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.

KALYDECO CFTR mutations chart

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

See the results from these studies.

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.

See the results from this study.

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

See more information about this study.

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.

See the results from this study.

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

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, 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. John’s wort.

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

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.

What should I tell my doctor before taking KALYDECO?

Before you take KALYDECO, tell your doctor if you:

  • have liver or kidney problems
  • drink grapefruit juice, or eat grapefruit or Seville oranges
  • are pregnant or plan to become pregnant. It is not known if KALYDECO will harm your unborn baby. You and your doctor should decide if you will take KALYDECO while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if KALYDECO passes into your breast milk. You and your doctor should decide if you will take KALYDECO while you are breastfeeding

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements as the dose of KALYDECO may need to be adjusted when taken with certain medications.

Especially tell your doctor if you take antifungal medications such as ketoconazole (e.g., Nizoral®), itraconazole (e.g., Sporanox®), posaconazole (e.g., Noxafil®), voriconazole (e.g., Vfend®), or fluconazole (e.g., Diflucan®); or antibiotics such as telithromycin (e.g., Ketek®), clarithromycin (e.g., Biaxin®), or erythromycin (e.g., Ery-Tab®).

What should I avoid while taking KALYDECO?

KALYDECO can cause dizziness in some people who take it. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how KALYDECO affects you.

You should avoid food containing grapefruit or Seville oranges while you are taking KALYDECO.

What are the possible side effects of KALYDECO?

  • KALYDECO can cause serious side effects
  • High liver enzymes in the blood have been reported in patients receiving KALYDECO
    • Your doctor will do blood tests to check your liver before you start KALYDECO, every 3 months during your first year of taking KALYDECO, and every year while you are taking KALYDECO
    • For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often
    • Call your doctor right away if you have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of your skin or the white part of your eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine
  • Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts
  • The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness

Tell your doctor if you have any side effect that bothers you or that does not go away. These are not all the possible side effects of KALYDECO. For more information, ask your doctor or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Click here for KALYDECO full Prescribing Information, including Patient Information.