from both parents or an F508del mutation and a ‘minimal function’ mutation. Kalydeco with ivacaftor / tezacaftor / elexacaftor was not compared with other treatments. Patients had an increase in ppFEV1 and a decrease in sweat chloride levels, similar to previous observations in adults and adolescents taking Kalydeco with ivacaftor / tezacaftor / elexacaftor. An additional study involved 75 children aged 2 to 5 years of age with either an F508del mutation from both parents or an F508del mutation and a ‘minimal function’ mutation, who were treated with Kalydeco plus ivacaftor / tezacaftor / elexacaftor. The combination treatment was not compared with other treatments. Treatment with Kalydeco plus ivacaftor / tezacaftor / elexacaftor reduced the level of chloride in patients’ sweat. This decrease in sweat chloride levels was similar to that seen in older patients. Other data also showed that the medicine behaves in the same way in the body of younger children as in that of older children and adults. Taken together, the data suggest that the medicine will be as effective in children aged 2 to 5 years as in older children.
For the full list of side effects and restrictions with Kalydeco, see the package leaflet.
The most common side effects with Kalydeco (which may affect more than 1 in 10 people) include headache, sore throat, upper respiratory tract infection (nose and throat infection), nasal congestion (blocked nose), abdominal (belly) pain, nasopharyngitis (inflammation of the nose and throat), diarrhoea, dizziness, rash, bacteria in sputum (phlegm) and an increase in certain liver enzymes. Serious side effects include increased liver enzymes, which can indicate liver damage, and abdominal pain.
Kalydeco used on its own or together with tezacaftor plus ivacaftor or with ivacaftor, tezacaftor and elexacaftor has been shown to improve lung function or sweat chloride levels in patients with specific mutations. The medicine has an acceptable safety profile. The European Medicines Agency therefore decided that the benefits of Kalydeco are greater than its risks and it can be authorised for use in the EU. The Agency also noted, however, that there were limited data on the longer-term effects of the medicine and that further data should be provided by the company.
The company that markets Kalydeco is conducting a study in children aged 2 to 5 years starting treatment to assess long-term effects of early treatment.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Kalydeco have also been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Kalydeco are continuously monitored. Side effects reported with Kalydeco are carefully evaluated and any necessary action taken to protect patients.
Kalydeco received a marketing authorisation valid throughout the EU on 23 July 2012.
This overview was last updated in 10-2023.
Product information
Latest procedure affecting product information: PSUSA/00009204/202301
19/12/2023
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Product details
- Name of medicine
- Kalydeco
- Active substance
- ivacaftor
- International non-proprietary name (INN) or common name
- ivacaftor
- Therapeutic area (MeSH)
- Cystic Fibrosis
- Anatomical therapeutic chemical (ATC) code
- R07AX02
Pharmacotherapeutic group
Other respiratory system productsTherapeutic 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 (see sections 4.4 and 5.1).
- 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, adolescents, and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene (see section 5.1).
Kalydeco granules are indicated for
- the treatment of infants aged at least 4 months, toddlers and children weighing 5 kg to less than 25 kg with cystic fibrosis (CF) who have an R117H CFTR mutation or 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).
- In a combination regimen with ivacaftor/tezacaftor/elexacaftor for the treatment of cystic fibrosis (CF) in paediatric patients aged 2 to less than 6 years who have at least one F508del mutation in the CFTR gene.
Assessment history
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