Study 4

Study 4 (G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, S549R)

In Study 4

  • 39 people with CF with a G1244E, G1349D, G178R, G551S, G970R*, S1251N, S1255P, S549N, or S549R mutation who were 6 years of age or older took KALYDECO or placebo (sugar pill) every 12 hours with fat-containing food. Each person had an 8-week treatment period with KALYDECO and an 8-week treatment period with placebo

*In this study, efficacy could not be established in people with the G970R mutation. KALYDECO is not indicated for people with CF who have the G970R mutation.

Improvement in lung function through 8 weeks of treatment

Overall, people who took KALYDECO had an improvement in lung function through 8 weeks of treatment

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.

Other study results

Overall, people who took KALYDECO experienced fewer CF respiratory symptoms through 8 weeks of treatment

Study 4: people 6 years of age or older

boy-coughing

In the overall population for the 9 mutations studied, people who took KALYDECO reported less coughing, less mucus, and less trouble breathing compared with people who took placebo.

Respiratory symptom results varied across the different mutations studied

  • Not all mutations showed the same level of benefit
  • Some patients experienced less improvement in CF respiratory symptoms compared with the average for all patients in the study. Others experienced more improvement
  • Talk to your doctor to learn more about your specific mutation

Assessed by the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score, a measure of respiratory symptoms relevant to people with CF such as cough, sputum production, and difficulty breathing.

Overall, people who took KALYDECO experienced an increase in body mass index (BMI) after 8 weeks of treatment

Study 4: people 6 years of age or older

Scale with Ruler

In the overall population for the 9 mutations studied, people who took KALYDECO had a 0.66 kg/m2 average increase in BMI compared with people who took placebo.

  • BMI is a measure of a person's weight relative to height
  • For example, if you are a 5'4", 110-lb adult, this would be a gain of about 4 lbs; if you are a 4'6", 65-lb child, this would be a gain of about 3 lbs

BMI results varied across the different mutations studied

  • Not all mutations showed the same level of benefit
  • Some patients experienced a smaller increase in BMI compared with the average for all patients in the study. Others experienced a larger increase
  • Talk to your doctor to learn more about your specific mutation

People who took KALYDECO had less salty sweat after 8 weeks of treatment

Study 4: people 6 years of age or older

Sweat Chloride

In the overall population for the 9 mutations studied, people who took KALYDECO had a 50 mmol/L average decrease in sweat chloride compared with people who took placebo.

Sweat chloride level results varied across the mutations studied

  • Not all mutations showed the same level of benefit
  • Some people experienced less improvement in sweat chloride levels compared with the average for all patients in the study. Others experienced more improvement
  • Talk to your healthcare provider to learn more about your specific mutation

A decrease in sweat chloride levels does not necessarily mean there will be an improvement in lung function (FEV1).

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.