Lumacaftor & Ivacaftor

Lumacaftor/ivacaftor is a prescription medication used to treat cystic fibrosis in adults and children who have a specific gene mutation.

It is a single product containing 2 medications: lumacaftor and ivacaftor. Both of these components work by targeting the underlying genetic mutation that causes cystic fibrosis and improving the function of a naturally occurring protein.

This medication comes in tablet form and is taken 2 times a day, with food.

Common side effects of lumacaftor/ivacaftor include shortness of breath, upper respiratory tract infections, nausea, diarrhea, and rash. Women who took lumacaftor/ivacaftor also had increased menstrual abnormalities such as increased bleeding.

Lumacaftor/ivacaftor may also cause dizziness. Do not drive or operate heavy machinery until you know how lumacaftor/ivacaftor affects you.

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.

Lumacaftor & Ivacaftor may be found in some form under the following brand names:

• Orkambi

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.

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements. Especially tell your doctor if you take:

• antibiotics such as clarithromycin (Biaxin), telithromycin (Ketek), erythromycin (Ilotycin, Ery-Ped, Ery-tab, Ery, Pce, Staticin, Erythra-Derm, Akne-mycin, T-Stat, Eryc), rifampin (Rifadin), and
• rifabutin (Mycobutin)
• antidepressants, such as citalopram (Celexa), escitalopram (Lexapro), and sertraline (Zoloft)
• antifungal medications such as itraconazole (Onmel, Sporanox), ketoconazole (Xolegel, Extina, Nizoral), posaconazole (Noxafil), and voriconazole (Vfend)
• benzodiazepines such as midazolam (Versed) and triazolam (Halcion)
• hormonal contraceptives
• ibuprofen (Advil, Midol, NeoProfen, Caldolor, Motrin, Ibu)
• immunosuppressants such as cyclosporine (Neoral, Restasis, Sandimmune, Gengraf), everolimus (Zortress, Afinitor), sirolimus (Rapamune), and tacrolimus (Protopic, Prograf, Hecoria)
• medications for seizures such as carbamazepine (Tegretol, Carbatrol, Equetro, Epitol), phenobarbital, and phenytoin (Phenytek, Dilantin, Dilantin-125)
• repaglinide (Prandin) or other medications for diabetes
• St. John’s wort
• warfarin (Coumadin, Jantoven)

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

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.

Medications can interact with certain foods. In some cases, this may be harmful and your doctor may advise you to avoid certain foods. In the case or lumacaftor/ivacaftor, there are no specific foods that you must exclude from your diet when receiving this medication.

Before taking lumacaftor/ivacaftor, tell your doctor about all of your medical conditions. Especially tell your doctor if you:

• are allergic to lumacaftor/ivacaftor or to any of its ingredients
• have liver problems
• have kidney problems
• are using any form of hormonal birth control
• are pregnant or breastfeeding

Tell your doctor about all the medicines you take including prescription and non-prescription medicines, vitamins, and herbal supplements.

Tell your doctor if you are pregnant or plan to become pregnant.

The FDA categorizes medications based on safety for use during pregnancy. Five categories - A, B, C, D, and X - are used to classify the possible risks to an unborn baby when a medication is taken during pregnancy.

Lumacaftor/ivacaftor falls into category B. There are no well-done studies that have been done in humans with Lumacaftor/ivacaftor. In animal studies, pregnant animals were given this medication, and the babies did not show any medical issues related to this medication.

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 lumacaftor/ivacaftor exactly as prescribed.

This medication comes in tablet form and is taken 2 times a day (12 hours apart), with food. Always take lumacaftor/ivacaftor tablets with fat-containing foods such as eggs, avocados, nuts, butter, peanut butter, cheese pizza, or whole-milk dairy products.

If you miss a dose within 6 hours of when you usually take it, take your dose 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 when you usually take it. Do not take 2 doses at the same time to make up for your missed dose.

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.

If you take too much lumacaftor/ivacaftor, call your healthcare provider or local Poison Control Center, or seek emergency medical attention right away.

If lumacaftor/ivacaftor is administered by a healthcare provider in a medical setting, it is unlikely that an overdose will occur. However, if overdose is suspected, seek emergency medical attention.

• Store lumacaftor/ivacaftor at room temperature.
• Keep this and all medicines out of the reach of children.