Study 7

Study 7 (2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, E831X, A455E, D1152H, D579G, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, and S977F)

KALYDECO was compared with placebo (sugar pill) in a clinical trial of people with certain CFTR mutations predicted to respond to KALYDECO.

  • The study included people with CF age 12 years and older with 1 copy of the F508del mutation and a second mutation that is predicted to respond to KALYDECO
    • Number of people who contributed to the data for placebo: 161
    • Number of people who contributed to the data for KALYDECO: 156
  • People took 1 tablet of KALYDECO (150 mg) or placebo every 12 hours, with fat-containing food, along with their other prescribed CF therapies for 8 weeks
  • The main endpoint studied was lung function (FEV1)*; other endpoints included were CF respiratory symptoms (CFQ-R Respiratory Domain Score) and safety

*FEV1, forced expiratory volume in 1 second.

CFQ-R, Cystic Fibrosis Questionnaire-Revised.

Considerations

  • Some but not all of the 28 approved mutations that are predicted to respond to KALYDECO were enrolled in Study 7. Please see the full Prescribing Information or talk to your healthcare provider to learn which mutations were studied
  • This is not intended to represent the full study design or results
  • Results may differ among patients and mutations

KALYDECO improved lung function* compared with placebo overall in Study 7

Improvement in lung function was measured as the difference in FEV1* from the start of the study to the average of Week 4 and Week 8.

*Lung function was measured as FEV1, or forced expiratory volume exhaled in 1 second.

People kept taking their other cystic fibrosis (CF) therapies, except inhaled hypertonic saline.

KALYDECO improved CF respiratory symptoms compared with placebo overall in Study 7

People who took KALYDECO were more likely to report less coughing, less mucus, and less trouble breathing after averaging the measurements taken of Week 4 and Week 8.

CF respiratory symptoms (such as coughing, sputum production, and difficulty breathing) were scored based on the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score.

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO.

Talk to your doctor to learn if you have an indicated CF gene mutation.

It is not known if KALYDECO is safe and effective in children under 12 months of age.

Important Safety Information

Who should not take KALYDECO?

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

  • the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • seizure medicines such as: phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

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

Important Safety Information

Who should not take KALYDECO?

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

  • the antibiotics rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
  • seizure medicines such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, and Equetro®), or phenytoin (Dilantin®, Phenytek®)
  • St. John’s wort (Hypericum perforatum)

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

What should I tell my doctor before taking KALYDECO?

Before you take KALYDECO, tell your doctor if you:

  • have liver or kidney problems
  • drink grapefruit juice, or eat grapefruit or Seville oranges
  • are pregnant or plan to become pregnant. It is not known if KALYDECO will harm your unborn baby. You and your doctor should decide if you will take KALYDECO while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if KALYDECO passes into your breast milk. You and your doctor should decide if you will take KALYDECO while you are breastfeeding

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

Tell your doctor about all the medicines you take, including prescription and non-prescription medicines, vitamins, and herbal supplements, as the dose of KALYDECO may need to be adjusted when taken with certain medications.

Especially tell your doctor if you take:

  • antifungal medications such as ketoconazole (e.g., Nizoral®), itraconazole (e.g., Sporanox®), posaconazole (e.g., Noxafil®), voriconazole (e.g., Vfend®), or fluconazole (e.g., Diflucan®)
  • antibiotics such as telithromycin (e.g., Ketek®), clarithromycin (e.g., Biaxin®), or erythromycin (e.g., Ery-Tab®)

What should I avoid while taking KALYDECO?

  • KALYDECO can cause dizziness in some people who take it. Do not drive a car, use machinery, or do anything that needs you to be alert until you know how KALYDECO affects you
  • You should avoid food containing grapefruit or Seville oranges while you are taking KALYDECO

What are the possible side effects of KALYDECO?

KALYDECO can cause serious side effects.

High liver enzymes in the blood have been reported in patients receiving KALYDECO. Your doctor will do blood tests to check your liver:

  • before you start KALYDECO
  • every 3 months during your first year of taking KALYDECO
  • every year while you are taking KALYDECO

For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often.

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

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts.

The most common side effects include:

  • headache
  • upper respiratory tract infection (common cold), including sore throat, nasal or sinus congestion, runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • nausea
  • dizziness

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 KALYDECO. 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 FDA at 1-800-FDA-1088.

Click here for KALYDECO full Prescribing Information, including Patient Information.