References
- Schneider EK, Reyes-Ortega F, Li J, et al. Can cystic fibrosis patients finally catch a breath with lumacaftor/ivacaftor? Clin Pharmacol Ther. 2017;101:130–141.
- Clancy JP, Rowe SM, Accurso FJ, et al. Results of a phase IIa study of VX=809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012;67:12–18.
- Massie J, Delatycki MB. Cystic fibrosis carrier screening. Paediatr Respir Rev. 2013;14:270–275.
- O’Sullivan BP, Freedman SD. Cystic fibrosis. Lancet. 2009;373:1891–1904.
- Zhang W, Zhang X, Zhang YH, et al. Lumacaftor/Ivacaftor combination for cystic fibrosis patients homozygous for phe508del-CFTR. Drugs Today. 2016;52:229–237.
- Schmidt BZ, Haaf JB, Leal T, et al. Cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis: current perspectives. Clin Pharmacol. 2016;8:127–140.
- Boyle MP. Nonclassic cystic fibrosis and CFTR-related diseases. Curr Opin Pulm Med. 2003;9:498–503.
- De Boeck K, Wilschanski M, Castellani C, et al. Cystic fibrosis: terminology and diagnostic algorithms. Thorax. 2006;61:627–635.
- Vertex Pharmaceuticals. Press release. Vertex receives EU approval for ORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation. 2015 Nov 20 [cited 2016 Jan 6]. Available from: http://investors.vrtx.com/releasedetail.cfm?ReleaseID=943778
- Pillarisetti N, Williamson E, Linnane B, et al. Infection, inflammation, and lung function decline in infants with cystic fibrosis. Am J Respir Crit Care Med. 2011;184:75–81.
- Lyczak JB, Cannon CL, Pier GB. Lung infections associated with cystic fibrosis. Clin Microbiol Rev. 2002;15(2):194–222.
- Bronstein MN, Sokol RJ, Abman SH, et al. Pancreatic insufficiency, growth, and nutrition in infants identified by newborn screening as having cystic fibrosis. J Pediatr. 1992;120:533–540.
- Gorter RR, Karimi A, Sleeboom C, et al. Clinical and genetic characteristics of meconium ileus in newborns with and without cystic fibrosis. J Pediatr Gastroenterol Nutr. 2010;50:569–572.
- Colombo C. Liver disease in cystic fibrosis. Curr Opin Pulm Med. 2007;13:529–536.
- Kaplan E, Shwachman H, Perlmutter AD, et al. Reproductive failure in males with cystic fibrosis. N Engl J Med. 1968;279:65–69.
- Elborn JS, Prescott RJ, Stack BH, et al. Elective versus symptomatic antibiotic treatment in cystic fibrosis patients with chronic Pseudomonas infection of the lungs. Thorax. 2000;55:355–358.
- Weinberger M. Airway reactivity in patients with CF. Clin Rev Allergy Immunol. 2002;23:77–85.
- Mayer M. Lumacaftor-ivacaftor (Orkambi) for cystic fibrosis: behind the breakthrough. Evid Based Med. 2016;21:83–86.
- Martiniano SL, Sagel SD, Zemanick ET. Cystic fibrosis: a model system for precision medicine. Curr Opin Pediatr. 2016;28:312–317.
- Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor, ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. N Eng J Med. 2015;376:220–231.
- AbbVie Inc. Pipeline. [ cited 2017 Apr 30]. Available from: https://www.abbvie.com/our-science/pipeline.html
- Galapagos Pipeline. [ cited2017 Apr 30]. Available from: http://www.glpg.com/rd-cystic-fibrosis
- The next generation fight against CF. Biotech primer. [ updated 2016 Jul 27; cited 2017 Apr 30]. Available from: https://weekly.biotechprimer.com/the-next-generation-fight-against-cf/
- Vertex pharmaceuticals research and pipeline. cited 2017 Apr 30. Available from: https://www.vrtx.com/pipeline-medicines/investigational-medicines-pipeline
- ORKAMBI (lumacaftor/ivacaftor). Prescribing Information. (MA): Vertex Pharmaceuticals Inc; 2016 Sep.
- Food and Drug Administration. Sponsor briefing document: ORKAMBI (Lumacaftor/Ivacaftor) for the treatment of cystic fibrosis in patients age 12 years and older who are homozygous for the F508del mutation in the CFTR gene. In: committee FACBMV-FP-Ada, editor. Silver Springs, MD: VERTEX Pharmaceuticals; 2015.
- ORKAMBI (lumacaftor/ivacaftor). European public assessment report product information. London (UK): Vertex Pharmaceuticals Inc.; 2016 Jul.
- Food and Drug Administration. Orkambi (lumacaftor/ivacaftor) oral tablet. [ cited 2017 Apr 30]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/0206038Orig1s000TOC.cfm
- European Medicines Agency. Orkambi. [cited 2017 Jul 24]. Available from: http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/human/medicines/003954/human_med_001935.jsp&mid=WC0b01ac058001d124
- Australian Therapeutic Goods Administration. Australian public assessment report for lumacaftor/Ivacaftor. 2016 Sep 8 [cited 2017 Jul 24]. Available from: http://search.tga.gov.au/s/search.html?collection=tga-artg&profile=record&meta_i=235759
- Boyle MP, Bell SC, Konstan MW, et al. A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for the treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomized controlled trial. Lancet Respir Med. 2014;2:527–538.
- Konstan MW, McKane EF, Moss RB, et al. Assessment of safety and efficacy of long-term treatment with combination lumacaftor and ivacaftor therapy in patients with cystic fibrosis homozygous for the F508del-CFTR mutation (PROGRESS): a phase 3, extension study. Lancet Respir Med. 2017;5:107–118.
- Farinha CM, Sousa M, Canato S, et al. Increased Efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR. Pharmacol Res Perspect. 2015;3:e00152.
- Gentzsch M, Ren HY, Houch SA, et al. Restoration of R11H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770. Am J Physiol Lung Mol Physiol. 2016;311:L550–L559.
- Kopeikin Z, Yuksek Z, Yang HY, et al. Combined effects of VX-770 and VX-809 on several functional abnormalities of F508del-CFTR channels. J Cystic Fibrosis. 2014;13:508–514.
- 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 USA. 2011;108:18843–18848.
- Matthes E, Goepp J, Carlile GW, et al. Low free drug concentration prevents inhibition of F508del CFTR functional expression by the potentiator VX-770 (ivacaftor). Brit J Pharmacol. 2016;173:459–470.
- Marigowda G, Liu F, Waltz D. Effect of bronchodilators in health individuals receiving lumacaftor/ivacaftor combination therapy. J Cystic Fibrosis. 2017;16:246–249.
- Pawaskar D, Marigowda G, Waltz D, et al. The effect of ciprofloxacin, itraconazole, and rifampin on the pharmacokinetics of lumacaftor in combination with ivacaftor in healthy individuals. Pediatr Pulmonol. 2014;49(S38):286.
- Jordan CL, Noah TL, Henry MM. Therapeutic challenges posed by critical drug-drug interactions in cystic fibrosis. Pediatr Pulmonol. 2016;51:S61–S70.
- Elborn JS, Ramsey BW, Boyle MP, et al. Efficacy and safety of lumacaftor/ivacaftor combination therapy in patients with cystic fibrosis homozygous for Phe508del CFTR by pulmonary function subgroup: a pooled analysis. Lancet Respir Med. 2016;4:617–626.
- Milla CE, Ratjen F, Marigowda G, et al. Lumacaftor/ivacaftor in patients aged 6–11 years with cystic fibrosis and homozygous for F508del-CFTR. Am J Respir Crit Care Med. 2017;195:912–920.
- Hubert D, Chiron R, Camara B, et al. Real-life initiation of lumacaftor/ivacaftor combination in adults with cystic fibrosis homozygous for the Phe508del CFTR mutation and severe lung disease. J Cyst Fibrosis. 2017;pii:S1569-1993(17)30086-3. DOI:10.1016/j.jcf.2017.03.003
- Jennings MT, Dezube R, Paranjape S, et al. An observational study of outcomes and tolerances in patients with cystic fibrosis initiated on lumacaftor/ivacaftor. Ann Am Thorac Soc. 2017.
- Food and Drug Administration. Press release. FDA approves new treatment for cystic fibrosis. 2015 Jul 2 [cited 2017 Jan 6]. Available from: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm453565.htm
- Vertex Pharmaceuticals. Press release. Health canada approves PrORKAMBI® (lumacaftor/ivacaftor) – the first medicine to treat the underlying cause of cystic fibrosis for people ages 12 and older with two copies of the F508del mutation. [cited 2017 Jul 24]. Available from: http://investors.vrtx.com/releasedetail.cfm?releaseid=951851
- Canadian Agency for Drugs and Technologies in Health Canadian Drug Expert Committee. Common drug review final recommendations; lumacaftor/ivacaftor. [ updated 2016 Oct 26; cited 2017 Apr 30]. Available from: https://www.cadth.ca/lumacaftorivacaftor
- Japanese Ministry of Health, Labour, and Welfare. [ cited 2017 May 4]. Available from: http://www.mhlw.go.jp/english/index.html
- National Institute for Health and Care Excellence. Lumacaftor–ivacaftor for treating cystic fibrosis homozygous for the F508del mutation. [ cited 2017 Apr 30]. Available from: https://www.nice.org.uk/guidance/ta398
- Silverman E. Orkambi’s Slick unveiling puts insurers in a bind. Managed Care Magazine. [ cited 2017 Apr 30]. Available from: https://www.managedcaremag.com/archives/2015/8/orkambis-slick-unveiling-puts-insurers-bind