KALYDECO® (ivacaftor) Study Results for 9 Additional Mutations

Study 4 (9 Additional Mutations)

In Study 4

  • 39 people with a G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, or S549R mutation who were 6 years of age or older took KALYDECO or placebo (sugar pill) every 12 hours with fat-containing food. Each person had an 8-week treatment period with KALYDECO and an 8-week treatment period with placebo

*In this study, efficacy could not be established in people with the G970R mutation. KALYDECO is not indicated for people with CF who have the G970R mutation.

Improvement in lung function through 8 weeks of treatment

Overall, people who took KALYDECO had an improvement in lung function through 8 weeks of treatment

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.

Other study results

Overall, people who took KALYDECO experienced fewer CF respiratory symptoms through 8 weeks of treatment

Study 4: people 6 years of age or older

boy-coughing

In the overall population for the 9 mutations studied, people who took KALYDECO reported less coughing, less mucus, and less trouble breathing compared with people who took placebo.

Respiratory symptom results varied across the different mutations studied

  • Not all mutations showed the same level of benefit
  • Some patients experienced less improvement in CF respiratory symptoms compared with the average for all patients in the study. Others experienced more improvement
  • Talk to your doctor to learn more about your specific mutation

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.

Overall, people who took KALYDECO experienced an increase in body mass index (BMI) after 8 weeks of treatment

Study 4: people 6 years of age or older

Scale with Ruler

In the overall population for the 9 mutations studied, people who took KALYDECO had a 0.66 kg/m2 average increase in BMI compared with people who took placebo.

  • BMI is a measure of a person’s weight relative to height
  • For example, if you are a 5’4”, 110-lb adult, this would be a gain of about 4 lbs; if you are a 4’6”, 65-lb child, this would be a gain of about 3 lbs

BMI results varied across the different mutations studied

  • Not all mutations showed the same level of benefit
  • Some patients experienced a smaller increase in BMI compared with the average for all patients in the study. Others experienced a larger increase
  • Talk to your doctor to learn more about your specific mutation

People who took KALYDECO had less salty sweat after 8 weeks of treatment

Study 4

Sweat Chloride

In the overall population for the 9 mutations studied, people who took KALYDECO had a 50 mmol/L average decrease in sweat chloride compared with people who took placebo.

Sweat chloride level results varied across the mutations studied

  • Not all mutations showed the same level of benefit
  • Some people experienced less improvement in sweat chloride levels compared with the average for all patients in the study. Others experienced more improvement
  • Talk to your healthcare provider to learn more about your specific mutation

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 one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.

KALYDECO is used for the treatment of CF in patients age 2 years and older who have an R117H mutation in their CF gene.

KALYDECO is not for use in people with CF due to other mutations in the CF gene. KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.

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.