A phase 3 clinical trial, ivacaftor (Kalydeco, Vertex Pharma) was generally safe to administer to infants aged 1-month to <4-months old, according to research published in Journal of Cystic Fibrosis.

According to results, ivacaftor improved CFTR function, pancreatic function, intestinal inflammation, and growth parameters over the 24-week treatment period.1

Adverse events were reported in four of seven infants (57.1%), but researchers say these were all mild in severity and none were considered related to ivacaftor.1

“Since pharmacokinetics was predictable, an ivacftor dosing regimen in infants 1 to <4 months of age based on weight and age is proposed,” the researchers concluded.1

Read the full article at www.cysticfibrosisjournal.com


  1. McNally P, et al. Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis. J of CF. 2024, April 4. doi:https://doi.org/10.1016/j.jcf.2024.03.012