Results With KALYDECO®

KALYDECO is for people with cystic fibrosis (CF) age 6 months and older with at least 1 mutation in their CF gene responsive to KALYDECO. It is not known if KALYDECO is safe and effective in children under 6 months of age.

The KALYDECO studies included patients in certain age groups and with certain mutations.

Age 6 months to less than 1 year old: Study 8: G178R, G551D.
Age 1 to less than 2 years old: Study 8: G178R, G551D, S549N.
Age 2 to less than 6 years old: Study 6: G551D, S549N.
Age 6 years and older: Study 2 (age 6 through 11): G551D. Study 4 (age 6 years and older): G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, S549R. Study 5 (age 6 years and older): R117H.
Age 12 years and older: Study 1: G551D. Study 7: 2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, A455E, D1152H, D579G, E831X, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, S977F.

*KALYDECO is not indicated for people with CF who have the G970R mutation.

Keep in mind that not all mutations were evaluated in clinical studies, and those that were evaluated were not studied in all age groups. However, KALYDECO is approved for people age 6 months and older with any of 38 indicated mutations.

Study 8


Children 6 months to less than 1 year old

Study 8 was a 24-week safety study of KALYDECO in children less than 2 years old. Nineteen children age 1 to less than 2 years old and 11 children age 6 months to less than 1 year old with CF were evaluated in this study. All patients enrolled took KALYDECO. No one in the study took placebo. The primary purpose of this study was to determine the safety and tolerability of KALYDECO.

See the results for children age 1 to less than 2 years old.

Mutations eligible to enroll in this study were:

G1244E, G1349D, G178R, G551D, G551S, R117H, S1251N, S1255P, S549N, S549R.

Mutations included in this study:

10 children had the G551D mutation, and 1 had the G178R mutation.

How KALYDECO was given

scale showing 15.4 to less than 31 pounds

Children who weighed ~15 pounds (7 kg) to less than ~31 pounds (14 kg) received 50 mg of KALYDECO oral granules.

All children took KALYDECO oral granules mixed with 1 teaspoon of age-appropriate soft food or liquid every 12 hours with fat-containing food. All children continued to take their other CF treatments.

Please note: A 25-mg dose is available for children who weigh ~11 pounds (5 kg) to less than ~15 pounds (7 kg).

Learn how to give your child KALYDECO oral granules.

Study results

Safety
exclamation mark

The safety of KALYDECO, observed in this study, was similar to what was observed in KALYDECO studies in people with CF age 2 years and older.

See additional safety information.

Other study results

Sweat chloride
decreased in sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -58.6 mmol/L at Week 24.

(average mmol/L beginning of study was 101.5)

Sweat chloride is the amount of salt in your child’s sweat. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1).

Study limitations

Because no one in this study took placebo, it is not known if changes in sweat chloride were due to KALYDECO.

KALYDECO was approved in people age 6 months to less than 1 year old based on the efficacy shown in studies of KALYDECO in older people as well as the safety assessment in this study.

Talk to your healthcare provider to learn more about how KALYDECO was approved in this age group.

Study 8


Children 1 year to less than 2 years old

Study 8 was a 24-week safety study of KALYDECO in children less than 2 years old. Nineteen children age 1 to less than 2 years old and 11 children age 6 months to less than 1 year old with CF were evaluated in this study. All patients enrolled took KALYDECO. No one in the study took placebo. The primary purpose of this study was to determine the safety and tolerability of KALYDECO.

See the results for children age 6 months to less than 1 year old.

Mutations eligible to enroll in this study were:

G1244E, G1349D, G178R, G551D, G551S, R117H, S1251N, S1255P, S549N, S549R.

Mutations included in this study:

16 children had the G551D mutation, 2 had the S549N mutation, and 1 had the G178R mutation.

How KALYDECO was given

scale showing 15.4 to less than 31 pounds

Children who weighed ~15 pounds (7 kg) to less than ~31 pounds (14 kg) received 50 mg of KALYDECO oral granules.

scale showing greater than or equal to 31 pounds

Children who weighed ~31 pounds (14 kg) or more received 75 mg of KALYDECO oral granules.

All children took KALYDECO oral granules mixed with 1 teaspoon of age-appropriate soft food or liquid every 12 hours with fat-containing food. All children continued to take their other CF treatments.

Learn how to give your child KALYDECO oral granules.

Study results

Safety
exclamation mark

The safety of KALYDECO, observed in this study, was similar to what was observed in KALYDECO studies in people with CF age 2 years and older.

See additional safety information.

Other study results

Sweat chloride
decreased in sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -73.5 mmol/L at Week 24.

(average mmol/L beginning of study was 104.1)

Sweat chloride is the amount of salt in your child’s sweat. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1).

Study limitations

Because no one in this study took placebo, it is not known if changes in sweat chloride were due to KALYDECO.

KALYDECO was approved in people age 1 to less than 2 years old based on the benefits shown in studies of KALYDECO in older people as well as the safety assessment in this study.

Talk to your healthcare provider to learn more about how KALYDECO was approved in this age group.

Study 6


Study 6 was a 24-week study in which the safety of KALYDECO was evaluated in 34 children age 2 to less than 6 years old with CF. All children in this study took KALYDECO. No one in this study took placebo. The primary purpose of this study was to determine the safety and tolerability of KALYDECO.

Mutations eligible to enroll in this study were:

G1244E, G1349D, G178R, G551D, G551S, G970R,* S1251N, S1255P, S549N, S549R.

*KALYDECO is not indicated for people with CF who have the G970R mutation.

Mutations included in this study:

32 children had the G551D mutation and 2 had the S549N mutation.

How KALYDECO was given

scale showing less than 31 pounds

Children who weighed less than ~31 pounds (14 kg) received 50 mg of KALYDECO oral granules.

scale showing greater than or equal to 31 pounds

Children who weighed ~31 pounds (14 kg) or more received 75 mg of KALYDECO oral granules.

All children took KALYDECO oral granules mixed with 1 teaspoon of soft food or liquid every 12 hours with fat-containing food. All children continued to take their other CF treatments.

Learn how to give your child KALYDECO oral granules.

Study results

Safety
exclamation mark

The type and frequency of side effects for children in the study were similar to those seen in KALYDECO studies with people with CF age 6 years and older.

High liver enzymes were more common in children who had abnormal liver enzymes before starting KALYDECO.

See additional safety information.

Other study results

Sweat chloride
decreased in sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -45 mmol/L at Week 24.

Sweat chloride is the amount of salt in your child’s sweat. A decrease in sweat chloride levels does not mean there will be an improvement in lung function (FEV1).

Study limitations

Because no one in this study took placebo, it is not known if changes in sweat chloride were due to KALYDECO.

KALYDECO was approved in people age 2 to less than 6 years old based on the benefits shown in studies of KALYDECO in older people as well as the safety assessment in this study.

Talk to your healthcare provider to learn more about how KALYDECO was approved in this age group.

Study 2


Study 2 was a 48-week study in which 52 people age 6 to less than 12 years old with a G551D mutation took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. Results were measured at different time points including 24 and 48 weeks. All people took 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, weight, and sweat chloride. The study also evaluated the safety of KALYDECO.

Mutation eligible to enroll and included in this study was:

G551D.

Study results

Lung function (FEV1)
increase in lung function

Lung function increased

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

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

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

Other study results

CF respiratory symptoms
person coughing

No difference was seen in respiratory symptoms

Compared to placebo 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.

Weight
weight increase

Weight increased

About 6 pounds (2.8 kg) on average, compared to placebo at Week 48.

Sweat chloride
decreased in sweat chloride

After taking KALYDECO:

Sweat chloride decreased on average by -53 mmol/L compared to placebo at Week 48.

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

KALYDECO has also been studied in people age 12 and older who have the G551D mutation. Be sure to review the information on this study in the Age 12 years and older section of this page.

Study 4

In Study 4, 39 people age 6 and older who had an eligible CF mutation (listed below) took KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours. All people took all their other CF treatments except for hypertonic saline. Each person had an 8-week treatment period with KALYDECO and an 8-week treatment period with placebo. The study evaluated lung function (FEV1). Other results studied were CF respiratory symptoms, body mass index (BMI), and sweat chloride. The study also evaluated the safety of KALYDECO.

Mutations eligible to enroll and included in this study were:

G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, S549R.

*KALYDECO is not indicated for people with CF who have the G970R mutation.

Important considerations

  • Results for the mutations studied 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
  • Talk to your healthcare provider to learn more about your specific mutation

Study results

Lung function (FEV1)
increase in lung function

Lung function increased

In the overall population for the 9 mutations studied, people had a 10.7 percentage points average improvement in lung function compared to placebo, from the start of the study through Week 8.

Results varied by mutation.

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

Other study results

CF respiratory symptoms
improvement in coughing

CF respiratory symptoms improved

In the overall population for the 9 mutations studied, people had a 9.6 point average improvement in CF respiratory symptoms compared to placebo, from the start of the study through 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.

Body mass index (BMI)
increase in body mass index

BMI increased

In the overall population for the 9 mutations studied, people had a 0.66 kg/m2 on average increase in BMI compared to placebo at Week 8.

Results varied by mutation.

BMI is a measure of someone’s weight in relation to his or her height. For example, for a 5'4", 110-lb adult, a 0.66 kg/m2 increase in BMI would be a gain of about 4 lb; for a 4'6", 65-lb child, this would be a gain of about 3 lb.

Sweat chloride
decreased in sweat chloride

Sweat chloride decreased

In the overall population for the 9 mutations studied, people had a -50 mmol/L average decrease in sweat chloride compared to placebo from the start of the study through Week 8.

Results varied by mutation.

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

Study 5

Study 5 was a 24-week study in which 69 people age 6 and older with an R117H mutation took either KALYDECO (150-mg tablets) or placebo with fat-containing food every 12 hours, along with their other CF treatments. The study evaluated lung function (FEV1). Other results studied were sweat chloride, CF respiratory symptoms, body mass index (BMI), and pulmonary exacerbations. The study also evaluated the safety of KALYDECO.

Mutation eligible to enroll and included in this study was:

R117H.

What was studied?

pair of lungs

Lung function (FEV1) was the main measure of this study

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

sweat chloride

Sweat chloride

person coughing

CF respiratory symptoms
(cough, mucus, and trouble breathing)

scale and measuring tape

Body mass index (BMI)

pulmonary exacerbations

Pulmonary exacerbations

exclamation mark

Safety

Some people in this study showed improvement in these measures, but others did not.

  • Please talk to your healthcare provider if you’d like to learn about the results of this study

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.

Mutation eligible to enroll and included in this study was:

G551D.

Study results

Lung function (FEV1)
increase in lung function

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

Other study results

CF respiratory symptoms
improvement in coughing

CF respiratory symptoms improved

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
decrease in 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 increase

Weight increased

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

Sweat chloride
decreased in sweat chloride

After taking KALYDECO:

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

Sweat chloride is the amount of salt in your 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 except for hypertonic saline. 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)
increase in lung function

Lung function increased

4.7 percentage points on average in the overall population studied compared to placebo, from the start of the study to the average of Week 4 and Week 8.

Results varied by mutation.

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

Other study results

CF respiratory symptoms
improvement in coughing

CF respiratory symptoms improved

9.7 points on average in the overall population studied 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.

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 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 6 months of age.

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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.

EXPAND

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 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 6 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.

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.