KALYDECO® (ivacaftor) Study Results for People With the G551D Mutation

Studies 1 and 2 (G551D)

In Study 1

  • 161 people with a G551D mutation who were 12 years of age or older took KALYDECO or placebo (sugar pill) every 12 hours with fat-containing food for 48 weeks

In Study 2

  • 52 people with a G551D mutation who were 6 to 11 years of age took KALYDECO every 12 hours with fat-containing food for 48 weeks

Improvement in lung function through 24 weeks of treatment

In 2 clinical studies (Studies 1 and 2): People who took KALYDECO had an improvement in lung function*

*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 through the entire length of the study (48 weeks).

Improvement in lung function through 48 weeks of treatment

Other study results

Changes in CF respiratory symptoms through 48 weeks of treatment with KALYDECO compared with people who took placebo

Study 1: people 12 years of age or older

boy-coughing

People in the study who took KALYDECO reported less coughing, less mucus, and less trouble breathing compared with people who took placebo.

Study 2: people 6 to 11 years of age

girl-coughing

People who took KALYDECO reported no difference in coughing, mucus, and trouble breathing compared with people who took placebo.

Assessed by the Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score, a measure of respiratory symptoms relevant to people with CF such as cough, sputum production, and difficulty breathing.

Less chance of having a pulmonary exacerbation through 48 weeks of treatment compared with placebo

Study 1: people 12 years of age or older

Woman with Lungs
  • Nearly 7 out of 10 people who took KALYDECO did not have a pulmonary exacerbation
  • In contrast, approximately 4 out of every 10 patients taking placebo did not have a pulmonary exacerbation
  • A pulmonary exacerbation was any new, or change in, antibiotics that people had to take because of 4 or more symptoms involving their sinuses or lungs

Pulmonary exacerbation was not evaluated in Study 2.

People who took KALYDECO gained more weight after 48 weeks of treatment compared with placebo

Study 1: people 12 years and older;
Study 2: people 6 to 11 years of age

scale

Weight increased about 6 pounds for people on KALYDECO.

People who took KALYDECO had less salty sweat after 48 weeks of treatment compared with people who took placebo

A decrease in sweat chloride levels does not necessarily mean there will be an improvement in lung function (FEV1).

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.