Today, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (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. Many of our Society’s members were extremely active in advocating for this extension- your hard work on behalf of the community is very much appreciated
Combination of cystic fibrosis medicines to treat patients with rare mutations | European Medicines Agency (EMA) [1]