Ivacaftor

1).

ORKAMBI (lumacaftor/ivacaftor) is indicated for the treatment of cystic fibrosis (CF) in patients 1 year of age and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Limitations of Use The efficacy and safety of ORKAMBI have not been established in patients with CF other than those homozygous for the F508del mutation. 1 Pediatrics Pediatrics (<1 year of age): No data in patients younger than 1 year of age are available to Health Canada; therefore, Health Canada has not authorized the use of ORKAMBI in this age group.

2 Geriatrics Geriatrics (≥65 years of age): No data in patients aged 65 years and older are available to Health Canada; therefore, Health Canada has not authorized the use of ORKAMBI in this age group.

1).

1) and Clinical Studies (14) ] . If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

KALYDECO is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator indicated for the treatment of cystic fibrosis (CF) in patients aged 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data.

1 , 14 ) If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.

( 1 )