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Vertex GPS™: Guidance & Patient Support

SYMDEKO® RESULTS

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

Study details

 

In a safety study of 70 children with cystic fibrosis (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

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

Sweat chloride levels through 24 weeks  

Decreased sweat chloride levels by 14.5 mmol/L on average

Sweat chloride is a measure of the amount of salt in a child’s sweat (mmol/L). People with CF have high levels of sweat chloride.

-14.5

DECREASED BY
mmol/L

Lung function (FEV1)* through 24 weeks  

Lung function changed by 0.9 percentage points on average

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second. Lung function was part of the safety assessment in this trial. 

+0.9

PERCENTAGE
POINT CHANGE

*FEV1=percent predicted forced expiratory volume in 1 second.

Study details

 

In a clinical study of 504 people with cystic fibrosis (CF), SYMDEKO and placebo were compared to determine the possible benefits and risks of SYMDEKO. Participants were age 12 years and older with 2 copies of the F508del mutation in the CF gene.

  • 248 people took SYMDEKO and 256 took placebo every 12 hours with fat-containing food for 24 weeks (~6 months)
  • Participants continued to take their other prescribed CF therapies

SYMDEKO improved lung function and reduced pulmonary exacerbations

Lung function (FEV1)*  through 24 weeks  

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

4

PERCENTAGE POINT

IMPROVEMENT

VS PLACEBO

Graph showing Lung function (FEV1) through 24 weeks Graph showing Lung function (FEV1) through 24 weeks Graph showing Lung function (FEV1) through 24 weeks

 

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second.

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

Pulmonary exacerbations through 24 weeks  

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

There were 78 pulmonary exacerbations in the SYMDEKO group and 122 in the placebo group.

Pulmonary exacerbations are defined as certain symptoms that require treatment with new oral, intravenous (IV), or inhaled antibiotics.

DECREASED BY

35%

VS PLACEBO

Additional clinical study results

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
  • BMI is a measure of someone’s weight in relation to his or her height

INCREASED BY

0.06 kg/m2

VS PLACEBO

CF respiratory symptoms through 24 weeks  

5.1 point change on average 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 amount of mucus coughed up.

CHANGED BY

5.1 points

VS PLACEBO

96 weeks

View the results of a long-term study of SYMDEKO.

*FEV1=percent predicted forced expiratory volume in 1 second.

†Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised Respiratory Domain score.

Study details

 

In a clinical study of 244 people with cystic fibrosis (CF), SYMDEKO was compared with ivacaftor 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		  

View all mutations that are responsive to SYMDEKO.

  • Each person had two 8-week treatment periods where they took either SYMDEKO, ivacaftor, 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

Lung function (FEV1)* average of Weeks 4 and 8 

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.

6.8

PERCENTAGE POINT

IMPROVEMENT

VS PLACEBO

2.1

PERCENTAGE POINT

IMPROVEMENT

VS IVACAFTOR

Graph showing Lung function (FEV1) average of Weeks 4 and 8 Graph showing Lung function (FEV1) average of Weeks 4 and 8 Graph showing Lung function (FEV1) average of Weeks 4 and 8

 

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

Lung function can be measured with an FEV1 test, which measures how much air a person can exhale in a forced breath in 1 second.

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

Additional clinical study results

CF respiratory symptoms average of 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

  • 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 compared with placebo).

A tool† was used to measure CF respiratory symptoms including cough, difficulty breathing, and amount of mucus coughed up.

IMPROVED BY

11.1 POINTS

VS PLACEBO

CHANGED BY

1.4 POINTS

VS IVACAFTOR

96 weeks

View the results of a long-term study of SYMDEKO.

*FEV1=percent predicted forced expiratory volume in 1 second.

†Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised Respiratory Domain score.

Learn about the possible side effects of SYMDEKO

Get side effect and safety information.

Important safety information⁠

Important Safety Information

Before taking SYMDEKO, tell your doctor about all of your medical conditions, including if you:

  • have or have had liver problems
  • are allergic to SYMDEKO or any ingredients in SYMDEKO. See the Patient Information for a list of ingredients
  • 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

What is SYMDEKO?⁠

What is SYMDEKO® (tezacaftor/ivacaftor and ivacaftor)?

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.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

SYMDEKO may affect the way other medicines work and other medicines may affect how SYMDEKO works. The dose of SYMDEKO may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • 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
  • antifungal medicines such as ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
  • antibiotics such as telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking SYMDEKO?

  • SYMDEKO 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 that contains grapefruit 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

 

  • Serious allergic reactions have happened to people who are treated with SYMDEKO. Call your doctor or go to the emergency room right away if you have any 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) has happened 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.

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 SYMDEKO. 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.

Important safety information⁠

Important Safety Information

Before taking SYMDEKO, tell your doctor about all of your medical conditions, including if you:

  • have or have had liver problems
  • are allergic to SYMDEKO or any ingredients in SYMDEKO. See the Patient Information for a list of ingredients
  • 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

What is SYMDEKO?⁠

What is SYMDEKO® (tezacaftor/ivacaftor and ivacaftor)?

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.

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

SYMDEKO may affect the way other medicines work and other medicines may affect how SYMDEKO works. The dose of SYMDEKO may need to be adjusted when taken with certain medicines. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

  • 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
  • antifungal medicines such as ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
  • antibiotics such as telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking SYMDEKO?

  • SYMDEKO 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 that contains grapefruit 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

 

  • Serious allergic reactions have happened to people who are treated with SYMDEKO. Call your doctor or go to the emergency room right away if you have any 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) has happened 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.

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 SYMDEKO. 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.