Vertex announced that the Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in patients with cystic fibrosis (CF) ≥2 years old who have one of five residual function mutations (2789+5G—>A, 3272-26A—>G, 3849+10kbC—>T, 711+3A—>G, and E831X) that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Today’s FDA approval follows the Agency’s recent approval for 23 other residual function mutations based on in vitro data. Kalydeco is now indicated to treat patients with CF aged ≥2 years who have one of 38 ivacaftor-responsive mutations in the CFTR gene. 

The five mutations were evaluated in the Phase 3, 8-week crossover EXPAND study in patients who have one mutation that results in residual CFTR function and one copy of the F508del mutation. The data showed that monotherapy with Kalydeco achieved the primary efficacy endpoint of absolute improvement in ppFEV1 and was well tolerated. 

Kalydeco, a CFTR potentiator, works by keeping the CFTR proteins at the cell surface open longer to enhance salt and water transport across the membrane.

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The product is supplied as 150mg strength tablets to be taken with fat-containing food. It is also available as 50mg and 75mg strength oral granulesto be administered with soft food or liquid with fat-containing food. 

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