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Results With ORKAMBI

See below to learn more about how ORKAMBI was studied and the results of the clinical studies. All of the results shown for each study below are an average of all people evaluated in each study.

ORKAMBI is for people with cystic fibrosis (CF) age 6 years and older with 2 copies of the F508del-CFTR mutation. It should not be used in people with other CFTR gene mutations and it is not known if ORKAMBI is safe and effective in younger children.

Remember, this website is for informational purposes only and is not intended to be a substitute for professional medical advice. You should talk to your healthcare provider about the clinical trial results and the possible side effects of ORKAMBI.

Studies in People Age 6 Through 11 Years

See the Study Results With ORKAMBI After 24 weeks of Treatment

Below, you'll find details on how ORKAMBI was studied in people age 6 through 11 years, as well as the results of the study. The primary purpose of the study below was to determine the safety of ORKAMBI in children age 6 through 11 years with 2 copies of the F508del-CFTR mutation.

  • How ORKAMBI® (lumacaftor/ivacaftor) Was Studied

    The primary purpose of the study below was to determine the safety of ORKAMBI in children age 6 through 11 years with 2 copies of the F508del mutation in their CFTR gene.

    How ORKAMBI was studied in children How ORKAMBI was studied in children How ORKAMBI was studied in children

    How ORKAMBI was studied in children

    The Study Evaluated:

    LUNG FUNCTION
    Lung function is measured with an FEV1 test, which measures how much air a person can exhale in a forced breath. Lung function was part of the safety assessment in this trial.

    SWEAT CHLORIDE LEVELS
    Children with CF have high levels of sweat chloride (mmol/L), which is a measure of the amount of salt in a child’s sweat. Children with sweat chloride levels of less than 30 mmol/L are unlikely to have CF.

  • Study Results

    Effect on Lung Function (FEV1) and Sweat Chloride

    After taking ORKAMBI for 24 weeks, lung function increased and sweat chloride levels decreased

    Lung function and sweat chloride results seen in children taking ORKAMBI

    Lung function and sweat chloride results seen in children taking ORKAMBI

    After ORKAMBI was stopped for 2 weeks, changes were seen in lung function and sweat chloride levels

    Lung function and sweat chloride results seen after children stopped taking ORKAMBI

    Lung function and sweat chloride results seen after children stopped taking ORKAMBI

    Because study participants did not take placebo, no comparisons could be made.

    It is not known if changes in lung function and sweat chloride levels were due to ORKAMBI. Changes in sweat chloride levels are not related to changes in lung function.

    “Both of my boys take an active role in their treatments and at the doctor’s office, asking about test results. They put in a lot of effort.”

    Heather, mother of 2 boys with CF

Studies in People Age 12 Years and Older

Study Results Suggest That ORKAMBI Can Have an Impact for Up to 2 Years

The possible benefits and risks of treatment with ORKAMBI in people with CF age 12 years and older with 2 copies of the F508del mutation were studied in 2 Short-Term Studies of 24 weeks. People who completed these studies were able to participate in a Long-Term Study for an additional 96 weeks.

Below, you'll find more details on how ORKAMBI was studied, as well as the study results. All of the results shown for each study are an average of all people evaluated in each study.

  • How ORKAMBI® (lumacaftor/ivacaftor) Was Studied

    Studied Up to 2 Years

    2 Short-Term Studies
    1 Long-Term Study

    The possible benefits and risks of treatment with ORKAMBI were studied for up to 2 years in people with CF age 12 and older.

    • In the Short-Term Studies, ORKAMBI and placebo (sugar pill) were compared. In the Long-Term Study, all participants took ORKAMBI
    • Every 12 hours, participants took 2 tablets of ORKAMBI (lumacaftor 200 mg/ivacaftor 125 mg) or placebo with fat-containing food. People continued to take their other prescribed CF treatments

    How ORKAMBI was studied in adults

    How ORKAMBI was studied in adults

    How ORKAMBI was studied in adults

    How ORKAMBI was studied in adults

    How ORKAMBI was studied in adults

    How ORKAMBI was studied in adults

    Considerations

    • The Long-Term Study did not have any participants who took placebo. All patients knew they were taking ORKAMBI, which may have influenced the results
    • The FDA did not consider the Long-Term Study when approving ORKAMBI, and it is not included in the full Prescribing Information. The Long-Term Study may not meet the FDA’s definition of an acceptable study because there was no placebo group included for comparison
    • At around Week 72, almost half the participants stopped taking part in the Long-Term Study because ORKAMBI was approved by the FDA at that time. Participants could start getting ORKAMBI from their own doctor
  • Study Results

    Lung Function (FEV1)

    Lung function results seen in adults taking ORKAMBI

    Lung function results seen in adults taking ORKAMBI

    Body Mass Index (BMI)

    BMI results seen in adults taking ORKAMBI

    BMI results seen in adults taking ORKAMBI

    CF Respiratory Symptoms

    CF respiratory results seen in adults taking ORKAMBI

    CF respiratory results seen in adults taking ORKAMBI

    About CF Respiratory Symptoms:

    • Includes cough, difficulty breathing, and amount of mucus coughed up
    • Based on the Cystic Fibrosis Questionnaire-Revised (CFQ-R) survey

    Pulmonary Exacerbations

    Pulmonary exacerbations results seen in adults taking ORKAMBI

    Other pulmonary exacerbation results from a separate analysis

    Pulmonary exacerbations results seen in adults taking ORKAMBI

    Other pulmonary exacerbation results from a separate analysis

    Learn more

    about another CF treatment option for eligible patients age 12 years and older.

Measuring the Results of Treatment

Each person may have a different experience with ORKAMBI. After beginning treatment, you or your child may not always feel a difference on a day-to-day or even week-to-week basis. That’s why it’s important to know what to look for and to discuss your results with your doctor.

After you or your child have been on ORKAMBI for a while, you may want to review certain measures with your doctor to compare them with how they were when treatment was first started.

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: cyclosporin, 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/or chest tightness
  • nausea
  • diarrhea
  • gas
  • rash
  • fatigue
  • flu or flu-like symptoms
  • increase in a certain blood enzyme called creatine phosphokinase
  • common cold, including sore throat, stuffy or runny nose
  • 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 may 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 6 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 6 years of age.