VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles

New England Journal of Medicine

Volumn 379, Issue 17, 2018, Pages 1612-1620

  Keating, Dominic ^a   Marigowda, Gautham ^c   Burr, Lucy ^b   Daines, Cori ^d   Mall, Marcus A ^e   McKone, Edward F ^f   Ramsey, Bonnie W ^g   Rowe, Steven M ^h   Sass, Laura A ⁱ   Tullis, Elizabeth ^j   McKee, Charlotte M ^c   Moskowitz, Samuel M ^c   Robertson, Sarah ^c   Savage, Jessica ^c   Simard, Christopher ^c   Van Goor, Fredrick ^c   Waltz, David ^c   Xuan, Fengjuan ^c   Young, Tim ^c   Taylor Cousar, Jennifer L ^k  

^a ALFRED HOSPITAL   (Australia)

^b MATER HOSPITAL   (Australia)

^c VERTEX PHARMACEUTICALS   (United States)

^d UNIVERSITY OF ARIZONA MEDICAL CENTER   (United States)

^e CHARITÉ UNIVERSITÄTSMEDIZIN BERLIN   (Germany)

^f German Center for Lung Research   (Germany)

^g ST VINCENT S UNIVERSITY HOSPITAL   (Ireland)

^h UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

ⁱ CHILDREN S HOSPITAL OF THE KING S DAUGHTERS   (United States)

^j ST MICHAEL'S HOSPITAL   (Canada)

^k DEPARTMENT OF MEDICINE   (United States)

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Author keywords

[No Author keywords available]

Indexed keywords

1,3 BENZODIOXOLE DERIVATIVE; AGENTS AFFECTING METABOLISM; ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; BILIRUBIN; CREATINE KINASE; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; IVACAFTOR; IVACAFTOR PLUS TEZACAFTOR; IVACAFTOR PLUS TEZACAFTOR PLUS VX 445; PHENYLALANINE; PLACEBO; TEZACAFTOR; UNCLASSIFIED DRUG; VX 445; AMINOPHENOL DERIVATIVE; CFTR PROTEIN, HUMAN; CHLORIDE; CHLORIDE CHANNEL STIMULATING AGENT; INDOLE DERIVATIVE; QUINOLONE DERIVATIVE;

ADULT; ALLELE; AMINO ACID SUBSTITUTION; ARTICLE; BRONCHOCONSTRICTION; CFTR GENE; CHLORIDE TRANSPORT; COHORT ANALYSIS; COMBINATION DRUG THERAPY; CONSTIPATION; CONTROLLED STUDY; COUGHING; CYSTIC FIBROSIS; CYSTIC FIBROSIS QUESTIONNAIRE; DIGESTIVE SYSTEM DISEASE ASSESSMENT; DISEASE SEVERITY; DOSE RESPONSE; DOUBLE BLIND PROCEDURE; DRUG DOSE COMPARISON; DRUG EFFICACY; DRUG SAFETY; DRUG WITHDRAWAL; FEMALE; FEVER; FORCED EXPIRATORY VOLUME; GENE DELETION; HEMOPTYSIS; HOMOZYGOSITY; HUMAN; IN VITRO STUDY; INTESTINE OBSTRUCTION; LUNG DISEASE; LUNG INFECTION; MAJOR CLINICAL STUDY; MALE; MYOPATHY; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL; PREDICTION; PRIORITY JOURNAL; PROTEIN TRANSPORT; RASH; RESPIRATORY DOMAIN SCORE; RESPIRATORY TRACT DISEASE ASSESSMENT; SIDE EFFECT; THORAX PAIN; VEIN THROMBOSIS; ADOLESCENT; CHEMISTRY; CLINICAL TRIAL; DRUG COMBINATION; DRUG EFFECT; GENETICS; GENOTYPE; METABOLISM; MULTICENTER STUDY; MUTATION; RANDOMIZED CONTROLLED TRIAL; SWEAT; YOUNG ADULT;

ADOLESCENT; ADULT; ALLELES; AMINOPHENOLS; BENZODIOXOLES; CHLORIDE CHANNEL AGONISTS; CHLORIDES; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DOUBLE-BLIND METHOD; DRUG COMBINATIONS; FEMALE; FORCED EXPIRATORY VOLUME; GENOTYPE; HUMANS; INDOLES; MALE; MUTATION; QUINOLONES; SWEAT; YOUNG ADULT;

EID: 85055433680     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1807120     Document Type: Article

References (7)

1. Davies JC, Moskowitz SM, Brown C, et al. VX-659-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med 2018; 379:1599-611.
2. Van Goor F, Hadida S, Grootenhuis PD, et al. Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809. Proc Natl Acad Sci U S A 2011; 108: 18843-8.
3. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. N Engl J Med 2011; 365: 1663-72.
4. Davies JC, Wainwright CE, Canny GJ, et al. Efficacy and safety of ivacaftor in patients aged 6 to 11 years with cystic fibrosis with a G551D mutation. Am J Respir Crit Care Med 2013; 187: 1219-25.
5. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Engl J Med 2015; 373: 220-31.
6. Rowe SM, Daines C, Ringshausen FC, et al. Tezacaftor-ivacaftor in residualfunction heterozygotes with cystic fibrosis. N Engl J Med 2017; 377: 2024-35.
7. Taylor-Cousar JL, Munck A, McKone EF, et al. Tezacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del. N Engl J Med 2017; 377: 2013-23.