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.

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 6 pounds (2.7 kg) 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 at Week 48.

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 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)

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

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

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

What is KALYDECO® (ivacaftor)?

What is KALYDECO®
(ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 4 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 4 months of age.

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
  • 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 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 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.

Call your doctor for medical advice about side effects. You are encouraged to 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.