Vertex Pharmaceuticals announced that the Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in children ages 2–5 with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco is now approved for use in patients ages ≥2 with any of the following 10 CFTR mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D. This expanded age approval is based on previously announced results of an open-label Phase 3 24-week study that evaluated the safety and pharmacokinetics of weight-based dosing of Kalydeco (50mg or 75mg twice daily) in children ages 2–5.

RELATED: FDA Expands Kalydeco Use in Cystic Fibrosis

Kalydeco potentiates the CFTR protein, a chloride channel present at the surface of epithelial cells in multiple organs. It facilitates increased chloride transport by potentiating the channel-open probability of the CFTR protein.

A new weight-based oral granule formulation of Kalydeco 50mg and 75mg that can be mixed in soft foods or liquids will be available for children in this age group who may be unable to swallow a tablet. Kalydeco is also available as 150mg tablets in 50-count cartons and 60-count bottles.

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