Overview of KALYDECO® (ivacaftor) Studies

How KALYDECO Was Studied

KALYDECO has been studied in people with one of the following mutations in their cystic fibrosis (CF) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, R117H, G970R,* and F508del.

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

KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.

Learn about how it was studied below. Then read more about the results of these studies on the following pages.

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 or placebo every 12 hours with fat-containing food for 48 weeks

See the results from these studies.

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.

See the results from this study.

Study 5 (R117H)

In Study 5

  • KALYDECO was studied in 69 people age 6 years and older with an R117H mutation in the CF gene. These people took KALYDECO or placebo every 12 hours with fat-containing food for 24 weeks

See the results from this study.

Study 6 (In patients age 2 to less than 6 years)

In this open-label safety study, 34 children age 2 to less than 6 years of age were given KALYDECO oral granules (50 mg or 75 mg) every 12 hours with fat-containing food for 24 weeks. Patients with the following mutations were eligible for this study: G551D, G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, or S549R. A total of 32 had the G551D mutation and 2 had the S549N mutation. Safety and tolerability was the main endpoint, or measure, of the pediatric clinical study (Study 6).

*KALYDECO is not indicated for people with CF who have the G970R mutation.

See the results from this study.

In Studies 1, 2, and 4, people continued to take their other CF therapies, except hypertonic saline. In Studies 5 and 6, people continued to take their other CF therapies, including hypertonic saline.

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.