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Clinical management of CF » CFTR modulators » CFTR modulators approved » About ivacaftor

About ivacaftor

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In Europe and the USA, ivacaftor is a prescription medicine used for the treatment of CF in individuals who are aged 6 years and older, and have at least one copy of the CFTR mutation, G551D, or any of the following gating mutations: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. In Canada, the same mutations apply in addition to G907R for the treatment of CF in individuals who are aged 6 years and older.

Ivacaftor facilitates increased Cl transport by potentiating the channel-open probability (or gating) of the G551D-CFTR protein.1,2
Its exact MoA is unknown; however, it has been suggested that ivacaftor may bind at a site close to the G551D mutation located on nucleotide-binding domain (NBD) of CFTR.3,4

Clinical trials showed that ivacaftor led to improvements in certain quality of life measurements compared with placebo:5, 6

  • Sustained improvements in lung function including a reduction in pulmonary exacerbations
  • Weight gain
  • Reduced hospitalisations

There were no significant safety signals identified in the pivotal trials and an ongoing study is assessing the long-term safety of ivacaftor.7

References
  1. Ramsey BW et al. N Engl J Med 2011;365:1663–72
  2. Accurso FJ et al. N Engl J Med 2010;363:1991–2003
  3. Van Goor et al. Proc Natl Acad Sci USA 2009;106:18825–30
  4. Bompadre S et al. J Gen Physiol 2007;129:285–98
  5. Ramsey BW et al. N Engl J Med 2011;365:1663–72
  6. Accurso FJ et al. N Engl J Med 2010;363:1991–2003
  7. Clinicaltrials.gov. http://clinicaltrials.gov/ct2/show/NCT01117012