This notice was prepared by Dale Whitby, PharmD and Jen Seeba, PharmD (PPAG Members)
On March 18, 2015 the FDA expanded approval of ivacaftor for the treatment of children ages two to five years with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor is the first available agent that targets the defective CFTR protein in patients with CF and was previously approved for use in patients 6 years and older.
Ivacaftor is approved for use in those patients with one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, orR117H. Of note, it is not effective in patients who are homozygous for the F508delmutation, the most frequent genotype in patients with CF accounting for approximately two-thirds of mutations in the North American and northern European populations.
Ivacaftor is currently available as 150-mg tablets. An oral granule formulation that can be mixed in soft food or liquid will also be available to aid administration in young children; this formulation will be available in 50-mg or 75-mg unit-dose packets.
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