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Clinical management of CF » CFTR modulators » CFTR modulators approved

CFTR modulators approved

The following CFTR modulator is currently approved for clinical use.

Ivacaftor

Ivacaftor (previously VX-770, commercialised as Kalydeco™) is currently the only approved CFTR potentiator by the US FDA and European Commission for the treatment of patients with CF aged 6 years and older who have at least one copy of the G551D mutation or any of the following gating mutations: G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.1,2 In Canada, ivacaftor was approved for use in patients with CF aged 6 years and older who have the aforementioned CTFR mutations in addition to G970R.2

Ivacaftor and lumacaftor combination
In July 2015, the US FDA approved the first drug,
lumacaftor 200 mg/ivacaftor 125 mg, for the treatment of patients with CF aged 12 years and older who have two copies of the F508del mutation.3 Patients with two copies of this mutation (one inherited from each parent) represent the largest population of CF patients.3

References
  1. US Food & Drug Administration Approves Kalydeco™ (ivacaftor) for Use in Eight Additional Mutations that Cause Cystic Fibrosis. 21 February 2014. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=827435
  2. Vertex Receives European Approval for Kalydeco™ (ivacaftor) in Eight Non-G551D Gating Mutations. http://investors.vrtx.com/releasedetail.cfm?ReleaseID=863535
  3. FDA News Release. FDA approves new treatment for cystic fibrosis. Available at: http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm453565.htm. July 2, 2015