Vertex Pharma’s KALYDECO gets FDA approval for use in infants with CF
Vertex Pharma’s (NASDAQ:VRTX) KALYDECO obtained FDA approval for use in infants with cystic fibrosis (CF) between the ages of six and 12 months.
KALYDECO is indicated in CF patients who have at least one mutation in the cystic fibrosis transmembrane conductance regulator gene that is responsive to the drug. KALYDECO is already approved in the U.S., Canada and the EU for the treatment of CF patients one year of age and older.
“Today’s approval for KALYDECO allows physicians to begin treating the underlying cause of CF in eligible infants as young as six months of age for the first time, with the potential to modify the course of the disease,” University of Washington School of Medicine’s Dr. Margaret Rosenfeld said in a statement.
The approval was based on safety data from Vertex’ Phase 3 trial in 11 infants between the ages of six and 12 months, which demonstrated a similar safety profile to that observed in previous Phase 3 studies of older children and adults.