FDA Expands Kalydeco Use in Cystic Fibrosis

Vertex announced that the Food and Drug Administration (FDA) has approved a supplemental new drug application (sNDA) for use of Kalydeco (ivacaftor) in patients with cystic fibrosis (CF) ages ≥6 years who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Kalydeco is now approved for use in patients with any of the following 10 CFTR mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The expanded approval is following a recommendation from the FDA’s Pulmonary-Allergy Drugs Advisory Committee (PADAC). It is supported by data from a Phase 3 trial that studied 69 patients with CF ages ≥6 years with the R117H mutation.

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.

Kalydeco is available as 150mg tablets in 50-count cartons and 60-count bottles.

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