KALYDECO® (ivacaftor) Study Results
for Children Age 2 to Less Than 6 Years
Study 6 (in patients 2 to less than 6 years)
In a 24-week, open-label, clinical study, KALYDECO oral granules were given to 34 children who were 2 to less than 6 years of age. Patients with the following mutations were eligible for this study: G551D, G1244E, G1349D, G178R, G551S, G970R,* S1251N, S1255P, S549N, or S549R. Of 34 patients enrolled, 32 had the G551D mutation and 2 had the S549N mutation.
*KALYDECO is not indicated for people with CF who have the G970R mutation.
Study 6 was a 24-week study in which all children took KALYDECO. Safety and tolerability was the main endpoint, or measure, of the pediatric clinical study.
Children who weighed 31 lbs (14 kg) or less received KALYDECO oral granules (50 mg) mixed in 1 teaspoon (5 mL) of soft food or liquid every 12 hours with fat-containing food.
Children who weighed more than 31 lbs (14 kg) received KALYDECO oral granules (75 mg) mixed in 1 teaspoon (5 mL) of soft food or liquid every 12 hours with fat-containing food.
The type and frequency of side effects for children age 2 to less than 6 years who participated in the 24-week study (Study 6) were similar to those seen in other clinical studies with patients 6 years and older.
High liver enzymes were more common in children who had abnormal liver enzymes before starting KALYDECO.
Learn about safety information for KALYDECO in people 6 years and older.
Select Safety Information
- KALYDECO can cause serious side effects
- High liver enzymes in the blood have been reported in patients receiving KALYDECO
- The doctor will do blood tests to check the liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while 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 the doctor right away if the patient has any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of his/her eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine
- Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. The 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), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness
Tell the doctor if the patient has any side effect that bothers him/her or that does not go away. These are not all the possible side effects of KALYDECO. For more information, ask the doctor or pharmacist.
If you have any questions about KALYDECO, talk to your child’s healthcare provider.
Sweat chloride results
A decrease in sweat chloride levels does not always 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 2 years and older who have one of the following mutations in their CF gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R.
KALYDECO is used for the treatment of CF in patients age 2 years and older who have an R117H mutation in their CF gene.
KALYDECO is not for use in people with CF due to other mutations in the CF gene. KALYDECO is not effective in patients with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.
It is not known if KALYDECO is safe and effective in children under 2 years 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 medications such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, Equetro®) or phenytoin (Dilantin®, Phenytek®); or St. John’s wort.
Talk to your doctor before taking KALYDECO if you take any of these medicines or supplements.