Vertex announced that the Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in patients with cystic fibrosis (CF) aged ≥2 years who have one of 23 residual function mutations in the CF transmembrane conductance regulator (CFTR) gene. 

The expanded indication now increases the the number of treatable mutations from 10 to 33. The FDA’s decision was based on analyses of in vitro data and real-world clinical data spanning over five years on the safety and efficacy of Kalydeco. Thein vitro cell-based model system was shown to reasonably predict clinical response to Kalydeco. Similar to the other mutations for which Kalydeco is approved to treat, the mutations included in the newest approval are known to have some CFTR protein on the cell surface and have shown in vitro increases in chloride transport as a response to Kalydeco by ≥10% of normal over baseline.  

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Kalydeco, a CFTR potentiator, was also evaluated in the Phase 3 EXPAND study that included patients with five “splice” mutations that are responsive to Kalydeco. The Company is in discussion with the FDA for the approval to treat patients with these mutations. 

Kalydeco is available as 150mg strength tablets and as 50mg and 75mg strength oral granule packets.

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