Product attributes
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, 3272 26A→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.
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 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.
