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RESULTS FOR CHILDREN AGE 6 THROUGH 11 YEARS

Aiden age 13, with gene mutation F508del/F508del

SYMDEKO is for people with cystic fibrosis (CF) age 6 years and older with 2 copies of the F508del mutation, 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.

Keep in mind, results shown are an average for all people studied and differed among individuals and mutations. Your child may have a different experience.

Study details

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.

  • 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

Safety study results

pair of lungs

SAFETY

through 24 weeks

The safety of SYMDEKO observed in the study was similar to what was observed in people age 12 years and older.

Please see Important Safety Information.

View Side Effects and Additional Safety Information in people age 12 years and older.

Safety study considerations

  • Because no one took placebo in the safety study, it is not known if the changes in lung function and sweat chloride levels were due to SYMDEKO
  • Changes in sweat chloride levels observed in the study are not related to changes in lung function
  • Keep in mind, all results shown are an average of all people studied and differed among individuals and mutations. Your child may have a different experience

Additional safety study results

pair of lungs

Sweat Chloride Levels

through 24 weeks
DECREASED BY
-14.5
mmol/L
4 PERCENTAGE POINT
IMPROVEMENT
VS PLACEBO

Decreased sweat chloride levels by 14.5 mmol/L on average

pair of lungs

Lung function (FEV1)*

through 24 weeks
+0.9 PERCENTAGE POINT
CHANGE
4 PERCENTAGE POINT
IMPROVEMENT
VS PLACEBO

Lung function changed by 0.9 percentage points on average

FEV1=percent predicted forced expiratory volume in 1 second

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.

Especially tell your doctor if you take:
  • 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?

SYMDEKO can cause serious side effects, including:
  • 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.

Click for full safety