Orkambi Indication Expanded to Include More Children With Cystic Fibrosis

The safety profile of Orkambi was found to be similar in patients aged 1 to less than 2 years compared with those 2 years of age and older.

The Food and Drug Administration (FDA) has expanded the approval of Orkambi® (lumacaftor/ivacaftor) to include children aged 12 to less than 24 months with cystic fibrosis who are homozygous for the F508del mutation (F/F genotype) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Previously, the treatment had been approved for patients 2 years of age and older.

The approval was supported by evidence from adequate and well-controlled studies of Orkambi in patients 12 years of age and older. Population pharmacokinetic analyses showed comparable drug exposure levels in patients 12 years and older and in those aged 1 through 11 years.

Additionally, the safety profile of Orkambi was found to be similar in patients aged 1 to less than 2 years and those 2 years of age and older based on data from a 24-week, open-label study (ClinicalTrials.gov Identifier: NCT03601637) that included 46 patients aged 1 to 2 years at screening. The most common adverse reactions reported in the trial were cough, infective pulmonary exacerbation of cystic fibrosis, pyrexia, and vomiting. Findings also showed reductions in sweat chloride concentration, though no direct correlation could be made between this and improvement in lung function.

Orkambi is supplied as tablets containing lumacaftor 100mg and ivacaftor 125mg, and lumacaftor 200mg and ivacaftor 125mg. For patients unable to swallow tablets, the oral granules formulation is available in unit-dose packets of lumacaftor 75mg and ivacaftor 94mg; lumacaftor 100mg and ivacaftor 125mg; and lumacaftor 150mg and ivacaftor 188mg.


  1. Vertex announces US FDA approval for Orkambi® (lumacaftor/ivacaftor) in children with cystic fibrosis ages 12 to <24 months. News release. September 2, 2022. https://www.businesswire.com/news/home/20220902005252/en/Vertex-Announces-U.S.-FDA-Approval-for-ORKAMBI%C2%AE-lumacaftorivacaftor-in-Children-With-Cystic-Fibrosis-Ages-12-to-24-months
  2. Rayment JH, Asfour F, Rosenfeld M, et al. A phase 3, open-label study of lumacaftor/ivacaftor in children 1 to less than 2 years of age with cystic fibrosis homozygous for F508delCFTR. American Journal of Respiratory and Critical Care Medicine. Published online June 29, 2022. doi:10.1164/rccm.202204-0734OC
  3. Orkambi. Package insert. Vertex; 2022. Accessed September 6, 2022. https://pi.vrtx.com/files/uspi_lumacaftor_ivacaftor.pdf