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Ivacaftor and lumacaftor

Generic name: ivacaftor and lumacaftor [ EYE-va-KAF-tor-and-LOO-ma-KAF-tor ]
Brand name: Orkambi
Dosage forms: oral granule for reconstitution (125 mg-100 mg; 188 mg-150 mg), oral tablet (125 mg-100 mg; 125 mg-200 mg)
Drug class: CFTR combinations

Medically reviewed by Drugs.com on Feb 2, 2023. Written by Cerner Multum.

What is ivacaftor and lumacaftor?

Ivacaftor and lumacaftor is a combination medicine used to treat cystic fibrosis in adults and children who are at least 2 years old.

Ivacaftor and lumacaftor is for use only in patients with a specific gene mutation related to cystic fibrosis. Before you take ivacaftor and lumacaftor, you may need a medical test to make sure you have this gene mutation.

Ivacaftor and lumacaftor may also be used for purposes not listed in this medication guide.

Warnings

Tell your doctor about all your current medicines and any you start or stop using. Many drugs can interact, and some drugs should not be used together.

Before taking this medicine

You should not use ivacaftor and lumacaftor if you are allergic to ivacaftor or lumacaftor.

Some medicines can cause unwanted or dangerous effects when used with ivacaftor and lumacaftor. Your doctor may change your treatment plan if you also use:

Tell your doctor if you have ever had:

Tell your doctor if you are pregnant or breastfeeding.

Ivacaftor and lumacaftor can make hormonal birth control less effective, including birth control pills, injections, implants, skin patches, and vaginal rings. To prevent pregnancy while using ivacaftor and lumacaftor, use a barrier form of birth control: condom, diaphragm, cervical cap, or contraceptive sponge.

Ivacaftor and lumacaftor is not approved for use by anyone younger than 2 years old.

How should I take ivacaftor and lumacaftor?

Follow all directions on your prescription label and read all medication guides or instruction sheets. Your doctor may occasionally change your dose. Use the medicine exactly as directed.

Take this medicine with food that contains fat, such as butter, peanut butter, eggs, avocados, nuts, whole milk, cheese, yogurt, or cheese pizza. Follow your doctor's dosing instructions very carefully.

Read and carefully follow any Instructions for Use provided with your medicine. Ask your doctor or pharmacist if you do not understand these instructions.

To use the oral granules: Mix the granules with 1 teaspoon of milk, juice, or soft food such as applesauce, yogurt, or pudding. The food or liquid should be at or below room temperature. Mix only 1 dose at a time, and use the mixture within 1 hour after mixing. Feed the child a high-fat food just before or just after giving the granule mixture.

Take your doses at regular intervals, 12 hours apart. This will keep a steady amount of the drug in your body at all times.

Ivacaftor and lumacaftor doses are based on age and weight in children. Children from 2 to 5 years old should take oral granules. Children 6 and older should take tablets.

You will need frequent blood tests to check your liver function. Your blood pressure may also need to be checked often.

A child using this medicine may need frequent eye exams to check for cataracts.

Store at room temperature away from moisture and heat. Keep each tablet in its daily blister strip until you are ready to take your dose.

What happens if I miss a dose?

Take the medicine as soon as you can, but skip the missed dose if you are more than 6 hours late for the dose. Do not take two doses at one time.

Be sure to take a missed dose with a food that contains fat.

If you miss doses or stop taking ivacaftor and lumacaftor for more than 1 week, call your doctor before you start taking the medicine again. You may need a different dose.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking ivacaftor and lumacaftor?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Ivacaftor and lumacaftor side effects

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Ivacaftor and lumacaftor may cause serious side effects. Call your doctor at once if you have:

  • shortness of breath, tightness in your chest; or

  • liver problems--nausea, upper stomach pain, itching, tired feeling, loss of appetite, dark urine, clay-colored stools, jaundice (yellowing of the skin or eyes).

Common side effects of ivacaftor and lumacaftor may include:

  • breathing problems;

  • feeling tired;

  • nausea, gas, diarrhea;

  • rash;

  • flu symptoms;

  • changes in your menstrual periods; or

  • cold symptoms such as runny or stuffy nose, sinus pain, sore throat.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Ivacaftor and lumacaftor dosing information

Usual Adult Dose for Cystic Fibrosis:

Lumacaftor 400 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food

Comments:
-A fat containing meal or snack should be consumed just before or just after dosing. Examples include eggs, avocados, nuts, butter, peanut butter, cheese pizza, or whole-milk dairy products.
-If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene; safety and efficacy in patients with cystic fibrosis other than homozygous F508del mutation has not been established.


Use: For the treatment of cystic fibrosis (CF) in patients who are homozygous for the F508del mutation in the CFTR gene.

Usual Pediatric Dose for Cystic Fibrosis:

Age: 2 through 5 years; weight less than 14 kg: Lumacaftor 100 mg/Ivacaftor 125 mg orally every 12 hours with fat-containing food
Age: 2 through 5 years: weight 14 kg or greater: Lumacaftor 150 mg/Ivacaftor 188 mg orally every 12 hours with fat-containing food

Age: 6 through 11 years: Lumacaftor 200 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food

Age: 12 years or older: Lumacaftor 400 mg/Ivacaftor 250 mg orally every 12 hours with fat-containing food

Comments:
-Pediatric patients 2 through 5 years of age should receive oral granules; pediatric patients over 6 years of age, should be dosed with tablets.
-A fat containing meal or snack should be consumed just before or just after dosing. Examples include eggs, avocados, nuts, butter, peanut butter, cheese pizza, or whole-milk dairy products.
-If the patient's genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of the F508del mutation on both alleles of the CFTR gene; safety and efficacy in patients with cystic fibrosis other than homozygous F508del mutation has not been established.

Use: For the treatment of cystic fibrosis (CF) in patients 2 years or older who are homozygous for the F508del mutation in the CFTR gene.

What other drugs will affect ivacaftor and lumacaftor?

During your first week of using ivacaftor and lumacaftor, your doctor may adjust your dose if you also use other medicines, especially:

This list is not complete. Other drugs may affect ivacaftor and lumacaftor, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.

Popular FAQ

Orkambi should be taken every 12 hours with fat-containing food such as eggs, avocados, nuts, butter, peanut butter, cheese, and whole-milk dairy products. Granules are available for younger children aged one through five years and these should be mixed with one teaspoon of soft food or liquid. Other therapies prescribed for CF - including bronchodilators, inhaled antibiotics, dornase alfa, and hypertonic saline - should continue to be used if you are prescribed Orkambi. Continue reading

More FAQ

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Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.