Symkevi
Authorised
This medicine is authorised for use in the European Union
tezacaftor / ivacaftor
MedicineHumanAuthorised
Overview
Symkevi is a medicine used to treat cystic fibrosis in patients aged 6 years and above. Cystic fibrosis is 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.
Symkevi is used in patients who have a mutation (change) called F508del in the gene for a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR).
Symkevi is used in patients who have inherited the F508del mutation from both parents and therefore have the mutation in both copies of the CFTR gene. It is 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, 327226A→G, or 3849+10kbC→T.
Symkevi contains the active substances tezacaftor and ivacaftor.
Cystic fibrosis is rare, and Symkevi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 27 February 2017.
Symkevi should only be prescribed by a doctor with experience in the treatment of cystic fibrosis, and only in patients confirmed to have the mutations mentioned above.
Symkevi is available as tablets. Treatment is combined with a tablet containing ivacaftor alone.
Symkevi should be taken in the morning and ivacaftor should be taken in the evening, about 12 hours later. The dose depends on the patient’s age and weight.
The doses of Symkevi and ivacaftor 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. The doses may also need to be adjusted in patients with reduced liver function.
For more information about using Symkevi, see the package leaflet or contact your doctor or pharmacist.
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 and digestive juices. The mutations reduce the number of CFTR proteins on the cell surface or affect the way the protein works.
One of the active substances in Symkevi, tezacaftor, increases the number of CFTR proteins on the cell and the other, ivacaftor, increases the activity of the defective CFTR protein. These actions restore the activity of CFTR protein and make mucus and digestive juices less thick, thereby helping to relieve symptoms of the disease.
Symkevi taken together with ivacaftor was effective at improving lung function in 3 main studies of patients with cystic fibrosis aged 6 years and above.
In the first 2 studies, the main measure of effectiveness was improvement 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.
The first study involved 510 patients aged 12 years or above with cystic fibrosis who inherited the F508del mutation from both parents. Symkevi, taken with ivacaftor, was compared with placebo (a dummy treatment). 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 aged 12 years or above with cystic fibrosis who inherited the F508del mutation from one parent and also had another specific CFTR mutation. Symkevi, taken with ivacaftor, was compared with ivacaftor taken alone and with placebo. Lung function was measured after 4 weeks and 8 weeks of treatment. Patients who took Symkevi and 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 ivacaftor alone and a reduction of 0.3 percentage points in patients who took placebo.
The effectiveness of Symkevi in a comparable group of children with cystic fibrosis aged 6 to 11 years was supported by a study in 54 children aged 6 to 11 years . This study looked at the effect of Symkevi on lung clearance index (LCI2.5), which indicates how well air is exchanged in the lungs. A fall in LCI2.5 indicates improvement. At the start of the study patients’ LCI2.5 was on average 9.56. After 8 weeks of treatment with Symkevi and ivacaftor, LCI2.5 decreased by 0.51. The effectiveness of Symkevi in children aged between 6 and 11 years was also supported by evidence that the medicine behaves in the same way in their body as in older patients. However the dose needs to be adjusted based on the patient’s weight and age.
The most common side effects with Symkevi (which may affect more than 1 in 10 people) are headache and nasopharyngitis (inflammation of the nose and throat).
For the full list of side effects and restrictions with Symkevi, see the package leaflet.
Symkevi is effective in patients with cystic fibrosis who have inherited the F508del mutation from both parents or patients who have the F508del mutation from one parent and certain other mutations. In the former group, Symkevi could be a treatment option for those who cannot take a combination of ivacaftor and lumacaftor (another cystic fibrosis medicine), due to side effects or interactions with other medicines they are taking. In the latter group, there is a lack of authorised therapies. Regarding safety, the side effects of Symkevi are considered acceptable. Therefore, the European Medicines Agency decided that Symkevi’s benefits are greater than its risks and it can be authorised for use in the EU.
Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Symkevi have been included in the summary of product characteristics and the package leaflet.
As for all medicines, data on the use of Symkevi are continuously monitored. Side effects reported with Symkevi are carefully evaluated and any necessary action taken to protect patients.
Symkevi received a marketing authorisation valid throughout the EU on 31 October 2018.
Product information
Latest procedure affecting product information: PSUSA/00010730/202302
07/12/2023
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
- Symkevi
- Active substance
- tezacaftor
- ivacaftor
- International non-proprietary name (INN) or common name
- tezacaftor
- ivacaftor
- Therapeutic area (MeSH)
- Cystic Fibrosis
- Anatomical therapeutic chemical (ATC) code
- R07AX31
Pharmacotherapeutic group
Other respiratory system productsTherapeutic indication
Symkevi is indicated in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 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 cystic fibrosis transmembrane conductance regulator (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.
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