Study 1
Study 1
Study 1
How TRIKAFTA was studied
How TRIKAFTA was studied
How TRIKAFTA was studied
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with placebo.
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with placebo.
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with placebo.
All people in this study had one copy of the F508del mutation. “A mutation defined in the study” refers to mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
All people in this study had one copy of the F508del mutation. “A mutation defined in the study” refers to mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
All people in this study had one copy of the F508del mutation. “A mutation defined in the study” refers to mutations that either do not make a CFTR protein or make a protein that is not responsive to ivacaftor and tezacaftor/ivacaftor.
403 people with cystic fibrosis (CF), 12 years and older, with one copy of the F508del mutation and a mutation defined in the study, participated in the 24-week study.
200 people took TRIKAFTA with fat-containing food.
Two tablets, each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg in the morning, and 1 tablet containing ivacaftor 150 mg in the evening about 12 hours later
203 people took placebo twice daily with fat-containing food about 12 hours apart.
All participants continued to take their prescribed CF therapies.
Study 1
Study 1
Study 1
Results
Results
Results
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
At 4 Weeks, Lung Function (FEV1*) Improved Significantly
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
Lung Function (FEV1*) Improvement Was Maintained Through 24 Weeks
Fewer Pulmonary Exacerbations
Through 24 weeks, the number of pulmonary exacerbations significantly decreased by 63% for people taking TRIKAFTA compared with placebo.
There were 41 pulmonary exacerbations in the TRIKAFTA group and 113 in the placebo group.
Pulmonary exacerbations are changes in certain symptoms that require treatment with oral, intravenous (IV), or inhaled antibiotics.
Additional Pulmonary Exacerbation Results
71% fewer pulmonary exacerbations that led to hospitalizations through 24 weeks.
9 in the TRIKAFTA group and 32 in the placebo group
78% fewer pulmonary exacerbations that led to IV antibiotics through 24 weeks.
11 in the TRIKAFTA group and 51 in the placebo group
This study was not designed to determine whether these changes were because of TRIKAFTA. These additional results are not included in the full Prescribing Information for TRIKAFTA.
This study was not designed to determine whether these changes were because of TRIKAFTA. These additional results are not included in the full Prescribing Information for TRIKAFTA.
This study was not designed to determine whether these changes were because of TRIKAFTA. These additional results are not included in the full Prescribing Information for TRIKAFTA.
Decrease in Sweat Chloride
Significant decrease of 41.2 mmol/L on average compared with placebo at 4 weeks. Results were maintained throughout the study, with a decrease of 41.8 mmol/L on average compared with placebo through 24 weeks.
On average, people taking TRIKAFTA started the study with a sweat chloride level of 102.3 mmol/L.
Sweat chloride is a measure of the amount of salt in a person's sweat.
Improvement in CF Respiratory Symptoms
People taking TRIKAFTA reported a significant 20.1-point average increase in CF respiratory symptom score compared with placebo at 4 weeks. Results were maintained throughout the study, with an increase of 20.2 points on average compared with placebo through 24 weeks.
On average, people taking TRIKAFTA began the study with a score of 68.3 points.
Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score.
The average increase in CFQ-R Respiratory Domain score means that, overall, the symptoms studied have improved. It does not mean there was an improvement in each symptom measured.
Increase in Body Mass Index (BMI†)
Significant BMI increase of 1 kg/m2 on average compared with placebo at 24 weeks.
†BMI=a measure of someone’s weight in relation to their height.
See the side effects reported in this study (Study 1), which included people 12 years and older with one copy of the F508del mutation and a mutation defined in the study.
Study 2
Study 2
Study 2
How TRIKAFTA was studied
How TRIKAFTA was studied
How TRIKAFTA was studied
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), a prescription medicine used for the treatment of people with cystic fibrosis (CF) with two F508del mutations. See Indication and Important Safety Information for SYMDEKO.
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), a prescription medicine used for the treatment of people with cystic fibrosis (CF) with two F508del mutations. See Indication and Important Safety Information for SYMDEKO.
This study was designed to determine the possible benefits and risks of TRIKAFTA compared with SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), a prescription medicine used for the treatment of people with cystic fibrosis (CF) with two F508del mutations. See Indication and Important Safety Information for SYMDEKO.
107 people with CF, 12 years and older, with two copies of the F508del mutation,
participated in the study.
For the first 4 weeks, everyone took SYMDEKO.
Then, people were randomly split into 2 groups:
55 people switched to TRIKAFTA for 4 weeks. They took TRIKAFTA with fat-containing food.
Two tablets, each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg in the morning, and 1 tablet containing ivacaftor 150 mg in the evening about 12 hours later.
52 people continued taking SYMDEKO for 4 more weeks. They took SYMDEKO with fat-containing food.
One tablet containing tezacaftor 100 mg/ivacaftor 150 mg in the morning and 1 tablet containing ivacaftor 150 mg in the evening about 12 hours later.
All participants discontinued any previous CFTR modulators but continued to take their other prescribed CF therapies.
Study 2
Study 2
Study 2
Results
Results
Results
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
The study results of TRIKAFTA are an average of all people studied and differed among individuals. Your experience may be different.
Significant Improvement in Lung Function (FEV1*)
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
*FEV1=forced expiratory volume, or how much air a person can exhale in a forced breath in 1 second.
Decrease in Sweat Chloride
Significant decrease of 45.1 mmol/L on average compared with SYMDEKO at 4 weeks.
On average, people taking TRIKAFTA started the study with a sweat chloride level of 91.4 mmol/L.
Sweat chloride is a measure of the amount of salt in a person's sweat.
Improvement in CF Respiratory Symptoms
People taking TRIKAFTA reported a significant 17.4-point average increase in CF respiratory symptom score compared with SYMDEKO at 4 weeks.
On average, people taking TRIKAFTA began the study with a score of 70.6 points.
Respiratory symptoms were measured using a tool called the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score.
The average increase in CFQ-R Respiratory Domain score means that, overall, the symptoms studied have improved. It does not mean there was an improvement in each symptom measured.
The side effects reported in Study 2 were similar to what was observed from Study 1. See the side effects reported in Study 1 here.
Keep Learning
About TRIKAFTA
About TRIKAFTA
About TRIKAFTA
Clayton, age 5
F508del/G542X
What is TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.
Talk to your doctor to learn if you have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under 2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, tell your doctor about all of your medical conditions, including if you:
- are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the Patient Information for a list of ingredients
- have kidney problems
- have or have had liver problems
- are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your doctor should decide if you will take TRIKAFTA while you are pregnant
- are breastfeeding or planning to breastfeed. It is not known if TRIKAFTA passes into your breast milk. You and your doctor should decide if you will take TRIKAFTA while you are breastfeeding
What is TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA.
Talk to your doctor to learn if you have an indicated CF gene mutation.
It is not known if TRIKAFTA is safe and effective in children under 2 years of age.
IMPORTANT SAFETY INFORMATION
Before taking TRIKAFTA, tell your doctor about all of your medical conditions, including if you:
- are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the Patient Information for a list of ingredients
- have kidney problems
- have or have had liver problems
- are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your doctor should decide if you will take TRIKAFTA while you are pregnant
- are breastfeeding or planning to breastfeed. It is not known if TRIKAFTA passes into your breast milk. You and your doctor should decide if you will take TRIKAFTA while you are breastfeeding
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.
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
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.
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.
You may report side effects to FDA at 1-800-FDA-1088.
For further information, please see full Prescribing Information, including Patient Information.