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RESULTS FOR PEOPLE WITH F508DEL/F508DEL MUTATIONS (STUDY 1)

Aiden age 13, with gene mutation F508del/F508del

SYMDEKO is for people with cystic fibrosis (CF) age 12 years and older with 2 copies of the F508del-CFTR 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 12 years.

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

Study details

This 24-week (6-month) clinical study included people with CF age 12 years and older with 2 copies of the F508del mutation in the CF gene. SYMDEKO and placebo were compared to determine the possible benefits and risks of SYMDEKO.

  • 248 people with CF took SYMDEKO and 256 took placebo every 12 hours with fat-containing food
  • Participants continued to take their other prescribed CF therapies

SYMDEKO improved lung function and reduced pulmonary exacerbations

pair of lungs

Lung function (FEV1)*

through 24 weeks
4PERCENTAGE POINT
IMPROVEMENT
VS PLACEBO

Improved lung function by 4 percentage points on average compared with those who took placebo.

change in lung function over time: SYMDEKO vs placebo
lung function chart labels
lung function chart

lung function chart labels

acute worsening of symptoms

Pulmonary exacerbations

through 24 weeks
DECREASED BY
35%
VS PLACEBO

Lowered the chance of having a pulmonary exacerbation by 35% on average compared with those who took placebo.

Additional clinical study results

bathroom scale and tape measure

Body Mass Index (BMI)

at 24 weeks

Increased by 0.06 kg/m2 on average compared with those who took placebo.

  • For example, a person who is 5’4” and 110 pounds would gain 0.35 pounds, or a person who is 5’10” and 160 pounds would gain 0.42 pounds
  • It cannot be determined if this change was due to SYMDEKO
person breathing out

CF Respiratory Symptoms

through 24 weeks

5.1 point change in certain CF respiratory symptoms compared with those who took placebo.

  • It cannot be determined if this change was due to SYMDEKO

A tool was used to measure CF respiratory symptoms including cough, difficulty breathing, and mucus production.

FEV1=forced expiratory volume in 1 second.
Respiratory symptoms were measured by the CFQ-R Respiratory Domain Score, which is a tool used in research to measure CF respiratory symptom improvement.

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 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.

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