Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation

Ivacaftor is the first CFTR modulator to show an improvement in CFTR function and clinical
benefit in patients with CF. Results from Phase 3 studies (VX08-770-102 [Study 102] and
VX08-770-103 [Study 103]) showed that ivacaftor is effective in the treatment of patients
with CF who have the G551D-CFTR mutation, as evidenced by sustained improvements in CFTR
channel function (measured by reduction in sweat chloride concentration) and corresponding
substantial, durable improvements in lung function, pulmonary exacerbations, respiratory
symptoms, and weight gain. Ivacaftor was also well tolerated, as evidenced by the rates and
reasons for premature discontinuation and results of safety assessments.

Ivacaftor (Trade Name Kalydeco; 150 mg tablets) was initially approved in the United States
for the treatment of CF in patients 6 years of age and older who have a G551D mutation in
the CFTR gene.

Inclusion Criteria:

- Subjects from Study 110 or Study 111 entering the ivacaftor arm must have completed
the assigned study drug treatment duration in the previous study

- Subjects from Study 113 entering the ivacaftor arm must have completed all study
related treatments through the Follow-up Visit and met the Study 113 responder
criteria during the previous study.

- Subjects entering the observational arm must have completed at least 4 weeks of study
drug treatment in their previous study (Study 110 or Study 111), must have completed
the previous study but do not wish to enroll in the ivacaftor arm, or must have
completed the previous study but do not meet the inclusion criteria of the ivacaftor
arm.

- Females of childbearing potential entering the ivacaftor arm must not be pregnant

- Subjects entering the ivacaftor arm must be willing to comply with contraception
requirements

Exclusion Criteria (Ivacaftor Arm Only):

- History of any illness or condition that might confound the results of the study or
pose an additional risk in administering ivacaftor to the subject

- Use of moderate or strong inhibitors or inducers of cytochrome P450 (CYP) 3A

- Evidence of cataract or lens opacity at or before the Day 1 Visit