Lumacaftor / ivacaftor

>10%

Dyspnea (13%)

Nasopharyngitis (13%)

Nausea (13%)

Diarrhea (12%)

1-10%

Menstrual abnormalities (10%)

Upper respiratory tract infection (10%)

Fatigue (9%)

Abnormal respiration (9%)

Increased blood CPK (7%)

Rash (7%)

Flatulence (7%)

Rhinorrhea (6%)

<1%

Increased ALT or AST

Increased bilirubin

Hepatic encephalopathy

Lumacaftor/ivacaftor is a prescription medication used to treat cystic fibrosis in patients age 6 and older. 

Lumacaftor/ivacaftor works by targeting the underlying genetic mutation that causes cystic fibrosis and improving the function of a naturally occurring protein.

This medication may be prescribed for other uses. Ask your doctor or pharmacist for more information.

Serious side effects have been reported with lumacaftor/ivacaftor. See the “Lumacaftor & Ivacaftor Precautions” section.

Common side effects of lumacaftor/ivacaftor include the following:

• shortness of breath
• common cold
• nausea
• diarrhea
• upper respiratory tract infection
• fatigue
• abnormal respiration
• rash
• flatulence
• running nose
• flu or flu-like symptoms
• changes in menstruation and bleeding abnormalities in women

This is not a complete list of lumacaftor/ivacaftor side effects. Ask your doctor or pharmacist for more information.

Tell your doctor if you have any side effect that bothers you or that does not go away.

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

Serious side effects have been reported with lumacaftor/ivacaftor including the following:

• Liver injury. Tell your healthcare provider right away if you have some or all of the following symptoms of liver problems.
  • pain or discomfort in the upper right stomach (abdominal) area
  • loss of appetite
  • dark, amber-colored urine
  • yellowing of your skin or the white part of your eyes
  • nausea or vomiting
  • confusion
• Respiratory problems. Lumacaftor/ivacaftor should be used cautiously in patients with poor lung function.
• Eye abnormalities. Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ivacaftor, a component of lumacaftor/ivacaftor. You should have regular eye examinations prior to and during treatment with lumacaftor/ivacaftor to look for cataracts.
• Blood pressure. Lumacaftor/ivacaftor can raise one's blood pressure. Your healthcare will monitor your blood pressure. 

It is not known if lumacaftor/ivacaftor causes drowsiness. Do not drive or operate heavy machinery until you know how lumacaftor/ivacaftor affects you.

Do not take lumacaftor/ivacaftor if you are allergic to lumacaftor/ivacaftor or to any of its ingredients.

Tell your doctor if you are breastfeeding or plan to breastfeed.

Lumacaftor/ivacaftor has been detected in human breast milk. Because of the possibility for adverse reactions in nursing infants from lumacaftor/ivacaftor, a choice should be made whether to stop nursing or to stop use of this medication. The importance of the drug to the mother should be considered.

Take this medication exactly as prescribed by your doctor. Follow the directions on your prescription label carefully.

The dose your doctor recommends may be based on the following:

• the condition being treated
• other medical conditions you have
• other medications you are taking
• your liver function
• age

The recommended dose of lumacaftor/ivacaftor in patients age 12 years and older: 2 tablets (each containing 200 mg lumacaftor and 125 mg ivacaftor) every 12 hours.

The recommended dose of lumacaftor/ivacaftor in patients age 6 through 11 years: 2 tablets (each containing 100 mg lumacaftor and 125 mg ivacaftor) every 12 hours.

The dose will be reduced if your liver is not working as it should.

It is very important that your doctor check your progress at regular visits to make sure that lumacaftor/ivacaftor is working properly. Blood tests will be needed to check for unwanted effects. You may also need to monitor your blood pressure at home. If you notice any changes to your normal blood pressure, call your doctor right away.

Birth control pills and other forms of hormone-based contraceptives (such as patches, rings, implants, and injections) may not work while you are using lumacaftor/ivacaftor. To keep from getting pregnant, use another form of birth control (including condoms, a diaphragm, or contraceptive foam or jelly) while using Orkambi®.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal (eg, St. John's wort) or vitamin supplements.

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:

• Difficult or labored breathing
• tightness in the chest

• Blurred vision
• dark urine
• dizziness
• headache
• loss of appetite
• nausea and vomiting
• nervousness
• pain or discomfort in upper right stomach area
• pounding in the ears
• slow or fast heartbeat
• unusual tiredness
• 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:

• Absent, missed, or irregular menstrual periods
• bloated
• body aches or pain
• chills
• cough
• cramps
• diarrhea
• ear congestion
• excess air or gas in the stomach or intestines
• fever
• full feeling
• heavy bleeding
• longer or heavier menstrual periods
• loss of voice
• muscle aches
• passing gas
• rash
• sneezing
• sore throat
• stopping of menstrual bleeding
• stuffy or runny nose
• unusual weakness

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.