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RESULTS FOR PEOPLE WITH OTHER RESPONSIVE MUTATIONS (STUDY 2)

Girl with other responsive gene mutation

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, all results shown are an average of all people studied and differed among individuals and mutations. Your experience may be different.

Study details

In a clinical study of 244 people with CF, SYMDEKO was compared with ivacaftor alone and placebo to determine the possible benefits and risks of SYMDEKO. Participants were age 12 years and older with the F508del mutation and a second mutation in the CF gene predicted to respond to SYMDEKO.

Mutations included in the clinical study
711+3AG

c.579+3A>G

A455E

c.1364C>A

E831X

c.2491G>T

R347H

c.1040G>A

S977F

c.2930C>T

2789+5GA

c.2657+5G>A

D110H

c.328G>C

L206W

c.617T>G

R352Q

c.1055G>A

3272-26AG

c.3140-26A>G

D579G

c.1736A>G

P67L

c.200C>T

R1070W

c.3208C>T

3849+10kbCT

c.3718-2477C>T

D1152H

c.3454G>C

R117C

c.349C>T

S945L

c.2834C>T

711+3AG

c.579+3A>G

L206W

c.617T>G

2789+5GA

c.2657+5G>A

P67L

c.200C>T

3272-26AG

c.3140-26A>G

R117C

c.349C>T

3849+10kbCT

c.3718-2477C>T

R347H

c.1040G>A

A455E

c.1364C>A

R352Q

c.1055G>A

D110H

c.328G>C

R1070W

c.3208C>T

D579G

c.1736A>G

S945L

c.2834C>T

D1152H

c.3454G>C

S977F

c.2930C>T

E831X

c.2491G>T

  • Each person had two 8-week treatment periods where they took either SYMDEKO, ivacaftor alone, or placebo every 12 hours with fat-containing food. Participants received 2 of the 3 possible treatments after completing both treatment periods
    • 161 patients took SYMDEKO, 156 patients took ivacaftor, and 161 patients took placebo
    • Between treatment periods there was an 8-week washout period where they did not take SYMDEKO, ivacaftor, or placebo
  • Participants continued to take their other prescribed CF therapies

SYMDEKO improved lung function

pair of lungs

Lung function (FEV1)*

average at Weeks 4 and 8
6.8

PERCENTAGE POINT
IMPROVEMENT
VS PLACEBO

2.1

PERCENTAGE POINT
IMPROVEMENT
VS IVACAFTOR

Improved lung function overall by an average of 6.8 percentage points compared with placebo. Additionally, improved lung function by an average of 2.1 percentage points compared with ivacaftor alone.

change in lung function over time: tezacaftor/ivacaftor vs ivacaftor vs placebo
lung function chart labels
change in lung function over time: tezacaftor/ivacaftor vs ivacaftor vs placebo

Changes in lung function varied by mutations (range -1.0 to 10.1 percentage points compared with placebo) and individuals.

Additional clinical study results

person breathing out

CF Respiratory Symptoms

average at Weeks 4 and 8

11.1 point overall improvement on average in CF respiratory symptoms compared with placebo

1.4 point change on average in CF respiratory symptoms compared with ivacaftor alone

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

Changes in CF respiratory symptoms varied by mutations (range -11.1 to 29.2 points) and individuals.

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