Kalydeco Approved for Cystic Fibrosis in Children as Young as 1 Month Old

Kalydeco approval has been expanded to include children 1 month to less than 4 months old with cystic fibrosis.

The Food and Drug Administration (FDA) has approved Kalydeco® (ivacaftor) for use in children with cystic fibrosis (CF) ages 1 month to less than 4 months old who have at least 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to ivacaftor, based on clinical and/or in vitro assay data. Previously, the treatment was not indicated for patients younger than 4 months old.

The approval was based on data from a phase 3 open-label study that evaluated the safety, pharmacokinetics and pharmacodynamics of ivacaftor in CF patients who were less than 24 months of age (ClinicalTrials.gov Identifier: NCT02725567). Findings showed that exposure of ivacaftor in pediatric patients 1 month to less than 24 months of age was within the range of exposure in adults and pediatric patients 6 years of age and older. Additionally, the safety profile was observed to be similar between these younger patients and patients 2 years and older.

Kalydeco is supplied in both 150mg tablets and oral granules containing 5.8mg, 13.4mg, 25mg, 50mg, and 75mg per unit-dose packet. The recommended dose for pediatric patients is based on weight.

“As a physician caring for infants and children with cystic fibrosis, I see the importance of initiating therapies early in life that may slow disease progression,” said Margaret Rosenfeld, M., MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine and one of the Principal Investigators for the Kalydeco® study in less than 24-month-olds. “Today’s approval provides many families and caregivers comfort in knowing that there is a highly effective modulator therapy available for their babies with CF.”

This article originally appeared on MPR


  1. Vertex announces US FDA approval for Kalydeco® (ivacaftor) to treat eligible infants with CF ages 1 month and older. News release. Vertex. March 3, 2023. https://www.businesswire.com/news/home/20230503006018/en/Vertex-Announces-U.S.-FDA-Approval-for-KALYDECO%C2%AE-ivacaftor-to-Treat-Eligible-Infants-With-CF-Ages-1-Month-and-Older.
  2. Kalydeco. Package insert. Vertex; 2023. Accessed May 4, 2023. https://pi.vrtx.com/files/uspi_ivacaftor.pdf.