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Physiologically Based Pharmacokinetic Modeling of Elexacaftor/Tezacaftor/Ivacaftor in Infants With Cystic Fibrosis [PDF]

CPT: Pharmacometrics & Systems Pharmacology
Ivacaftor is the only cystic fibrosis transmembrane conductance regulator modulator approved for infants ≥ 1 month. The elexacaftor/tezacaftor/ivacaftor combination, approved for children aged ≥ 2 years, has been shown to significantly slow CF ...
Ngoc Hoa Truong, Sihem Benaboud, Naïm Bouazza, Eric Deneuville, Anne Mornand, Philippe Reix, Mélanie Ribault, Pierre‐Régis Burgel, Léo Froelicher‐Bournaud, Steeve Rouillon, Mohammed Rohi Sanoufi, Gabrielle Lui, Saïk Urien, Jean‐Marc Tréluyer, Isabelle Sermet‐Gaudelus, Frantz Foissac, Modul‐CF study group   +16 more
doaj   +2 more sources

Repurposing ivacaftor to attenuate LPS-induced acute lung injury: evidence from a non-cystic fibrosis model [PDF]

Frontiers in Pharmacology
In this study, we explicitly evaluate the anti-inflammatory effects of ivacaftor as a route-of-delivery comparison (intraperitoneal vs. intratracheal) in a reproducible LPS-induced lung inflammation model. We evaluated the effects of ivacaftor [40 mg/kg,
Xiaoxuan Han, Yimin Zhu, Christopher W. Armstrong, Danni Li, Rachel McQuade, Andrew Jarnicki, Elena K. Schneider-Futschik   +6 more
doaj   +2 more sources

Repurposing of ivacaftor shows potential to treat ROR1 expressing high-grade serous ovarian cancer [PDF]

Therapeutic Advances in Medical Oncology
Background: Drug repurposing has emerged as an effective strategy to accelerate drug discovery. Using the pipeline established from a large collaborative drug repurposing project focused on high-grade serous ovarian cancer (HGSOC), we identified ...
Dongli Liu, Michelle Wong-Brown, Farhana Amy Sarker, Bayley Matthews, Jessica Morrison, Kristie-Ann Dickson, Amani Alghalayini, Jana Stojanova, King Man Wan, Jennifer Duggan, Christine Loo, Gill Stannard, Elyse Powell, Brigitte Hodder, Ellen Barlow, Deborah J. Marsh, Caroline E. Ford, Nikola A. Bowden   +17 more
doaj   +2 more sources

Changes in sputum viscoelastic properties and airway inflammation in primary ciliary dyskinesia are comparable to cystic fibrosis on elexacaftor/tezacaftor/ivacaftor therapy. [PDF]

Eur Respir J
Background Primary ciliary dyskinesia (PCD) and cystic fibrosis (CF) are muco-obstructive lung diseases that are caused by distinct genetically determined defects in mucociliary clearance; however, knowledge on the relative severity of airway mucus ...
Nussstein H, Urbantat RM, Fentker K, Loewe A, Duerr J, Haji M, Doellinger F, Stahl M, Graeber SY, Gradzielski M, Röhmel J, Mertins P, Schaupp L, Mall MA.   +13 more
europepmc   +2 more sources

Transcriptomic Responses to Ivacaftor and Prediction of Ivacaftor Clinical Responsiveness

American Journal of Respiratory Cell and Molecular Biology, 2019
Abstract Ivacaftor is a drug that was recently approved by the U.S. Food and Drug Administration for the treatment of patients with cystic fibrosis (CF) and at least one copy of the G511D mutation in the CFTR (CF transmembrane conductance regulator) gene. The transcriptomic effect of ivacaftor in patients with CF remains unclear. Here,
Jay Kolls, Kong Chen, Wei Chen
exaly   +4 more sources

The potentiator ivacaftor is essential for pharmacological restoration of F508del-CFTR function and mucociliary clearance in cystic fibrosis

JCI Insight
Pharmacological rescue of F508del-CFTR by the triple combination CFTR modulator therapy elexacaftor/tezacaftor/ivacaftor (ETI) leads to unprecedented clinical benefits in patients with cystic fibrosis (CF).
Anita Balázs, Tihomir Rubil, Christine K. Wong, Jasmin Berger, Marika Drescher, Kathrin Seidel, Mirjam Stahl, Simon Y. Graeber, Marcus A. Mall   +8 more
doaj   +2 more sources

Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data.

PLoS ONE, 2023
ObjectivesCystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific gene mutations ...
Lisa J McGarry, Zahra Bhaiwala, Andrea Lopez, Conor Chandler, Christopher G Pelligra, Jaime L Rubin, Theodore G Liou   +6 more
doaj   +3 more sources

Insights Into Patient Variability During Ivacaftor-Lumacaftor Therapy in Cystic Fibrosis

Frontiers in Pharmacology, 2021
Background: The advent of cystic fibrosis transmembrane conductance regulator protein (CFTR) modulators like ivacaftor have revolutionised the treatment of cystic fibrosis (CF).
Patrick O. Hanafin, Isabelle Sermet-Gaudelus, Matthias Griese, Matthias Kappler, Helmut Ellemunter, Carsten Schwarz, Carsten Schwarz, John Wilson, John Wilson, Marsha Tan, Tony Velkov, Gauri G. Rao, Elena K. Schneider-Futschik   +12 more
doaj   +1 more source

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

Frontiers in Pharmacology, 2021
Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare
Khadidja Abdallah, Kris De Boeck, Marc Dooms, Steven Simoens   +3 more
doaj   +1 more source

Effects of CFTR modulators on serum biomarkers of liver fibrosis in children with cystic fibrosis

Hepatology Communications, 2023
The cystic fibrosis (CF) transmembrane conductance regulator corrector/potentiator combinations lumacaftor/ivacaftor and elexacaftor/tezacaftor/ivacaftor improve sweat chloride, pulmonary function, and nutrition.
Steven Levitte, Yonathan Fuchs, Russell Wise, Zachary M. Sellers   +3 more
doaj   +1 more source