A safety evaluation of ivacaftor for the treatment of cystic fibrosis

Expert Opinion on Drug Safety

Volumn 15, Issue 5, 2016, Pages 709-715

  McColley, Susanna A ^a,b,c  

^a NORTHWESTERN UNIVERSITY   (United States)

^b CHILDREN'S MEMORIAL HOSPITAL   (United States)

^c NORTHWESTERN UNIVERSITY   (United States)

Author keywords

cataract; Cystic fibrosis; ivacaftor; transaminitis

Indexed keywords

CYTOCHROME P450 3A; IVACAFTOR; LUMACAFTOR; MULTIDRUG RESISTANCE PROTEIN; AMINOPHENOL DERIVATIVE; CHLORIDE CHANNEL STIMULATING AGENT; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; QUINOLONE DERIVATIVE;

CATARACT; CLINICAL TRIAL (TOPIC); CYSTIC FIBROSIS; DISEASE EXACERBATION; DRUG EFFICACY; DRUG FORMULATION; DRUG MECHANISM; DRUG METABOLISM; DRUG MONITORING; DRUG SAFETY; DRUG SCREENING; HUMAN; HYPERTRANSAMINASEMIA; LIVER DISEASE; LUNG FUNCTION; NONHUMAN; NUTRITIONAL STATUS; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); POSTMARKETING SURVEILLANCE; REVIEW; RISK BENEFIT ANALYSIS; SYSTEMATIC REVIEW; ANIMAL; CHILD; DRUG INTERACTION; GENETICS; MUTATION; QUALITY OF LIFE; RAT;

AMINOPHENOLS; ANIMALS; CHILD; CHLORIDE CHANNEL AGONISTS; CYSTIC FIBROSIS; CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR; DRUG INTERACTIONS; HUMANS; MUTATION; QUALITY OF LIFE; QUINOLONES; RATS;

EID: 84964067974     PISSN: 14740338     EISSN: 1744764X     Source Type: Journal    
DOI: 10.1517/14740338.2016.1165666     Document Type: Review

References (36)

1. Rowe SM, Miller S, Sorscher EJ. Cystic fibrosis. N Engl J Med. 2005;352:1992-2001.
2. De Boeck K, Kent L, Davies J, et al. CFTR biomarkers: time for promotion to surrogate end-point. Eur Respir J. 2013;41:203-216.
3. Cystic Fibrosis Foundation Patient Data Registry Report. [cited 2015 Sep 29]. Available from: www.cff.org/livingwithcf/qualityimprove ment/patientdataregistryreport.
4. Cystic Fibrosis Trust. [cited 2016 Jan 2]. Available from: http://www. cysticfibrosis.org.uk/about-cf/frequently-asked-questions#na.
5. The Canadian Cystic Fibrosis Registry. 2013. Annual Report; [cited 2016 Jan 2]. Available from: http://www.cysticfibrosis.ca/wp-con tent/uploads/2015/02/Canadian-CF-Registry-2013-FINAL.pdf.
6. Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med. 2013;187:680-689.
7. Smyth AR, Bell SC, Bojcin S, et al. European Cystic Fibrosis Society standards of care: best practice guidelines. J Cyst Fibros. 2014;13: S23-S42.
8. Sawicki GS, Tiddens H. Managing treatment complexity in cystic fibrosis: challenges and opportunities. Pediatr Pulmonol. 2012;47:523-533.
9. Modi AC, Lim CS, Yu N, et al. A multi-method assessment of treatment adherence for children with cystic fibrosis. J Cyst Fibros. 2006;5:177-185.
10. Rommens JM, Iannuzzi MC, Kerem B, et al. Identification of the cystic fibrosis gene: chromosome walking and jumping. Science. 1989;245:1059-6511.
11. ClinicalTrials.gov. A service of the National Institutes of Health. [cited 2015 Oct 17]. Available from: http://www.clinicaltrials.gov/ct2/results?term=CFTR&Search=Search.
12. Konstan M, Kerem E, DeBoeck K, et al. Ataluren treatment of patients with nonsense mutation cystic fibrosis. Lancet Respir Med. Early Online Publication; cited 2014 May 16. doi:10.1016/S2213-2600(14)70100-6.
13. McColley SA. Ivacaftor therapy for cystic fibrosis. Expert Opin Orphan Drugs. 2:1225-1232. [cited 2014 Jul 21]. doi:10.1517/21678707.2014.942220.
14. Van Goor F, Hadida S, Grootenhuis PD, et al. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770. Proc Natl Acad Sci U S A. 2009;106:18825-18830.
15. Vertex Drug Information. [cited 2016 Jan 2]. http://pi.vrtx.com/files/uspi-lumacaftor-ivacaftor.pdf.
16. Accurso FJ, Rowe SM, Clancy J, et al. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. New England J Med. 2010;363:1991-2003.
17. Ramsey BW, Davies J, McElvaney NG, et al. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. New England J Med. 2011;365:1663-1672.
18. 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;187:1219-1225.
19. De Boeck K, Munck A, Walker S, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis and a non-G551D gating mutation. J Cyst Fibros. 2014;13:674-680.
20. Moss RB, Flume PA, Elborn JS, et al. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an R117H-CFTR mutation: A double blind, randomised controlled trial. Lancet Respir Med. 2015;3:524-533.
21. Borowitz D, Lubarsky B, Wilschanski M, et al. Nutritional status improved in cystic fibrosis patients with the G551D mutation after treatment with ivacaftor. Dig Dis Sci. 2016;61:198-207.
22. McKone EF, Borowitz D, Drevinek P, et al. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: A phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014;2:902-910.
23. Barry PJ, Plant BJ, Nair A, et al. Effects of ivacaftor in cystic fibrosis patients carrying the G551D mutation with severe lung disease. Chest. 2014;146:152-158.
24. Taylor-Cousar J, Niknian M, GIlmartin G, et al. Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: safety and efficacy in an expanded access program in the United States. J Cyst Fibros. 2016;15:116-122.
25. Sawicki GS, McKone EF, Pasta DJ, et al. Sustained benefit from ivacaftor demonstrated by combining clinical trial and CF patient registry data. Am J Respir Crit Care Med. 2015;192:836-842.
26. Hayes D, McCoy KS, Sheikh SI. Improvement of sinus disease in cystic fibrosis with ivacaftor therapy. Am J of Respir Crit Care Med. 2014;190:468.
27. Hayes D, Warren PS, McCoy KS, et al. Improvement of hepatic steatosis in cystic fibrosis with ivacaftor therapy. J Pediatr Gastroenterol Nutr. 2015;60:578-579.
28. Safe M, Gifford AJ, Jaffe A, et al. Resolution of intestinal histopathology changes in cystic fibrosis after treatment with ivacaftor. Ann Am Thorac Soc. 2016;13:297-298.
29. Flume PA, Liou TG, Borowitz DS, et al Ivacaftor in subjects with cystic fibrosis who are homozygous for the F508del-CFTR mutation. Chest. 2012;142:718-724.
30. McColley SA. Combination lumacaftor and ivacaftor therapy for cystic fibrosis. Expert Opin Orphan Drugs. 2015. doi:10.1517/21678707.2016.1133282.
31. Wainwright CE, Elborn JS, Ramsey BW, et al. Lumacaftor-ivacaftor in patients with cystic fibrosis homozygous for Phe508del CFTR. New England J Med. 2015;373:220-231.
32. MedWatch The FDA Safety Information and Adverse Event Reporting Program. [cited 2016 Jan 2]. http://www.fda.gov/Safety/MedWatch/SafetyInformation/ucm429779.htm.
33. Kaminski R, Nazareth D. A successful uncomplicated CF pregnancy while remaining on ivacaftor. Journal of Cystic Fibrosis. 2016;15:133-134.
34. Sawicki GS, Dasenbrook E, Fink AK, et al. Rate of uptake of ivacaftor use after U.S. Food and Drug Administration approval among patients enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry. Ann Am Thorac Soc. 2015;12:1146-1152.
35. Cholon DM, Quinney NL, Fulcher ML, et al. Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis. Sci Transl Med. 2014 Jul 23;6(246):246ra96. doi:10.1126/scitranslmed.3008680.
36. Veit G, Avramescu RG, Perdomo D, et al. Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression. Sci Transl Med. 2014 Jul 23;6(246):246ra97. doi:10.1126/scitranslmed.3008889.