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
Reference
- 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
