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Everyone’s cystic fibrosis (CF) is different, but ultimately, it still works beneath the surface. Since CF is working beneath the surface, the treatment should be, too. With ORKAMBI, you can treat the underlying cause of CF in people age 2 years or older with 2 copies of the F508del-CFTR mutation.

The Underlying Cause of CF

CF is caused by cystic fibrosis transmembrane conductance regulator (CFTR) protein defects. A mutation in the genes of a person with CF may make defective CFTR proteins that:

  • Don't open correctly
  • Don't get to the cell surface, where they are normally located

Because of these defects, chloride ions cannot flow freely into or out of the cells as they should, leading to an imbalance of salt and water. In the lungs, this can lead to thick, sticky mucus.

ORKAMBI® (lumacaftor/ivacaftor) Targets the Underlying Cause of CF in People Age 2 Years and Older With 2 Copies of the F508del Mutation

Watch this video to see how ORKAMBI works, or read below to learn more about ORKAMBI.

animation showing how ORKAMBI® (lumacaftor/ivacaftor) works

ORKAMBI is made up of lumacaftor and ivacaftor, which work on certain defects of the CFTR protein at the cellular level:

the cell of someone with CF without ORKAMBI® (lumacaftor/ivacaftor) and with ORKAMBI, showing how Ivacaftor and Lumacaftor work together

the cell of someone with CF without ORKAMBI and with ORKAMBI, showing how Ivacaftor and Lumacaftor work together

What is known about how ORKAMBI® (lumacaftor/ivacaftor) 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.

Important Safety Information for ORKAMBI® (lumacaftor/ivacaftor)

Do not take ORKAMBI® (lumacaftor/ivacaftor) 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 and anti-anxiety medicines: triazolam (Halcion®) or midazolam (Dormicum®, Hypnovel®, and Versed®)
  • immunosuppressant medicines: cyclosporine, everolimus (Zortress®), sirolimus (Rapamune®), or tacrolimus (Astagraf XL®, Envarsus XR®, Prograf®, 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 you take ORKAMBI, tell your doctor about all of your medical conditions, including 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.

Especially tell your doctor if you take:

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

Tell your doctor if you stop ORKAMBI for more than 1 week. Your doctor may need to change your dose of ORKAMBI or other medicines you take.

What are the possible side effects of ORKAMBI?

ORKAMBI can cause serious side effects, including:

  • Worsening of liver function in people with severe liver disease. The worsening of liver function can be serious or cause death. Talk to your doctor if you have been told you have liver disease as your doctor may need to adjust the dose of ORKAMBI.
  • High liver enzymes in the blood, which can be a sign of liver injury in people 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
  • Breathing problems such as shortness of breath or chest tightness in patients when starting ORKAMBI, especially in patients who have poor lung function. If you have poor lung function, your doctor may monitor you more closely when you start ORKAMBI.
  • An increase in blood pressure in some people receiving ORKAMBI. Your doctor should monitor your blood pressure during treatment with ORKAMBI.
  • Abnormality of the eye lens (cataract) in some children and adolescents receiving ORKAMBI. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with ORKAMBI to look for cataracts.
The most common side effects of ORKAMBI include:
  • breathing problems such as shortness of breath and chest tightness
  • nausea
  • diarrhea
  • fatigue
  • increase in a certain blood enzyme called creatine phosphokinase
  • rash
  • gas
  • common cold, including sore throat, stuffy or runny nose
  • flu or flu-like symptoms
  • irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding

Side effects seen in children are similar to those seen in adults and adolescents. Additional common side effects seen in children include:

  • cough with sputum
  • stuffy nose
  • headache
  • stomach pain
  • increase in sputum

These are not all the possible side effects of ORKAMBI. Call your doctor for medical advice about side effects. You are encouraged to report side effects to FDA at 1-800-FDA-1088.

For further information, please see full Prescribing Information, including Patient Information.

What is ORKAMBI?

ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 2 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene.

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

It is not known if ORKAMBI is safe and effective in children under 2 years of age.

Click here for ORKAMBI® (lumacaftor/ivacaftor) Important Safety Information and full Prescribing Information, including Patient Information.