KALYDECO® Results

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Age 12 years and older

Study 1

Study 1 was a 48-week study in which 161 people age 12 and older with a G551D mutation took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours, along with their other cystic fibrosis (CF) treatments. Results were measured at different time points, including 24 and 48 weeks. All people took all their other CF treatments except for hypertonic saline through the entire length of the study (48 weeks). The study evaluated lung function (FEV1*). Other results studied were CF respiratory symptoms, pulmonary exacerbations, weight, and sweat chloride. The study also evaluated the safety of KALYDECO.

*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.

Mutation eligible to enroll and included in this study was:

G551D.

Study results

Lung function (FEV1)

Lung function increased

10.6 percentage points greater average improvement, compared to placebo through 24 weeks.

10.5 percentage points greater average improvement, compared to placebo through 48 weeks.

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. 

Other study results

CF respiratory symptoms

8.6 points on average, compared to placebo, from the start of the study through Week 48.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

Pulmonary exacerbations

Pulmonary exacerbations decreased

Nearly 7 out of 10 people who took KALYDECO did not have a pulmonary exacerbation, compared to around 4 out of 10 people who took placebo at Week 48.

Pulmonary exacerbations are defined as changes in certain symptoms requiring changes in the use of oral, IV, or inhaled antibiotics.

Weight

Weight increased

About 2.7 kg (~6 lb) on average, compared to placebo at Week 48.

Sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -48 mmol/L compared to placebo through Week 48.

Sweat chloride is a measure of the amount of salt in a person's sweat. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1).

Study 7

Study 7 was an 8-week study in which people age 12 and older with 1 copy of the F508del mutation and a second mutation predicted to respond to KALYDECO took either KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. All people took all their other CF treatments. The study evaluated lung function (FEV1). CF respiratory symptoms and safety were also studied.

In this study, 156 people took KALYDECO (150-mg tablets) and 161 people took placebo.

Mutations enrolled in this study were:

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

Important considerations

  • Results varied by mutation. Not all mutations showed the same level of benefit when taking KALYDECO
  • Some people experienced less improvement compared to the average for all the people in the study. Others experienced more improvement
  • This is not intended to represent the full study design or results
  • Talk to your healthcare provider for more information

Study results

Lung function (FEV1)

Lung function increased

In the overall population studied, lung function increased by 4.7 percentage points on average compared to placebo from the start of the study to the average of Week 4 and Week 8.

Results varied by mutation.

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. 

Other study results

CF respiratory symptoms

CF respiratory symptoms improved

In the overall population studied, CF respiratory symptoms improved by 9.7 points on average compared to placebo from the start of the study to the average of Week 4 and Week 8.

Results varied by mutation.

Respiratory symptoms are measured by the CFQ-R Respiratory Domain score, which is a tool used to measure respiratory symptoms, including coughing, mucus, and trouble breathing.

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IMPORTANT SAFETY INFORMATION

Before taking KALYDECO, tell your doctor about all of your medical conditions, including if you:

  • have liver or kidney problems
  • are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
  • 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

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) 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 1 month of age.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • the antibiotics rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
  • seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
  • St. John’s wort
  • antifungal medicines such as ketoconazole, itraconazole (SPORANOX®), posaconazole (NOXAFIL®), voriconazole (VFEND®), or fluconazole (DIFLUCAN®)
  • antibiotics such as telithromycin, clarithromycin (BIAXIN®), or erythromycin (ERY-TAB®)

What should I avoid while taking KALYDECO?

  • KALYDECO can cause dizziness in some people who take it. If you experience dizziness, do not drive or operate machines until symptoms improve
  • Avoid food or drink containing grapefruit while you are taking KALYDECO

What are the possible side effects of KALYDECO?

KALYDECO can cause serious side effects, including:

  • High liver enzymes in the blood, which have happened in people 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 people who have had high liver enzymes in the past, your 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
  • Serious allergic reactions have happened to people who are treated with KALYDECO. Call your healthcare provider or go to the emergency room right away if you have symptoms of an allergic reaction. Symptoms of an allergic reaction may include:

 

  • rash or hives
  • tightness of the chest or throat or difficulty breathing
  • light-headedness or dizziness
  • Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.

The most common side effects of KALYDECO 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.

Use of KALYDECO in children aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.

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.

People with CF pictured may or may not be taking KALYDECO.

    Indication

    What is KALYDECO® (ivacaftor)?

    KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) 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 1 month of age.

      Important safety information

      IMPORTANT SAFETY INFORMATION

      Before taking KALYDECO, tell your doctor about all of your medical conditions, including if you:

      • have liver or kidney problems
      • are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
      • 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

      Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

      KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

      Especially tell your doctor if you take:

      • the antibiotics rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
      • seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
      • St. John’s wort
      • antifungal medicines such as ketoconazole, itraconazole (SPORANOX®), posaconazole (NOXAFIL®), voriconazole (VFEND®), or fluconazole (DIFLUCAN®)
      • antibiotics such as telithromycin, clarithromycin (BIAXIN®), or erythromycin (ERY-TAB®)

      What should I avoid while taking KALYDECO?

      • KALYDECO can cause dizziness in some people who take it. If you experience dizziness, do not drive or operate machines until symptoms improve
      • Avoid food or drink containing grapefruit while you are taking KALYDECO

      What are the possible side effects of KALYDECO?

      KALYDECO can cause serious side effects, including:

      • High liver enzymes in the blood, which have happened in people 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 people who have had high liver enzymes in the past, your 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
      • Serious allergic reactions have happened to people who are treated with KALYDECO. Call your healthcare provider or go to the emergency room right away if you have symptoms of an allergic reaction. Symptoms of an allergic reaction may include:

       

      • rash or hives
      • tightness of the chest or throat or difficulty breathing
      • light-headedness or dizziness
      • Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.

      The most common side effects of KALYDECO 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.

      Use of KALYDECO in children aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.

      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.

      People with CF pictured may or may not be taking KALYDECO.

      IMPORTANT SAFETY INFORMATION

      Before taking KALYDECO, tell your doctor about all of your medical conditions, including if you:

      • have liver or kidney problems
      • are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
      • 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

      What is KALYDECO® (ivacaftor)?

      KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) 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 1 month of age.