Combination treatment with the cystic fibrosis transmembrane conductance regulator (CFTR) modulators tezacaftor and ivacaftor are designed to target the underlying cause of disease in patients with cystic fibrosis.
Receive either 100 mg of tezacaftor once daily and 150 mg of ivacaftor twice daily.
The rate of pulmonary exacerbation was 35% lower in the tezacaftor–ivacaftor group than in the placebo group.
Fewer patients in the tezacaftor–ivacaftor group than in the placebo group had respiratory adverse events, none of which led to discontinuation.
The combination of tezacaftor and ivacaftor was efficacious and safe in patients 12 years of age or older who had cystic fibrosis and were homozygous for the CFTR Phe508del mutation.