Overview of KALYDECO® Studies

KALYDECO is for people with cystic fibrosis (CF) age 12 months and older with at least 1 mutation in their CF gene responsive to KALYDECO. It is not known if KALYDECO is safe and effective in younger children.

Who is KALYDECO for?

KALYDECO is for the treatment of cystic fibrosis (CF) in people age 12 months and older who have one of the following 38 CF gene mutations:

2789+5G→A D110H F1052V G551S R117H S549R
3272-26A→G D1152H F1074L K1060T R347H S945L
3849+10kbC→T D1270N G1069R L206W R352Q S977F
711+3A→G D579G G1244E P67L R74W  
A1067T E193K G1349D R1070Q S1251N  
A455E E56K G178R R1070W S1255P  
D110E E831X G551D R117C S549N  
2789+5G→A E831X R1070W
3272-26A→G F1052V R117C
3849+10kbC→T F1074L R117H
711+3A→G G1069R R347H
A1067T G1244E R352Q
A455E G1349D R74W
D110E G178R S1251N
D110H G551D S1255P
D1152H G551S S549N
D1270N K1060T S549R
D579G L206W S945L
E193K P67L S977F
E56K R1070Q  
  • The following mutations were not included in the clinical studies but are predicted to respond based on results from a laboratory setting: A1067T, D110E, D110H, D1270N, E56K, E193K, F1052V, F1074L, G1069R, K1060T, R74W, and R1070Q
  • F508del and 26 other mutations are not responsive to KALYDECO based on clinical and/or laboratory data

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 12 months of age.

Keep in mind that not all mutations were evaluated in clinical studies, and those that were evaluated were not studied in all age groups. However, KALYDECO is approved for people age 12 months and older with any of the 38 indicated mutations listed above.

How was KALYDECO studied?

The effect of KALYDECO on several different measures has been studied in the KALYDECO studies.

lung function

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second.

sweat chloride levels

Sweat chloride is a measure of the amount of salt in your sweat. People with CF have high levels of sweat chloride.

body mass index

BMI is a measure of someone’s weight in relation to his or her height.

CF respiratory symptoms

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

pulmonary exacerbations

Pulmonary exacerbations are defined as changes in certain symptoms requiring changes in the use of oral, IV, or inhaled antibiotics.

Keep in mind that not all studies evaluated the same measures, and results in studies with KALYDECO are an average of all people studied and differed among individuals and mutations. Your experience may be different.

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 months 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 12 months of age.

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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 medicines such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

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

EXPAND

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 months 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 12 months 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 medicines such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

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

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®)
  • 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
  • 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
  • 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), including sore throat, nasal or sinus congestion, runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • nausea
  • 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 are encouraged to report side effects to FDA at 1-800-FDA-1088.

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