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Tezacaftor

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https://www.readbyqxmd.com/read/30073878/tezacaftor-for-the-treatment-of-cystic-fibrosis
#1
Marc Sala, Manu Jain
Cystic fibrosis (CF) is the most common, life-limiting autosomal recessive disease in Caucasians, and is caused by defects in production of the CFTR ion channel. Until recently, there were no available treatments targeting the disease-causing defects in CFTR but newly developed CFTR modulators are changing the course of disease in CF. The newest modulator, tezacaftor, is a CFTR corrector that was recently approved by the FDA to be used in combination with the first approved CFTR potentiator, ivacaftor. Areas Covered: A detailed review of the clinical trials and published literature, focusing on safety and efficacy, leading to the approval of tezacaftor in cystic fibrosis...
August 3, 2018: Expert Review of Respiratory Medicine
https://www.readbyqxmd.com/read/29627168/cystic-fibrosis-papers-of-the-year-2017
#2
REVIEW
Iolo Doull
The number of published articles on Cystic Fibrosis (CF) continues to increase year on year. The evidence base for small molecule therapies in CF has continued to expand, with evidence for lumacaftor/ivacaftor in younger patients and longer-term evidence in adults, and pivotal studies on tezacaftor/ivacaftor. There were reports on emerging CFTR mutation agnostic therapies, and new evidence for long standing therapies.
March 15, 2018: Paediatric Respiratory Reviews
https://www.readbyqxmd.com/read/29595119/cytochrome-p450-3a4-induction-lumacaftor-versus-ivacaftor-potentially-resulting-in-significantly-reduced-plasma-concentration-of-ivacaftor
#3
Elena K Schneider
Since release of ivacaftor-lumacaftor several red-flags have been raised that highlight the clinical efficacy of this combination strategy may be be limited due to antagonistic drug-drug interactions. </p><p> Method: The effect of ivacaftor, its major metabolites M1 and M6, lumacaftor and the novel cystic fibrosis transmembrane conductance regulator (CFTR) modulator tezacaftor at 10 µg/mL on the enzymatic activity of the major xenobiotic metabolizing enzymes CYP1A2 and CYP3A4 as well as the minor enzymes CYP2B6 and CYP2C9 was assayed...
March 27, 2018: Drug Metabolism Letters
https://www.readbyqxmd.com/read/29538046/cystic-fibrosis-transmembrane-conductance-regulator-modulators-precision-medicine-in-cystic-fibrosis
#4
Elizabeth B Burgener, Richard B Moss
PURPOSE OF REVIEW: The aim of this study was to describe the newest development in cystic fibrosis (CF) care, CF transmembrane conductance regulator (CFTR) modulator therapies. RECENT FINDINGS: Phase II results showing CFTR modulator triple therapies are more effective than current CFTR modulators. SUMMARY: CFTR modulator therapy targets the protein defective in CF and boosts its function, but the drug must match mutation pathobiology. Ivacaftor, a CFTR potentiator, was the first modulator approved in 2012, with impressive improvement in lung function and other measures of disease in patients with gating and other residual function mutations (∼10% of CF patients)...
June 2018: Current Opinion in Pediatrics
https://www.readbyqxmd.com/read/29279204/ivacaftor-restores-cftr-dependent-sweat-gland-fluid-secretion-in-cystic-fibrosis-subjects-with-s945l-alleles
#5
Jeeyeon Kim, Zoe Davies, Colleen Dunn, Jeffrey J Wine, Carlos Milla
BACKGROUND: To determine in vivo effects of CFTR modulators on mutation S945L. METHODS: We measured effects of CFTR modulators on CFTR-dependent sweating ('C-sweat') in two pancreatic sufficient cystic fibrosis (CF) subjects. S1 (S945L/G542X) took ivacaftor and S2 (S945L/F508del) took ivacaftor+tezacaftor. Sweating was stimulated pharmacologically to produce sequentially both CFTR-independent (methacholine stimulated) M-sweat and C-sweat; and the ratio of these was compared...
March 2018: Journal of Cystic Fibrosis: Official Journal of the European Cystic Fibrosis Society
https://www.readbyqxmd.com/read/29248431/tezacaftor-ivacaftor-is-safe-and-efficacious-in-patients-with-cystic-fibrosis-with-phe508del-mutations
#6
Tony Kirby
No abstract text is available yet for this article.
January 2018: Lancet Respiratory Medicine
https://www.readbyqxmd.com/read/29099344/tezacaftor-ivacaftor-in-patients-with-cystic-fibrosis-homozygous-for-phe508del
#7
RANDOMIZED CONTROLLED TRIAL
Jennifer L Taylor-Cousar, Anne Munck, Edward F McKone, Cornelis K van der Ent, Alexander Moeller, Christopher Simard, Linda T Wang, Edward P Ingenito, Charlotte McKee, Yimeng Lu, Julie Lekstrom-Himes, J Stuart Elborn
BACKGROUND: Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor (VX-661) and ivacaftor (VX-770) was designed to target the underlying cause of disease in patients with cystic fibrosis. METHODS: In this phase 3, randomized, double-blind, multicenter, placebo-controlled, parallel-group trial, we evaluated combination therapy with tezacaftor and ivacaftor in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation...
November 23, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29099333/tezacaftor-ivacaftor-in-residual-function-heterozygotes-with-cystic-fibrosis
#8
RANDOMIZED CONTROLLED TRIAL
Steven M Rowe, Cori Daines, Felix C Ringshausen, Eitan Kerem, John Wilson, Elizabeth Tullis, Nitin Nair, Christopher Simard, Linda Han, Edward P Ingenito, Charlotte McKee, Julie Lekstrom-Himes, Jane C Davies
BACKGROUND: Cystic fibrosis is an autosomal recessive disease caused by mutations in the CFTR gene that lead to progressive respiratory decline. Some mutant CFTR proteins show residual function and respond to the CFTR potentiator ivacaftor in vitro, whereas ivacaftor alone does not restore activity to Phe508del mutant CFTR. METHODS: We conducted a randomized, double-blind, placebo-controlled, phase 3, crossover trial to evaluate the efficacy and safety of ivacaftor alone or in combination with tezacaftor, a CFTR corrector, in 248 patients 12 years of age or older who had cystic fibrosis and were heterozygous for the Phe508del mutation and a CFTR mutation associated with residual CFTR function...
November 23, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28930490/tezacaftor-ivacaftor-in-subjects-with-cystic-fibrosis-and-f508del-f508del-cftr-or-f508del-g551d-cftr
#9
Scott H Donaldson, Joseph M Pilewski, Matthias Griese, Jon Cooke, Lakshmi Viswanathan, Elizabeth Tullis, Jane C Davies, Julie A Lekstrom-Himes, Linda T Wang
RATIONALE: Tezacaftor (formerly VX-661) is an investigational small molecule that improves processing and trafficking of the cystic fibrosis transmembrane conductance regulator (CFTR) in vitro, and improves CFTR function alone and in combination with ivacaftor. OBJECTIVES: To evaluate the safety and efficacy of tezacaftor monotherapy and of tezacaftor/ivacaftor combination therapy in subjects with cystic fibrosis homozygous for F508del or compound heterozygous for F508del and G551D...
January 15, 2018: American Journal of Respiratory and Critical Care Medicine
https://www.readbyqxmd.com/read/28785019/correction-of-cftr-function-in-nasal-epithelial-cells-from-cystic-fibrosis-patients-predicts-improvement-of-respiratory-function-by-cftr-modulators
#10
Iwona M Pranke, Aurélie Hatton, Juliette Simonin, Jean Philippe Jais, Françoise Le Pimpec-Barthes, Ania Carsin, Pierre Bonnette, Michael Fayon, Nathalie Stremler-Le Bel, Dominique Grenet, Matthieu Thumerel, Julie Mazenq, Valerie Urbach, Myriam Mesbahi, Emanuelle Girodon-Boulandet, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus
Clinical studies with modulators of the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein have demonstrated that functional restoration of the mutated CFTR can lead to substantial clinical benefit. However, studies have shown highly variable patient responses. The objective of this study was to determine a biomarker predictive of the clinical response. CFTR function was assessed in vivo via nasal potential difference (NPD) and in human nasal epithelial (HNE) cultures by the response to Forskolin/IBMX and the CFTR potentiator VX-770 in short-circuit-current (∆I scF/I+V ) experiments...
August 7, 2017: Scientific Reports
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