Overview of KALYDECO® Studies
How KALYDECO Was Studied
The mutations in the chart below have been studied with KALYDECO in a clinical study and/or in a laboratory setting.
CF Mutations Responsive to KALYDECO
Choose a mutation below for more information about how it was clinically studied with KALYDECO.
F508del and 26 other mutations are not responsive to KALYDECO based on in vitro assay and/or clinical data. (Click to view the complete list of mutations included in the full Prescribing Information).
Learn about how KALYDECO (ivacaftor) was studied in certain mutations below. Then read more about these studies on the following pages.
Studies 1 and 2 (G551D)
In Study 1
- 161 people with CF 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 CF 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
Study 4 (G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, S549R)
In Study 4
- 39 people with CF 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.
Study 5 (R117H)
In Study 5
- KALYDECO was studied in 69 people with CF 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
Study 6 (In children with CF age 2 to less than 6 years)
In Study 6
- KALYDECO oral granules (50 mg or 75 mg) were given every 12 hours with fat-containing food for 24 weeks. Children with CF 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.
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)
In Study 7
- In a study with two 8-week treatment periods, KALYDECO was studied in people with CF age 12 and older with 1 copy of the F508del mutation and a mutation predicted to be responsive to KALYDECO. People who took KALYDECO (150 mg) were compared to those who took placebo (sugar pill). Both KALYDECO and placebo were taken every 12 hours, with fat-containing food
In Studies 1, 2, and 4, people continued to take their other CF therapies, except hypertonic saline. In Studies 5, 6, and 7, people continued to take their other CF therapies, including hypertonic saline.
Laboratory Studies (A1067T, D110E, D110H, D1270N, E56K, E193K, F1052V, F1074L, G1069R, K1060T, R74W, and R1070Q)
In Laboratory Studies
- A1067T, D110E, D110H, D1270N, E56K, E193K, F1052V, F1074L, G1069R, K1060T, R74W, and R1070Q were determined to be responsive to KALYDECO based only on studies in a laboratory setting. Please talk to your healthcare provider and see the full Prescribing Information for additional detail on these mutations
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. Johnâ€™s wort.
Talk to your doctor before taking KALYDECO if you take any of these medicines or supplements.