Safety & Side Effects

KALYDECO is for people with cystic fibrosis (CF) age 12 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 younger children.

How the safety of KALYDECO^® was assessed in clinical studies

The overall safety profile of KALYDECO is based on data from 3 placebo-controlled studies. The safety data from the 3 clinical studies were combined and reviewed together, or "pooled," to arrive at the safety profile for KALYDECO.

Across these studies, a total of 353 people 6 years of age and older with the G551D mutation (Studies 1 and 2) or 2 copies of the F508del* mutation (Study 3) in their CFTR gene took KALYDECO or placebo every 12 hours with fat-containing food. 221 people received KALYDECO and 132 people received placebo.

*KALYDECO is not approved and is not effective in people with CF with two copies of the F508del mutation (F508del/F508del) in the CF gene.

In addition, safety was assessed in the following clinical studies:

Study 4: Study of KALYDECO in 39 people with CF age 6 years and older with a G1244E, G1349D, G178R, G551S, G970R,^† S1251N, S1255P, S549N, or S549R mutation. People in the study took either KALYDECO (150 mg tablets) or placebo 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
^†KALYDECO is not indicated for people with CF who have the G970R mutation.
Study 5: A 24-week study in which KALYDECO was studied in 69 people with CF age 6 years and older with an R117H mutation. People in the study took either KALYDECO (150 mg tablets) or placebo every 12 hours with fat-containing food
Study 6: A 24-week study in which KALYDECO was studied in 34 children with CF age 2 to less than 6 years with a G551D or S549N mutation. All children in this study took KALYDECO (50 mg or 75 mg oral granules) every 12 hours with fat-containing food. No one in this study took placebo
Study 7: An 8-week study in which KALYDECO was studied in people with CF age 12 years and older with 1 copy of the F508del mutation and a 2789+5G→A, 3272-26A→G, 3849+10kbC→T, 711+3A→G, A455E, D1152H, D579G, E831X, L206W, P67L, R1070W, R117C, R347H, R352Q, S945L, or S977F mutation. In this study, 156 people took KALYDECO (150 mg tablets) and 161 people took placebo every 12 hours with fat-containing food
Study 8: A 24-week study in which KALYDECO was studied in 19 children with CF age 12 months to less than 2 years with a G178R, G551D, or S549N mutation. All children in this study took KALYDECO (50 mg or 75 mg oral granules) every 12 hours with fat-containing food. No one in this study took placebo

The safety profile for Studies 4, 5, 6, 7, and 8 was similar to that observed in Studies 1 and 2.

Remember, this website is for informational purposes only and is not intended to be a substitute for professional medical advice. You should talk to your healthcare provider about the clinical study results and the possible side effects of 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.

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.

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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.