FDA OKs Vertex KALYDECO to Treat Infants with Cystic Fibrosis

Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced the U.S. Food and Drug Administration (FDA) approved KALYDECO^® (ivacaftor) for use in children with cystic fibrosis (CF) ages six months to less than 12 months 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 is already approved in the U.S., Canada and EU for the treatment of CF in patients ages 12 months 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,” said Margaret Rosenfeld, M.D., MPH, Seattle Children’s Research Institute and Department of Pediatrics, University of Washington School of Medicine.

This FDA approval is based on data from a 24-week Phase 3 open-label safety cohort (ARRIVAL) of 11 children with CF aged six months to less than 12 months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). The study demonstrated a safety profile similar to that observed in previous Phase 3 studies of older children and adults; most adverse events were mild or moderate in severity, and no patient discontinued therapy due to adverse events. The most common adverse events (≥30%) were cough (64%), nasal congestion (36%) and rhinorrhea (36%). Three serious adverse events, all considered unrelated to study treatment by the investigators, were observed in three patients.

https://www.biospace.com/article/releases/fda-approves-kalydeco-ivacaftor-as-first-and-only-cftr-modulator-to-treat-eligible-infants-with-cf-as-early-as-six-months-of-age/