New possibilities for targeted therapy of cystic fibrosis
https://doi.org/10.18093/0869-0189-2025-35-2-167-176
Abstract
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.
Supporting agencies: There was no sponsorship or financial support for the article
About the Authors
E. I. Kondratyeva
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation; State Budgetary Healthcare Institution of the Moscow region “Research Clinical Institute of Childhood”, Healthcare Ministry of Moscow Region
Russian Federation
Competing Interests:
Russian Federation
Elena I. Kondratyeva, Doctor of Medicine, Professor, Deputy Director of the Center, Head of the Department
Cystic Fibrosis Center; Scientific and Clinical Department of Cystic Fibrosis; Department of Genetics of Diseases of the Respiratory System
115522; ul. Moskvorechye 1; Moscow; 141009; ul. Kominterna 124A, build. 1; Moscow Region; Mytishchi
tel.: (495) 324-20-24
Scopus ID: 35196167800; Web of Science Researcher ID: АВВ-9783–2021
Competing Interests:
There is no conflict of interest
S. N. Avdeev
Federal State Autonomous Educational Institution of Higher Education I.M.Sechenov First Moscow State Medical University of the Ministry of Health of the Russian Federation (Sechenov University)
Russian Federation
Competing Interests:
Russian Federation
Sergey N. Avdeev, Doctor of Medicine, Professor, Academician of Russian Academy of Sciences, Director of the National Medical Research Center, Head of the Department, Chief Pulmonologist of the Ministry of Health of the Russian Federation
National Medical Research Center for Pulmonology; N.V. Sklifosovsky Institute of Clinical Medicine; Department of Pulmonology; Federal State Budgetary Institution “Pulmonology Scientific Research Institute” under Federal Medical and Biological Agency of Russian Federation; Clinical Department
119991; ul. Trubetskaya 8, build. 2; Moscow
tel.: (495) 708-35-76
Competing Interests:
There is no conflict of interest
S. I. Kutsev
Federal State Budgetary Scientific Institution “Research Centre for Medical Genetics”, Ministry of Science and Higher Education of the Russian Federation
Russian Federation
Competing Interests:
Russian Federation
Sergey I. Kutsev, Doctor of Medicine, Professor, Academician of Russian Academy of Sciences, Director of the Federal State Budgetary Scientific Institution, Chief Freelance Specialist in Medical Genetics, Ministry of Health of the Russian Federation, Chairman of the Ethics Committee, Ministry of Health of the Russian Federation, President of the Association of Medical Geneticists
115522; ul. Moskvorechye 1; Moscow
tel: (495) 612-00-37
Competing Interests:
There is no conflict of interest
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Kondratyeva E.I., Avdeev S.N., Kutsev S.I. New possibilities for targeted therapy of cystic fibrosis. PULMONOLOGIYA. 2025;35(2):167-176. (In Russ.) https://doi.org/10.18093/0869-0189-2025-35-2-167-176
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