Safety & Side Effects

How the safety of KALYDECO® was assessed in clinical studies

Serious and most common
side effects

Additional safety considerations

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 cystic fibrosis (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 evaluated in 36 children with CF. Nineteen of the children were age 12 months to less than 2 years. Eleven of the children were 6 months to less than 1 year. Six of the children were 4 months to less than 6 months. All children in this study took KALYDECO (25-mg, 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.

Serious and most common
side effects

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 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.

 


 

Additional safety considerations

Do not take KALYDECO if you take certain medicines or herbal supplements, such as:

Antibiotics Seizure medicines, such as: Herbal supplement

rifampin
(RIFAMATE®, RIFATER®)

rifabutin
(MYCOBUTIN®)

phenobarbital

carbamazepine
(TEGRETOL®, CARBATROL®, EQUETRO®)

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.
Antibiotics
rifampin
(RIFAMATE®, RIFATER®)

 

rifabutin
(MYCOBUTIN®)
Seizure medicines, such as:
phenobarbital

 

carbamazepine
(TEGRETOL®, CARBATROL®, EQUETRO®)

 

phenytoin
(DILANTIN®, PHENYTEK®)
Herbal supplement
St. John’s wort

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

 

Before taking KALYDECO®

What to tell your 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 taking certain medications.

 

Especially tell your doctor if you take antifungal medications or antibiotics such as: 
Antifungal medications Antibiotics
ketoconazole (NIZORAL®)
itraconazole (SPORANOX®)
posaconazole (NOXAFIL®)
voriconazole (VFEND®)
fluconazole (DIFLUCAN®)
telithromycin (KETEK®)
clarithromycin (BIAXIN®)
erythromycin (ERY-TAB®)
Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.
Always check with your CF treatment team before taking any new medicines.
Always talk to your doctor before taking any new medicines.
Especially tell your doctor if you take antifungal medications or antibiotics such as: 
Antifungal medications
ketoconazole (NIZORAL®)
itraconazole (SPORANOX®)
posaconazole (NOXAFIL®)
voriconazole (VFEND®)
fluconazole (DIFLUCAN®)
Antibiotics
telithromycin (KETEK®)
clarithromycin (BIAXIN®)
erythromycin (ERY-TAB®)
Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.
Always check with your CF treatment team before taking any new medicines.
Always talk to your doctor before taking any new medicines.