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# Vertex sNDA for Expanded Kalydeco Use Approved
- URL: https://www.fdaweb.com/vertex-snda-for-expanded-kalydeco-use-approved/
- Published: 2019-04-30T12:00:00.000Z
- Updated: 2026-09-15T01:23:34.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5143989

FDA has approved a Vertex Pharmaceuticals’ supplemental NDA for Kalydeco (ivacaftor) to expand its 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 the drug based on clinical 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.

Expanded approval was based on data from a 24-week Phase 3 open-label safety cohort (ARRIVAL) involving 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 company says 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, it says.