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 43 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. Seven of the children were age 1 month to less than 4 months. All children in this study took KALYDECO 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, including:
High liver enzymes in the blood, which have happened in people 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 people who have had high liver enzymes in the past, your 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
Serious allergic reactions have happened to people who are treated with KALYDECO.
Call your healthcare provider or go to the emergency room right away if you have symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
- rash or hives
- tightness of the chest or throat or difficulty breathing
- light-headedness or dizziness
Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO.
Your doctor should perform eye examinations before 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.
Before taking KALYDECO®
Before taking KALYDECO, tell your doctor about all of your medical conditions, including if you:
- have liver or kidney problems
- are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
- 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
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Ask your doctor or pharmacist for a list of these medicines if you are not sure.
Especially tell your doctor if you take:
| Antibiotics | Seizure medicines | Herbal supplement |
|---|---|---|
|
rifampin rifabutin |
phenobarbital carbamazepine phenytoin |
St. John’s wort |
| Antifungal medicines | Antibiotics | |
|
ketoconazole itraconazole posaconazole voriconazole fluconazole |
telithromycin clarithromycin erythromycin |
| Antibiotics |
| rifampin (RIFAMATE®, RIFATER®) rifabutin (MYCOBUTIN®) |
| Seizure medications |
| phenobarbital
carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®) phenytoin (DILANTIN®, PHENYTEK®) |
| Herbal supplement |
| St. John’s wort |
| Antifungal medications |
| ketoconazole
itraconazole (SPORANOX®) posaconazole (NOXAFIL®) voriconazole (VFEND®) fluconazole (DIFLUCAN®) |
| Antibiotics |
| telithromycin
clarithromycin (BIAXIN®) erythromycin (ERY-TAB®) |
IMPORTANT SAFETY INFORMATION
Before taking KALYDECO, tell your doctor about all of your medical conditions, including if you:
- have liver or kidney problems
- are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
- 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
What is KALYDECO® (ivacaftor)?
What is KALYDECO®
(ivacaftor)?
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) 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 1 month of age.
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Ask your doctor or pharmacist for a list of these medicines if you are not sure.
Especially tell your doctor if you take:
- the antibiotics rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
- seizure medicines such as phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
- St. John’s wort
- antifungal medicines such as ketoconazole, itraconazole (SPORANOX®), posaconazole (NOXAFIL®), voriconazole (VFEND®), or fluconazole (DIFLUCAN®)
- antibiotics such as telithromycin, clarithromycin (BIAXIN®), or erythromycin (ERY-TAB®)
What should I avoid while taking KALYDECO?
- KALYDECO can cause dizziness in some people who take it. If you experience dizziness, do not drive or operate machines until symptoms improve
- Avoid food or drink containing grapefruit while you are taking KALYDECO
What are the possible side effects of KALYDECO?
KALYDECO can cause serious side effects, including:
- High liver enzymes in the blood, which have happened in people 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 people who have had high liver enzymes in the past, your 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
- Serious allergic reactions have happened to people who are treated with KALYDECO. Call your healthcare provider or go to the emergency room right away if you have symptoms of an allergic reaction. Symptoms of an allergic reaction may include:
- rash or hives
- tightness of the chest or throat or difficulty breathing
- light-headedness or dizziness
- Abnormality of the eye lens (cataract), which has happened in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations before 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.
Use of KALYDECO in children aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.
Call your doctor for medical advice about side effects. You may 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.