Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor
Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF). We provide an overview of the body of literature on ELX/TEZ/IVA published between November 2019 and February 2023 after approval b...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-03-01
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| Series: | Frontiers in Pharmacology |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fphar.2023.1158207/full |
| _version_ | 1797866895439822848 |
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| author | Burkhard Tümmler Burkhard Tümmler |
| author_facet | Burkhard Tümmler Burkhard Tümmler |
| author_sort | Burkhard Tümmler |
| collection | DOAJ |
| description | Triple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF). We provide an overview of the body of literature on ELX/TEZ/IVA published between November 2019 and February 2023 after approval by the regulators. Recombinant ELX/TEZ/IVA-bound Phe508del CFTR exhibits a wild type conformation in vitro, but in patient’s tissue a CFTR glyoisoform is synthesized that is distinct from the wild type and Phe508del isoforms. ELX/TEZ/IVA therapy improved the quality of life of people with CF in the real-life setting irrespective of their anthropometry and lung function at baseline. ELX/TEZ/IVA improved sinonasal and abdominal disease, lung function and morphology, airway microbiology and the basic defect of impaired epithelial chloride and bicarbonate transport. Pregnancy rates were increasing in women with CF. Side effects of mental status changes deserve particular attention in the future. |
| first_indexed | 2024-04-09T23:31:37Z |
| format | Article |
| id | doaj.art-3996ce11d4c8459592f7483088957eff |
| institution | Directory Open Access Journal |
| issn | 1663-9812 |
| language | English |
| last_indexed | 2024-04-09T23:31:37Z |
| publishDate | 2023-03-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Pharmacology |
| spelling | doaj.art-3996ce11d4c8459592f7483088957eff2023-03-21T04:30:55ZengFrontiers Media S.A.Frontiers in Pharmacology1663-98122023-03-011410.3389/fphar.2023.11582071158207Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—IvacaftorBurkhard Tümmler0Burkhard Tümmler1Department of Pediatric Pneumology, Allergology and Neonatology, Hannover Medical School, Hannover, GermanyBiomedical Research in Endstage and Obstructive Lung Disease Hannover (BREATH), German Center for Lung Research, Hannover Medical School, Hannover, GermanyTriple combination therapy with the CFTR modulators elexacaftor (ELX), tezacaftor (TEZ) and ivacaftor (IVA) has been qualified as a game changer in cystic fibrosis (CF). We provide an overview of the body of literature on ELX/TEZ/IVA published between November 2019 and February 2023 after approval by the regulators. Recombinant ELX/TEZ/IVA-bound Phe508del CFTR exhibits a wild type conformation in vitro, but in patient’s tissue a CFTR glyoisoform is synthesized that is distinct from the wild type and Phe508del isoforms. ELX/TEZ/IVA therapy improved the quality of life of people with CF in the real-life setting irrespective of their anthropometry and lung function at baseline. ELX/TEZ/IVA improved sinonasal and abdominal disease, lung function and morphology, airway microbiology and the basic defect of impaired epithelial chloride and bicarbonate transport. Pregnancy rates were increasing in women with CF. Side effects of mental status changes deserve particular attention in the future.https://www.frontiersin.org/articles/10.3389/fphar.2023.1158207/fullelexacaftortezacaftorivacaftorCFTRcystic fibrosis |
| spellingShingle | Burkhard Tümmler Burkhard Tümmler Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor Frontiers in Pharmacology elexacaftor tezacaftor ivacaftor CFTR cystic fibrosis |
| title | Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor |
| title_full | Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor |
| title_fullStr | Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor |
| title_full_unstemmed | Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor |
| title_short | Post-approval studies with the CFTR modulators Elexacaftor-Tezacaftor—Ivacaftor |
| title_sort | post approval studies with the cftr modulators elexacaftor tezacaftor ivacaftor |
| topic | elexacaftor tezacaftor ivacaftor CFTR cystic fibrosis |
| url | https://www.frontiersin.org/articles/10.3389/fphar.2023.1158207/full |
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