The US Food and Drug Administration approved Kalydeco for use in children with cystic fibrosis ages 1 month to less than 4 months old who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.
Kalydeco (Vertex Pharmaceuticals) is already approved in the US and EU for the treatment of cystic fibrosis in patients ages 4 months and older.
“Treating the underlying cause of cystic fibrosis as early as possible is important, and this approval, the first for a CFTR modulator in this age group, means families will now have a medicine for eligible infants,” says Carmen Bozic, MD, executive vice president, global medicines development and medical affairs, and chief medical officer at Vertex, in a release.
The approval was supported by a cohort in a phase 3, 24-week, open-label study to evaluate the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in subjects with cystic fibrosis who are less than 24 months of age and have an ivacaftor-responsive CFTR mutation. This cohort demonstrated a safety profile similar to that observed in older children and adults.
“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,” says Margaret Rosenfeld, MD, 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, in a release. “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.”
Kalydeco was first approved in 2012 in the US and is now available in more than 30 countries.