Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del<i>CFTR</i>

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. (Funded by Vertex Pharmaceuticals; EVOLVE ClinicalTrials.gov number, NCT02347657 .).