Frequently Asked Questions

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.

Learn the answers to some of the most frequently asked questions about KALYDECO®. If you have more questions, you should talk to your doctor or CF care team.

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.

CF Mutations Responsive to KALYDECO

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

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 (Hypericum perforatum).

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

No, KALYDECO is not a cure for CF.

KALYDECO affects the CFTR protein "gating" in people with at least one mutation in their CF gene that is responsive to KALYDECO. KALYDECO helps the "gates" of the CFTR proteins stay open longer, allowing more chloride ions to move into and out of the cells. The movement of chloride ions may help keep a balance of salt and water in the lungs. KALYDECO does not increase the amount of CFTR proteins at the cell surface.

What is known about how KALYDECO works was learned from studies conducted in a laboratory. Keep in mind that results from laboratory studies do not always match how these medicines work in a person. If you have any questions about your treatment, please speak with your healthcare provider.

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®).

Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine or stop taking a medicine.

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.

  • 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, your 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.

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, your 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

Take KALYDECO orally every 12 hours with fat-containing food unless otherwise directed by your healthcare provider.

KALYDECO should be taken with fat-containing food because fat-containing food helps the body absorb KALYDECO better.

If you have questions about KALYDECO, please talk to your doctor. You can also find information about KALYDECO in the full Prescribing Information, including Patient Information.

Prescriptions for KALYDECO can only be filled at authorized specialty pharmacies. If you'd like to see a list of specialty pharmacies and specialty distributors, click here. Based on your health plan, there may be restrictions on which pharmacies you can use.

You may cut along the dotted line to separate your doses from the blister card.

KALYDECO should be stored at 68°F to 77°F (20°C to 25°C).

Do not use KALYDECO after the expiration date on the package.

Keep KALYDECO and all medicines out of the reach of children.

For people 6 years and older, KALYDECO tablets are supplied as light blue, film-coated, capsule-shaped tablets containing 150 mg of ivacaftor. Each tablet is printed with the characters "V 150" on one side and plain on the other.

KALYDECO® (ivacaftor) 150 mg tablets packaging

Not actual size.

If you miss a dose of KALYDECO and it is within 6 hours of when you usually take it, take the dose of KALYDECO as prescribed with fat-containing food as soon as possible.

If you miss a dose and it is more than 6 hours after the time you usually take it, skip that dose only and take the next dose with fat-containing food when you usually take it.

Do not take 2 doses at the same time to make up for a missed dose.

If you have questions about KALYDECO, please talk to your doctor. You can also find information about KALYDECO in the full Prescribing Information, including Patient Information.

KALYDECO should be stored at room temperature between 68°F to 77°F (20°C to 25°C).

Do not use KALYDECO after the expiration date on the package.

Keep KALYDECO and all medicines out of the reach of children.

KALYDECO is supplied as oral granules (50 mg or 75 mg packets) for people age 12 months to less than 6 years.

KALYDECO 50 mg and 75 mg oral granules packaging

Not actual size.

The recommended dose of KALYDECO (oral granules) for children age 12 months to less than 6 years is weight-based. Children age 12 months to less than 6 years who weigh between ~15 lbs and less than ~31 lbs (7 kg to less than 14 kg) should take one 50 mg packet mixed with one teaspoon (5 mL) of soft food or liquid every 12 hours for a total daily dose of 100 mg.

Children age 12 months to less than 6 years who weigh ~31 lbs (14 kg) or more should take one 75 mg packet mixed with one teaspoon (5 mL) of soft food or liquid every 12 hours for a total daily dose of 150 mg.

Your child's dose may be different. Your doctor should tell you how much KALYDECO your child should take and when to take it.

If your child misses a dose of KALYDECO and it is within 6 hours of when your child usually takes it, give the dose of KALYDECO as prescribed with fat-containing food as soon as possible.

If your child misses a dose and it is more than 6 hours after the time your child usually takes it, skip that dose only and give the next dose with fat-containing food when your child usually takes it.

Do not give 2 doses at the same time to make up for a missed dose.

Yes, you can mix KALYDECO granules with fat-containing food, for example, 1 teaspoon (5 mL) of whole-milk yogurt or milk, to give to your child. But your child should still eat fat-containing food just before or after taking the spoonful of the mixture.

The granules should be mixed with soft food or liquid at room temperature or below. The granules should not be mixed in items that are frozen or hot.

The granules should be taken within 1 hour of mixing with soft food or liquid. Make sure all medicine is taken.

The entire contents of each packet should be mixed with 1 teaspoon (5 mL) of age-appropriate soft food or liquid. The mixture should be taken within 1 hour of being mixed. Make sure all medicine is taken.

The granule formulation is sweetened but unflavored.

No, granules should be kept in the foil packet until they are ready to be used.

The packets are child-resistant.

KALYDECO granules (50 mg or 75 mg) are for use in children 1 to less than 6 years of age, while KALYDECO tablets (150 mg) are for use in children age 6 years and older. You and your child's healthcare provider should discuss when to transition your child to KALYDECO tablets (150 mg).

Yes, you will need a new prescription when your child changes to a new strength or dosage form.

If you have questions about KALYDECO, please talk to your doctor. You can also find information about KALYDECO in the full Prescribing Information, including Patient Information.

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.

EXPAND

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.