Alyftrek

Alyftrek is a medicine used in people aged 6 years and older to treat cystic fibrosis, an inherited disease that has severe effects on the lungs, the digestive system and other organs. Cystic fibrosis can be caused by various mutations (changes) in the gene that contains instructions for making a protein called ‘cystic fibrosis transmembrane conductance regulator’ (CFTR). Mutations in the CFTR gene are grouped into five different classes (class I to class V) based on the problems they cause with the production of the CFTR protein. Alyftrek is used in people whose cystic fibrosis is caused by at least one mutation that is not a class I mutation. Class I mutations are mutations that result in no CFTR protein being produced. Cystic fibrosis is rare, and Alyftrek was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 12 November 2021. Alyftrek contains the active substances deutivacaftor, tezacaftor and vanzacaftor.