HOW SYMDEKO WAS STUDIED
SYMDEKO is for people with cystic fibrosis (CF) age 6 years and older with 2 copies of the F508del mutation in the CF gene or with at least 1 mutation in the CF gene that is responsive to treatment with SYMDEKO. It is not known if SYMDEKO is safe and effective in patients younger than 6 years of age.
Study in children age 6 through 11 years
In a safety study of 70 children with CF, the safety of SYMDEKO was evaluated. Participants were age 6 through 11 years with 2 copies of the F508del mutation or 1 copy of the F508del mutation and a second mutation in the CF gene predicted to respond to SYMDEKO.
Safety study
The primary purpose of the 24-week (~6-month) safety study of 70 children age 6 through 11 years with eligible mutations was to determine the safety of SYMDEKO.
The study also evaluated changes in lung function and sweat chloride levels.
Lung function is determined with an FEV1 test, which measures how much air a person can exhale in a forced breath. Lung function was part of the safety assessment in this trial.
Sweat chloride is a measure of the amount of salt in a child’s sweat (mmol/L). Sweat chloride level measurement is used to help diagnose CF. High sweat chloride levels are a hallmark of CF.
How SYMDEKO was given
- Each child took SYMDEKO every 12 hours with fat-containing food for 24 weeks (~6 months)
- Participants continued to take their other prescribed
CF therapies - All patients knew they were taking SYMDEKO, and no children in the study took placebo
Studies in people age 12 years and older
clinical setting
Additional mutations that were not included in a clinical study, and are predicted to respond based on results from a laboratory setting:
|
A1067T
c.3199G>A |
E56K
c.166G>A |
F1074L
c.3222T>A |
|
D110E
c.330C>A |
E193K
c.577G>A |
K1060T
c.3179A>C |
|
D1270N
c.3808G>A |
F1052V
c.3154T>G |
R74W
c.220C>T |
Important Safety Information
Do not take SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) if you take certain medicines or herbal supplements such as:
- antibiotics such as rifampin (Rifamate®, Rifater®) or rifabutin (Mycobutin®)
- seizure medicines such as phenobarbital, carbamazepine (Tegretol®, Carbatrol®, Equetro®), or phenytoin (Dilantin®, Phenytek®)
- St. John’s wort
Talk to your doctor before taking SYMDEKO if you take any of the medicines or herbal supplements listed above.
Before taking SYMDEKO, tell your doctor about all of your medical conditions, including if you:
- have or have had liver problems
- have kidney problems
- are pregnant or plan to become pregnant. It is not known if SYMDEKO will harm your unborn baby. You and your doctor should decide if you will take SYMDEKO while you are pregnant
- are breastfeeding or planning to breastfeed. It is not known if SYMDEKO passes into your breast milk. You and your doctor should decide if you will take SYMDEKO while you are breastfeeding
SYMDEKO may affect the way other medicines work, and other medicines may affect how SYMDEKO works.
Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements, because the dose of SYMDEKO may need to be adjusted when taken with certain medicines.
- antifungal medicines 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 SYMDEKO?
- SYMDEKO 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 SYMDEKO affects you
- Avoid food or drink that contains grapefruit or Seville oranges while you are taking SYMDEKO
What are the possible side effects of SYMDEKO?
- High liver enzymes in the blood have been reported in people treated with
SYMDEKO or treated with ivacaftor alone. Your doctor will do blood tests to check your liver:
- before you start SYMDEKO
- every 3 months during your first year of taking SYMDEKO
- every year while you are taking SYMDEKO
Your doctor may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past.
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) in some children and adolescents treated with SYMDEKO or with ivacaftor alone. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with SYMDEKO to look for cataracts
The most common side effects of SYMDEKO include headache, nausea, sinus congestion, and dizziness.
These are not all the possible side effects of SYMDEKO. 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.
What is SYMDEKO?
SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO.
Talk to your doctor to learn if you have an indicated CF gene mutation.
It is not known if SYMDEKO is safe and effective in children under 6 years of age.
What is SYMDEKO?
SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO.
Talk to your doctor to learn if you have an indicated CF gene mutation.
It is not known if SYMDEKO is safe and effective in children under 12 years of age.
