A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation

This was a Phase 3, randomized, double-blind, placebo-controlled, parallel-group multicenter
study of orally administered lumacaftor in combination with ivacaftor in participants aged
12 years and older with CF who are homozygous for the F508del-CFTR mutation.

The study included a Screening Period (Day -28 through Day -1), a Treatment Period (Day 1
[first dose of study drug] to Week 24 ± 5 days), and a Safety Follow-up Visit (4 weeks ± 7
days after the Week 24 Visit).

Inclusion Criteria:

- Confirmed diagnosis of CF

- Homozygous for the F508del CFTR mutation

- Forced expiratory volume in 1 second (FEV1) greater than or equal to (>=) 40 percent
(%) and less than or equal to (=<) 90% of predicted normal for age, sex, and height

- Willing to remain on a stable CF medication regimen through Week 24 or, if
applicable, the Safety Follow up Visit

Exclusion Criteria:

- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in
therapy (including antibiotics) for pulmonary disease within 4 weeks before first
dose of study drug

- History of solid organ or hematological transplantation

- History of alcohol or drug abuse in the past year

- Ongoing or prior participation in an investigational drug study (including studies
investigating lumacaftor and/or ivacaftor) within 30 days of screening.

- Use of strong inhibitors, moderate inducers, or strong inducers of Cytochrome P450 3A
(CYP3A) within 14 days before Day 1 of dosing