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Lumacaftor/ivacaftor

(Orkambi®)

Orkambi®

Drug updated on 9/4/2024

Dosage FormTablet (oral; lumacaftor/ivacaftor: 100/125 mg, 200/125 mg); Granules (oral; lumacaftor/ivacaftor: 75/94 mg, 100/125 mg, 150/188 mg)
Drug ClassCFTR potentiators
Ongoing and
Completed Studies		ClinicalTrials.gov

Indication

  • Indicated for the treatment of cystic fibrosis (CF) in patients age aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.

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Summary
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  • Orkambi (lumacaftor/ivacaftor) is indicated for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who are homozygous for the F508del mutation in the CFTR gene.
  • This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
  • Lumacaftor/Ivacaftor (LUM/IVA): Dual therapy improved FEV1 % predicted compared to placebo, with meta-analysis showing a mean difference (MD) of 2.38 (1.62-3.15, P<0.00001); triple therapy demonstrated greater relative and absolute changes in FEV1 % predicted, and significant improvement in BMI (MD 0.21, 0.03-0.39, P=0.02).
  • Tezacaftor/Ivacaftor (TEZ/IVA): Dual therapy improved FEV1 % predicted compared to placebo at all time points, with significant improvements in ppFEV1 in meta-analysis (P=0.002); no reported increases in blood pressure in RCTs.
  • Elexacaftor-Tezacaftor-Ivacaftor (ELX/TEZ/IVA): Triple therapy resulted in greater relative and absolute changes in FEV1 % predicted, significant improvements in BMI, and little to no difference in adverse events compared to control in individuals 12 years and older.
  • LUM/IVA: Higher frequency of respiratory-related adverse events (AEs) and discontinuations were observed in real-world studies, with RCTs showing increased respiratory AEs compared to placebo, including early transient breathlessness and increased systolic and diastolic blood pressure over 120 weeks. Meta-analysis revealed an increased proportion of discontinued treatments due to AEs (OR 2.71, 1.3-5.63, P=0.008).
  • TEZ/IVA: No significant impact on the number of participants reporting AEs in meta-analysis (P=0.53); RCTs reported no increases in blood pressure and one death deemed unrelated to the study drug.
  • Treatment effects varied significantly by genotype, particularly for F508del, G551D, and R117H mutations, and age-specific outcomes showed differing effectiveness and safety profiles, especially noting a lack of data in children under 12 years for some therapies. Differences were also observed between real-world studies and RCTs in the frequency and types of adverse events reported.