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Kalydeco

ivacaftor

Used for Cystic Fibrosis

Kalydeco (ivacaftor) is a medication used to treat cystic fibrosis (CF) in people aged 4 months and older. It's only for people who have a special mutation (change in DNA) in the CFTR (cystic fibrosis transmembrane conductance regulator) gene that responds to Kalydeco (ivacaftor). This medication is typically only available at certain pharmacies.

Reviewed by:

Allison Gourley, PharmD

Michelle Nguyen, PharmD

Last reviewed on December 1, 2021

Basics Side effects Pros & cons Pharmacist tips FAQs Warnings Dosage Interactions Alternatives Latest news Images Prices Medicare

What is Kalydeco (ivacaftor)?

What is Kalydeco (ivacaftor) used for?

Cystic fibrosis

How Kalydeco (ivacaftor) works

Kalydeco (ivacaftor) is a CFTR potentiator. It helps the CFTR protein work better by keeping chloride "gates" open so that salt and water can move more freely in the lungs. This helps prevent mucus buildup in the lungs, which helps improve cystic fibrosis and its related symptoms.

Are you looking for information on Orkambi (lumacaftor / ivacaftor), Symdeko (tezacaftor / ivacaftor), or Trikafta (elexacaftor / tezacaftor / ivacaftor) instead?

Drug Facts

Common BrandsKalydeco

Drug ClassCFTR potentiator

Controlled Substance ClassificationNot a controlled medication

Generic StatusNo lower-cost generic available

AvailabilityPrescription only

What are the side effects of Kalydeco (ivacaftor)?

The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.

Common Side Effects

Headache (24%)
Sore throat (22%)
Upper respiratory tract infection (cough, cold; 22%)
Stuffy nose (20%)
Stomach pain (16%)
Common cold (15%)
Diarrhea (13%)
Rash (13%)
Nausea (12%)
Dizziness (9%)

Other Side Effects

High blood sugar
Joint pain
Chest pain
Muscle pain
Wheezing
Acne

Serious Side Effects

Contact your healthcare provider immediately if you experience any of the following.

Liver damage: upper right stomach pain or discomfort, dark-colored urine, pale-colored stools, yellowing of the skin or eyes

Source: DailyMed

The following side effects have also been reported

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Body aches or pain

chest pain

chills

cough

difficulty with breathing or trouble breathing

ear congestion

fever

headache

loss of voice

runny or stuffy nose

sneezing

sore throat

unusual tiredness or weakness

Less common

Dark urine

fever with or without chills

light-colored stools

loss of appetite

nausea

stomach pain

vomiting

yellow eyes or skin

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Blemishes on the skin

diarrhea

difficulty with moving

dizziness

muscle aches, pain, or stiffness

pain in the joints

pimples

rash

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

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

Pros and cons of Kalydeco (ivacaftor)

Pros

Available in an oral granule form, which can be mixed in soft foods or liquids for children who cannot swallow tablets

Works well to treat cystic fibrosis in people with a CFTR mutation

Dose can be adjusted for people with mild liver disease

Cons

Must take with certain foods for it to work well (foods that are high in fat)

Only available as a brand medication, so it can be very expensive

Interacts with a lot of medications, such as rifampin, St. John's wort, and phenytoin

Pharmacist tips for Kalydeco (ivacaftor)

Keep in mind that taking Kalydeco (ivacaftor) every 12 hours is not the same as taking it twice a day. Set alarms or reminders for yourself to help remember to take it on time every 12 hours.

Always take Kalydeco (ivacaftor) with foods that are high in fat to help your body absorb the medication better.

Avoid foods or drinks with grapefruit, because these might raise the amount of Kalydeco (ivacaftor) in your body.

If you're taking the Kalydeco (ivacaftor) oral granules, pour and mix all of the granules into 1 teaspoon (about 5 mL) of soft food or liquid that's at or below room temperature. Some examples of soft foods include puréed fruits or vegetables, applesauce, yogurt, infant formula, or juice. Make sure your child takes the mixture within an hour of mixing it, right before or right after eating a high-fat meal.

Talk to your provider before starting, changing, or stopping any medications while taking Kalydeco (ivacaftor), including prescription and non-prescription medications, vitamins, and supplements. Kalydeco (ivacaftor) can interact with a lot of medications, so it's important that your provider knows about all the medications you take or plan to take to make sure they're safe for you.

Frequently asked questions about Kalydeco (ivacaftor)

What is a CFTR gene mutation and what does Kalydeco (ivacaftor) have to do with it?

Kalydeco (ivacaftor) only works in people who have a special mutation (change in DNA) in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. The CFTR gene gives the body instructions to make the CFTR protein. Normally, the CFTR protein helps the lungs keep a healthy balance of salt and water. When there's a mutation in this gene, the protein doesn't work properly. Mucus gets trapped in the lungs and cause airway infections or other CF complications. Kalydeco (ivacaftor) helps the CFTR protein work better so that salt and water can move more freely in the lungs and not get trapped or cause mucus buildup. Check out this list of mutations that Kalydeco (ivacaftor) is eligible for.

What are some examples of high-fat foods that I should take with Kalydeco (ivacaftor)?

Butter on toast or bagels, peanut butter, eggs, avocados, and whole-milk dairy products (e.g., whole-milk, yogurt, cheese) are good examples of foods that are high in fat. For infants, breast milk and infant formula are high-fat meal options as well. These high-fat foods will help your body absorb Kalydeco (ivacaftor). You won't get as much medication in your system if you take it on an empty stomach or with a low-fat meal.

What should I do if I miss my Kalydeco (ivacaftor) dose?

If less than 6 hours have passed since your missed dose, go ahead and take your Kalydeco (ivacaftor) dose with food (something fatty) as soon as possible. If more than 6 hours have passed, don't take your missed dose. In that case, just take your next dose at the regular time with a high-fat meal. Don't take 2 doses at the same time.

What if I don't know what gene mutation I have or my child has before starting Kalydeco (ivacaftor)?

If you don’t know your or your child's CF gene mutations, make sure to work with your healthcare provider to find out. Knowing your mutations can help your provider decide if this medication will even work for you or your child. If you're not eligible for Kalydeco (ivacaftor), you might need to use a different treatment for your CF.

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What are the risks and warnings for Kalydeco (ivacaftor)?

Kalydeco (ivacaftor) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.

High liver test results

Risk factors: History of liver problems

Kalydeco (ivacaftor) can sometimes cause abnormally high liver test results. Your provider will check your liver before you start Kalydeco (ivacaftor), then every 3 months for the next year, and then every year after that. If you have a history of liver problems, your provider might check your liver labs more often. While taking this medication, if you experience stomach pain, dark-colored urine, pale-colored stools, or yellowing of the skin or eyes, get medical help right away, as these can be signs of a liver problem.

Interactions with other medications

Taking Kalydeco (ivacaftor) along with certain medications can affect the amount of Kalydeco (ivacaftor) in your body, how well it works, and the risk of side effects.

Some medications, like the antibiotic, rifampin, and a common supplement, St. John's wort, can lower the amount of Kalydeco (ivacaftor) in your body. This can make Kalydeco (ivacaftor) not work as well.

Other medications, like the antifungal, fluconazole (Diflucan), can raise the levels of Kalydeco (ivacaftor) in your body. You'll also want to avoid grapefruit and grapefruit juice while taking this medication, because grapefruit can cause you to have too much medication in your body as well. This can lead to a higher risk of side effects.

In addition, Kalydeco (ivacaftor) can affect how other medications work, so make sure to tell your provider and pharmacist about all the medications and supplements you're taking before starting Kalydeco (ivacaftor).

Cataracts

Risk factors: Taking corticosteroid medications | Exposure to radiation

Kalydeco (ivacaftor) can cause cataracts (clouding of the lens of the eye) in children. Your child's provider will likely give your child an eye exam before starting the medication and throughout treatment.

Kalydeco (ivacaftor) dosage forms

Typical dosing for Kalydeco (ivacaftor)

Tablets for adults and children age 6 years and older: The typical dose is 150 mg by mouth every 12 hours with a high-fat meal.
Oral granules for children younger than 6 years of age: The dose will depend on the child’s age and weight. Dosage starts from 25 mg to 75 mg by mouth every 12 hours with a high-fat meal.

The dose might differ if you or your child have liver problems or if you're taking certain medications that can interact with Kalydeco (ivacaftor).

Interactions between Kalydeco (ivacaftor) and other drugs

Kalydeco (ivacaftor) may interact with certain medications or supplements. Always let your doctor and pharmacist know about any other medications or supplements (including prescribed and over-the-counter medications, vitamins, and dietary or herbal supplements) that you are currently taking. The list below does not include all possible drug interactions with Kalydeco (ivacaftor). Please note that only the generic name of each medication is listed below.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.