New possibilities for targeted therapy of cystic fibrosis

Cystic fibrosis (CF) is an autosomal recessive disorder caused by dysfunction of the CF transmembrane conductance regulator (CFTR) protein that can manifest at birth and progresses throughout life. CF results in multisystem disease with significantly reduced life expectancy. The aim of the review was to analyze the use of the CFTR modulator elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) to justify the use of the new-generation modulator vanzacaftor (VNZ) / TEZ / deitivacaftor (D-IVA) and evaluate its efficacy. Results. We analyzed 35 scientific publications on CF and its targeted therapy and described the efficacy and safety of the VNZ/TEZ/D-IVA combination in patients with CF (n = 1,049) according to the phase III studies. Conclusion. VNZ/TEZ/D-IVA is a next generation CFTR modulator with increased potential to further improve clinical outcomes and further enhance long-term outcomes by restoring normal CFTR function in a larger number of CF patients. Additionally, VNZ/TEZ/D-IVA may improve treatment convenience for patients and caregivers.