Study 7

Study 7 (2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, E831X, A455E, D1152H, D579G, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, and S977F)

KALYDECO was compared with placebo (sugar pill) in a clinical trial of people with certain CFTR mutations predicted to respond to KALYDECO.

  • The study included people with CF age 12 years and older with 1 copy of the F508del mutation and a second mutation that is predicted to respond to KALYDECO
    • Number of people who contributed to the data for placebo: 161
    • Number of people who contributed to the data for KALYDECO: 156
  • People took 1 tablet of KALYDECO (150 mg) or placebo every 12 hours, with fat-containing food, along with their other prescribed CF therapies for 8 weeks
  • The main endpoint studied was lung function (FEV1)*; other endpoints included were CF respiratory symptoms (CFQ-R Respiratory Domain Score) and safety

*FEV1, forced expiratory volume in 1 second.

CFQ-R, Cystic Fibrosis Questionnaire-Revised.

Considerations

  • Some but not all of the 28 approved mutations that are predicted to respond to KALYDECO were enrolled in Study 7. Please see the full Prescribing Information or talk to your healthcare provider to learn which mutations were studied
  • This is not intended to represent the full study design or results
  • Results may differ among patients and mutations

KALYDECO improved lung function* compared with placebo overall in Study 7

Improvement in lung function was measured as the difference in FEV1* from the start of the study to the average of Week 4 and Week 8.

*Lung function was measured as FEV1, or forced expiratory volume exhaled in 1 second.

People kept taking their other cystic fibrosis (CF) therapies, except inhaled hypertonic saline.

KALYDECO improved CF respiratory symptoms compared with placebo overall in Study 7

People who took KALYDECO were more likely to report less coughing, less mucus, and less trouble breathing after averaging the measurements taken of Week 4 and Week 8.

CF respiratory symptoms (such as coughing, sputum production, and difficulty breathing) were scored based on the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score.

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have at least one mutation in their CF gene that is responsive to KALYDECO.

Talk to your doctor to learn if you have an indicated CF gene mutation.

It is not known if KALYDECO is safe and effective in children under 2 years of age.

Important Safety Information

Who should not take KALYDECO?

Do not take KALYDECO if you take certain medicines or herbal supplements such as: the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®); seizure medications such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, Equetro®) or phenytoin (Dilantin®, Phenytek®); or St. John’s wort.

Talk to your doctor before taking KALYDECO if you take any of these medicines or supplements.