NEW YORK – The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Vertex Pharmaceuticals' cystic fibrosis treatment Kaftrio (ivacaftor/tezacaftor/elexacaftor) in combination with Kalydeco (ivacaftor) for patients 2 years and older with at least one non-class I mutation in the CFTR gene.
The recommendation, if adopted by the European Commission, would expand the label for the CFTR modulator Kaftrio, making it available to thousands of additional cystic fibrosis patients in Europe, according to London-based Vertex. The combination of Kaftrio and Kalydeco is currently approved in the EU for cystic fibrosis patients 2 years and older with at least one copy of the CFTR F508del mutation.
Cystic fibrosis, a progressive genetic disease, is characterized by the buildup of a thick mucus that can affect multiple organs, including the lungs, liver, pancreas, gastrointestinal tract, sinuses, sweat glands, and reproductive tract, and is caused by CFTR mutations that impede normal protein production. Most patients harbor the CFTR F508del mutation but the condition can be caused by many different types of mutations in the gene.
Kaftrio is sold in the US under the brand name Trikafta for patients 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical or in vitro data. The US Food and Drug Administration expanded use of the treatment in 2023 based on data from a Phase III open-label study that enrolled 75 cystic fibrosis patients 2 to 5 years of age. Previously, it was approved in the US for older patients.
In this Phase III study, the drug improved sweat chloride concentration and lung function in patients, according to data published in the American Journal of Respiratory and Critical Care Medicine, and Trikafta's safety profile was consistent with what has been observed in older age groups.