Symkevi

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](/en/medicines/human/orphan-designations/eu-3-17-1828)’ (a medicine used in rare diseases) on 27 February 2017\.