A new small molecule combination treatment for cystic fibrosis has been given a positive opinion by the European Medicines Agency – expanding the repertoire of protein modulators for treating this disease. The combination, Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor), was developed by Vertex Pharmaceuticals Inc. It modulates the cystic fibrosis transmembrane conductance regulator (CFTR) protein which is produced by a gene of the same name.
