EMA’s human medicines committee (CHMP) has recommended extending the therapeutic indication of two medicines, Kaftrio (ivacaftor / tezacaftor / elexacaftor) and Kalydeco (ivacaftor) for the treatment of cystic fibrosis, to include their use in combination for patients aged two years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Cystic fibrosis is an inherited disease that has severe effects on the lungs, the digestive system and other organs. It comes with high premature mortality. Cystic fibrosis can be caused by various mutations in the gene for a protein called the ‘cystic fibrosis transmembrane conductance regulator’. The CFTR protein regulates the production of mucus in the lungs and digestive juices in the gut. Some mutations affect the way the protein works, resulting in mucus and digestive fluids being too thick, which leads to blockages, inflammation, increased risk of lung infections, and poor digestion and growth. Other mutations, called class I mutations, result in no CFTR protein being produced…
