AKA Orkambi (lumacaftor/ivacaftor)

• Drug used in the treatment of Cystic Fibrosis
• transmembrane conductance regulator potentiator
• Approved for ages 2 and older
• Review

• Cataracts were found in juvenile rats in pre-clinical toxicology studies
• Cataracts have been discovered in a few children taking this drug. The concomitant use of steroids or other pre-existing coniditions could not be excluded.
  • Lumacaftor/Ivacaftor in Patients Aged 6–11 Years with Cystic Fibrosis and Homozygous for F508del-CFTR
  • The impact of 12 months treatment with ivacaftor on Scottish paediatric patients with cystic fibrosis with the G551D mutation: a review
• Drug manufacturer recommends baseline and follow-up eye examinations of unspecified interval or duration
  • Prescribing Information