Frequently Asked Questions (FAQs)

Below are answers to some of the most frequently asked questions about ORKAMBI®. If you'd like more information, or have additional questions, please speak with your healthcare provider.

ORKAMBI is a prescription medicine available as tablets used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have 2 copies of the F508del  mutation (F508del/F508del ) in their cystic fibrosis transmembrane conductance regulator, or CFTR, gene.

ORKAMBI should not be used in patients other than those who have 2 copies of the F508del  mutation in their CFTR gene.

It is not known if ORKAMBI is safe and effective in children under 6 years of age.

No, ORKAMBI does not cure cystic fibrosis (CF). It is a chronic therapy that you take every day, as directed by your healthcare provider, in addition to your other CF therapies. Please talk to your healthcare provider if you have any questions about your treatment.

The F508del  mutation is the most common CFTR  mutation. It affects how many CFTR proteins there are at the cell surface and how those proteins at the cell surface work. Learn more.

ORKAMBI treats F508del-CFTR protein defects in people age 6 years and older with CF. It should only be used in people who have 2 copies of the F508del  mutation.

ORKAMBI is made up of lumacaftor and ivacaftor, which work together to target the underlying cause of cystic fibrosis (CF) in people age 6 years and older with 2 copies of the F508del  mutation—not just the symptoms.

  • Lumacaftor helps to get more F508del-CFTR proteins to the cell surface and helps them stay there longer
  • Ivacaftor helps the CFTR proteins at the cell surface work better

As a result, ORKAMBI lets more chloride ions pass into and out of the cell. This may help keep a balance of salt and water in organs such as the lungs. Learn more.

What is known about how ORKAMBI works was learned from studies conducted in a laboratory. Keep in mind that 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.

You should speak with your healthcare provider to find out which CFTR mutations you have.

ORKAMBI should not be used in people other than those who have 2 copies of the F508del mutation in their CFTR  gene.

It is not known if ORKAMBI is safe and effective in children under 6 years of age.

Do not take ORKAMBI if you take certain medicines or herbal supplements such as:

  • Antibiotics: rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • Seizure medicines: phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • Sedatives/anti-anxiety medicines: triazolam (Halcion®) or midazolam (Dormicum®, Hypnovel®, and Versed®)
  • Immunosuppressant medicines: everolimus (Zortress®), sirolimus (Rapamune®), or tacrolimus (Astagraf XL®, Envarsus XR®, Prograf®, and Protopic®)
  • St. John’s wort (Hypericum perforatum )

Talk to your doctor before taking ORKAMBI if you take any of the medicines or supplements listed above.

Before starting ORKAMBI, there are some important things you should discuss with your doctor, including your medical history and the medicines you take. Tell your doctor if you:

  • Have or have had liver problems
  • Have had an organ transplant
  • Have kidney problems
  • Are using birth control (hormonal contraceptives including oral, injectable, transdermal, or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI. Talk to your doctor about the best birth-control method you should use while taking ORKAMBI
  • Are pregnant or plan to become pregnant. It is not known if ORKAMBI will harm your unborn baby. You and your doctor should decide if you will take ORKAMBI while you are pregnant
  • Are breastfeeding or planning to breastfeed. It is not known if ORKAMBI passes into your breast milk. You and your doctor should decide if you will take ORKAMBI while you are breastfeeding

ORKAMBI may affect the way other medicines work, and other medicines may affect how ORKAMBI works.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements, because the dose of ORKAMBI may need to be adjusted when taken with certain medicines.

Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • Antifungal medicines such as ketoconazole (eg, Nizoral®), itraconazole (eg, Sporanox®), posaconazole (eg, Noxafil®), or voriconazole (eg, Vfend®)
  • Antibiotics such as telithromycin (eg, Ketek®), clarithromycin (eg, Biaxin®), or erythromycin (eg, Ery-Tab®)

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

If you have any questions about ORKAMBI, please talk to your healthcare provider. For more information, please read the Patient Information section of the full Prescribing Information.

Yes. The two active ingredients, lumacaftor and ivacaftor, are mixed together in each tablet when the tablets are being made. Each tablet contains the same amount of each active ingredient.

Every patient's experience is different. Review the clinical trial results for ORKAMBI in people age 12 years and older.

Review the clinical trial results for ORKAMBI in people age 6 through 11 years.

ORKAMBI can cause serious side effects.

High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI. Your doctor will do blood tests to check your liver:

  • Before you start ORKAMBI
  • Every 3 months during your first year of taking ORKAMBI
  • Every year while you are taking ORKAMBI

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
  • Confusion

Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If you have poor lung function, your doctor may monitor you more closely when you start ORKAMBI.

An increase in blood pressure has been seen in some patients treated with ORKAMBI. Your doctor should monitor your blood pressure during treatment with ORKAMBI.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI. If you are a child or adolescent, your doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.

The most common side effects of ORKAMBI include:

  • Shortness of breath and/or chest tightness
  • Upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose
  • Gastrointestinal symptoms, including nausea, diarrhea, or gas
  • Rash
  • Fatigue
  • Flu or flu-like symptoms
  • Increase in muscle enzyme levels
  • Irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding

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 ORKAMBI. 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 the FDA at 1-800-FDA-1088.

Vertex Guidance & Patient Support (Vertex GPS) provides one-on-one reimbursement support and product-related support for eligible patients who are prescribed ORKAMBI. This includes information about insurance coverage and potential financial assistance for patients who are 6 years of age or older, have 2 copies of the F508del  mutation in their CFTR gene, and have a prescription for ORKAMBI. Caregivers of patients younger than 18 years of age must be the patient’s parent or legal guardian. For more information, visit VertexGPS.com or speak with your healthcare provider.

Prescriptions for ORKAMBI can only be filled at authorized specialty pharmacies. You can view a full list of these pharmacies at www.vrtx.com/authorized-distributors. Based on your health plan, there may be restrictions on which pharmacies you can use.

ORKAMBI should not be used in people other than those who have 2 copies of the F508del  mutation in their CFTR  gene.

It is not known if ORKAMBI is safe and effective in children under 6 years of age.

Do not take ORKAMBI if you take certain medicines or herbal supplements such as:

  • Antibiotics: rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • Seizure medicines: phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • Sedatives/anti-anxiety medicines: triazolam (Halcion®) or midazolam (Dormicum®, Hypnovel®, and Versed®)
  • Immunosuppressant medicines: everolimus (Zortress®), sirolimus (Rapamune®), or tacrolimus (Astagraf XL®, Envarsus XR®, Prograf®, and Protopic®)
  • St. John’s wort (Hypericum perforatum )

Talk to your doctor before taking ORKAMBI if you take any of the medicines or supplements listed above.

Before starting ORKAMBI, there are some important things you should discuss with your doctor, including your medical history and the medicines you take. Tell your doctor if you:

  • Have or have had liver problems
  • Have had an organ transplant
  • Have kidney problems
  • Are using birth control (hormonal contraceptives including oral, injectable, transdermal, or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI. Talk to your doctor about the best birth-control method you should use while taking ORKAMBI
  • Are pregnant or plan to become pregnant. It is not known if ORKAMBI will harm your unborn baby. You and your doctor should decide if you will take ORKAMBI while you are pregnant
  • Are breastfeeding or planning to breastfeed. It is not known if ORKAMBI passes into your breast milk. You and your doctor should decide if you will take ORKAMBI while you are breastfeeding

ORKAMBI may affect the way other medicines work, and other medicines may affect how ORKAMBI works.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements, because the dose of ORKAMBI may need to be adjusted when taken with certain medicines.

Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • Antifungal medicines such as ketoconazole (eg, Nizoral®), itraconazole (eg, Sporanox®), posaconazole (eg, Noxafil®), or voriconazole (eg, Vfend®)
  • Antibiotics such as telithromycin (eg, Ketek®), clarithromycin (eg, Biaxin®), or erythromycin (eg, Ery-Tab®)

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

The recommended dose of ORKAMBI is 2 tablets every 12 hours with fat-containing foods, although your dose may be different. Your healthcare provider will tell you how much ORKAMBI to take and when to take it. Learn more.

Fat helps the body absorb ORKAMBI better, so ORKAMBI should always be taken with fat-containing foods. Learn more.

There is no specific amount of fat that should be taken with ORKAMBI, but you should always take ORKAMBI with fat-containing foods because fat helps the body absorb ORKAMBI better.
A typical cystic fibrosis (CF) diet will include fat-containing foods. If you have questions, your healthcare provider can help you choose healthy, fat-containing meals and snacks.

You can also download a list of fat-containing meals in the ORKAMBI food guide.

Some children have difficulty swallowing pills. The good news is that with a little help, your child can learn how to swallow pills more easily.

Ask the healthcare providers at your child's CF Center for any helpful suggestions they may have.

It is unknown if ORKAMBI causes dizziness. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how ORKAMBI affects you.

ORKAMBI can cause serious side effects.

High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI. Your doctor will do blood tests to check your liver:

  • Before you start ORKAMBI
  • Every 3 months during your first year of taking ORKAMBI
  • Every year while you are taking ORKAMBI

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
  • Confusion

Respiratory events such as shortness of breath or chest tightness were observed in patients when starting ORKAMBI. If you have poor lung function, your doctor may monitor you more closely when you start ORKAMBI.

An increase in blood pressure has been seen in some patients treated with ORKAMBI. Your doctor should monitor your blood pressure during treatment with ORKAMBI.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving ORKAMBI and ivacaftor, a component of ORKAMBI. If you are a child or adolescent, your doctor should perform eye examinations prior to and during treatment with ORKAMBI to look for cataracts.

The most common side effects of ORKAMBI include:

  • Shortness of breath and/or chest tightness
  • Upper respiratory tract infection (common cold), including sore throat, stuffy or runny nose
  • Gastrointestinal symptoms, including nausea, diarrhea, or gas
  • Rash
  • Fatigue
  • Flu or flu-like symptoms
  • Increase in muscle enzyme levels
  • Irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding

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 ORKAMBI. 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 the FDA at 1-800-FDA-1088.

High liver enzymes in the blood, which can be a sign of liver injury, have been reported in patients receiving ORKAMBI.

You need liver tests because your doctor will want to make sure ORKAMBI is not affecting your liver. Your doctor will do blood tests to check your liver:

  • Before you start ORKAMBI
  • Every 3 months during your first year of taking ORKAMBI
  • Every year while you are taking ORKAMBI

Your doctor may choose to do these tests more frequently.


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
  • Confusion

If you need more information, ask your doctor.

An increase in blood pressure has been seen in some patients treated with ORKAMBI. Your doctor should monitor your blood pressure during treatment with ORKAMBI.

Store ORKAMBI at room temperature between 68°F and 77°F (20°C to 25°C). Do not use ORKAMBI after the expiration date on the package. Keep ORKAMBI and all medicines out of the reach of children and pets.

Body mass index (BMI) was assessed in ORKAMBI clinical trials of people age 12 years and older. BMI is a measure of a person’s weight relative to height. Every person has a different treatment experience with ORKAMBI. Learn more about BMI results in clinical trials here. And for additional information, talk with your healthcare provider.

Yes, you can eat grapefruits and oranges while taking ORKAMBI.

You should talk with your healthcare provider about drinking alcohol while taking ORKAMBI. It is not known how alcohol interacts with ORKAMBI.