Study design
Study design
971 people age 12 years and older participated across 2 studies comparing ALYFTREK™ to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor).
See Indication and Important Safety Information for TRIKAFTA.
Study design
The purpose of these studies was to determine if the effect of ALYFTREK on lung function was similar to (not worse than) TRIKAFTA.
Everyone in the study took TRIKAFTA twice a day with fat-containing food for 4 weeks. Then 480 people started taking ALYFTREK once a day with fat-containing food.
People who had previously stopped or interrupted treatment due to side effects with medicines similar to ALYFTREK or its components were not included in this study.
People who had previously stopped or interrupted treatment due to side effects with medicines similar to ALYFTREK or its components were not included in this study.
Lung function
Lung function was measured by the change in FEV1 from baseline. People who started taking ALYFTREK saw a change of 0.2% on average in both studies compared with people who continued taking TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor).
The study results of ALYFTREK represent the group of people studied and results differed among individuals. Your experience may be different.
Sweat chloride
Sweat chloride is a measure of how much salt is in a person’s sweat. Sweat chloride was measured in the ALYFTREK studies because sweat chloride levels may indicate how the CFTR protein is functioning.
Compared with people taking TRIKAFTA, people taking ALYFTREK saw a reduction in sweat chloride of 8.4 mmol/L on average in one study, and 2.8 mmol/L on average in the other study.
The clinical importance of these differences in sweat chloride has not been shown in clinical trials.
The study results of ALYFTREK are an average of all people studied and differed among individuals. Your experience may be different.
Additional results
The changes in pulmonary exacerbations and respiratory symptoms seen with ALYFTREK were similar to the changes seen with TRIKAFTA.
Pulmonary exacerbations
Pulmonary exacerbations are changes in certain symptoms that require treatment with oral, intravenous (IV), or inhaled antibiotics.
In one study, there were 67 pulmonary exacerbations in the ALYFTREK group and 90 in the TRIKAFTA group through week 52. In the other study, there were 86 pulmonary exacerbations in the ALYFTREK group and 79 in the TRIKAFTA group.
CF respiratory symptoms
Respiratory symptoms, such as coughing, mucus, and trouble breathing, were measured using a tool called the Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain score. An average increase in this score means that, overall, the CF respiratory symptoms studied have improved. It does not mean there was an improvement in each symptom measured.
In one study, people taking ALYFTREK saw a 2.3-point average increase in CFQ-R Respiratory Domain score from baseline through week 24 compared with people taking TRIKAFTA. In the other study, people taking ALYFTREK saw a 0.1-point average decrease compared with people taking TRIKAFTA.
The studies were not designed to demonstrate if these additional results were caused by ALYFTREK, or if there was a difference between the ALYFTREK group and the TRIKAFTA group, or if ALYFTREK was similar to (no worse than) TRIKAFTA.
The results are an average of all people studied and differed among individuals and mutations. Your experience may be different.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about ALYFTREK?
Elevated liver enzymes have been observed in patients taking ALYFTREK. Cases of serious liver damage and liver failure leading to transplantation and death have been seen in some people with or without a history of liver problems taking elexacaftor/tezacaftor/ivacaftor (TRIKAFTA®), a medicine which has the same or similar active ingredients as ALYFTREK.
- Your healthcare provider will do blood tests to check your liver:
- before you start ALYFTREK
- every month during your first 6 months of taking ALYFTREK
- then every 3 months during the next 12 months of taking ALYFTREK
- then at least every year while you are taking ALYFTREK
What is ALYFTREK™ (vanzacaftor/tezacaftor/deutivacaftor)?
ALYFTREK is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Talk to your healthcare provider to learn if you have an indicated CF gene mutation.
It is not known if ALYFTREK is safe and effective in children under 6 years of age.
- Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury
- Stop taking ALYFTREK and call your healthcare provider right away if you have any of the following symptoms of liver problems:
- pain, swelling, or discomfort in the upper right stomach (abdominal) area
- yellowing of your skin or the white part of your eyes
- mental changes
- nausea or vomiting
- dark, amber-colored urine
- loss of appetite
- fluid in your stomach area (ascites)
What should I tell my healthcare provider before taking ALYFTREK?
Before taking ALYFTREK, tell your healthcare provider about all of your medical conditions, including if you:
- have or have had liver problems
- are allergic to ALYFTREK or any ingredients in ALYFTREK. See the Medication Guide for a complete list of ingredients in ALYFTREK
- have taken another medicine with elexacaftor, tezacaftor, or ivacaftor before and temporarily or permanently stopped because of side effects. Your healthcare provider may want to see you more often
- have kidney problems
- are pregnant or plan to become pregnant. It is not known if ALYFTREK will harm your unborn baby. You and your healthcare provider should decide if you will take ALYFTREK while you are pregnant
- are breastfeeding or planning to breastfeed. It is not known if ALYFTREK passes into your breast milk. You and your healthcare provider should decide if you will take ALYFTREK while you are breastfeeding
Tell your healthcare provider about all the medicines you take, including prescription and over‑the‑counter medicines, vitamins, and herbal supplements.
ALYFTREK may affect the way other medicines work, and other medicines may affect how ALYFTREK works. The dose of ALYFTREK may need to be adjusted when taken with certain medicines. Ask your healthcare provider or pharmacist for a list of these medicines if you are not sure.
Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
What should I avoid while taking ALYFTREK?
- Avoid food or drink that contains grapefruit while you are taking ALYFTREK
What are the possible or reasonably likely side effects of ALYFTREK?
ALYFTREK can cause serious side effects, including:
- See “What is the most important information I should know about ALYFTREK?”
- Serious Allergic Reactions can happen to people who are treated with ALYFTREK. Call your healthcare provider 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 ALYFTREK. If you are a child or adolescent, your healthcare provider should perform eye examinations before and during treatment with ALYFTREK to look for cataracts
The most common side effects of ALYFTREK include:
- cough
- pain or swelling of your nose or throat (nasopharyngitis)
- upper respiratory tract infection (common cold) including stuffy and runny nose
- headache
- mouth or throat pain
- flu (influenza)
- tiredness
- increase in liver enzymes
- rash
- sinus congestion
Your healthcare provider should monitor you during treatment with ALYFTREK. You may require additional monitoring if your treatment with a medicine that works like ALYFTREK has been previously stopped or interrupted because of side effects.
Tell your healthcare provider if you have any side effect that bothers you or that does not go away.
These are not all of the possible side effects of ALYFTREK. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see full Prescribing Information, including Medication Guide with Important Warning.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about TRIKAFTA?
TRIKAFTA can cause serious liver damage and liver failure. Liver failure leading to transplantation and death has been seen in some people with or without a history of liver problems taking TRIKAFTA.
Your healthcare provider will do blood tests to check your liver:
- before you start TRIKAFTA
- then every month during your first 6 months of taking TRIKAFTA
- then every 3 months during the next 12 months of taking TRIKAFTA
- then at least every year while you are taking TRIKAFTA
What is TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in people 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 healthcare provider 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.
Your healthcare provider may do blood tests to check the liver more often if you have had high liver enzymes in your blood in the past or are experiencing signs or symptoms of liver injury.
Stop taking TRIKAFTA and call your healthcare provider right away if you have any of the following symptoms of liver problems:
- pain, swelling, or discomfort in the upper right stomach (abdominal) area
- yellowing of your skin or the white part of your eyes
- mental changes
- nausea or vomiting
- dark, amber-colored urine
- loss of appetite
- fluid in your stomach area (ascites)
What should I tell my healthcare provider before taking TRIKAFTA?
Before taking TRIKAFTA, tell your healthcare provider about all of your medical conditions, including if you:
- have or have had liver problems
- are allergic to TRIKAFTA or any ingredients in TRIKAFTA. See the end of the Medication Guide for a complete list of ingredients in TRIKAFTA
- have kidney problems
- are pregnant or plan to become pregnant. It is not known if TRIKAFTA will harm your unborn baby. You and your healthcare provider 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 healthcare provider should decide if you will take TRIKAFTA while you are breastfeeding
Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.
TRIKAFTA may affect the way other medicines work and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Ask your healthcare provider or pharmacist for a list of these medicines if you are not sure.
Especially tell your healthcare provider 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 including ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), voriconazole (such as VFEND®), or fluconazole (such as DIFLUCAN®)
- antibiotics including telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)
Know the medicines you take. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.
What should I avoid while taking TRIKAFTA?
- Avoid food or drink that contains grapefruit while you are taking TRIKAFTA
What are the possible or reasonably likely side effects of TRIKAFTA?
TRIKAFTA can cause serious side effects, including:
- See “What is the most important information I should know about TRIKAFTA?”
- Serious Allergic Reactions can happen to people who are treated with TRIKAFTA. Call your healthcare provider 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
- swelling of the face, lips, and/or tongue, or difficulty swallowing
- light-headedness or dizziness
- Abnormality of the eye lens (cataract) has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your healthcare provider should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts
The most common side effects of TRIKAFTA include:
- headache
- upper respiratory tract infection (common cold) including stuffy and runny nose
- stomach (abdominal) pain
- diarrhea
- rash
- increase in liver enzymes
- increase in a certain blood enzyme called creatine phosphokinase
- flu (influenza)
- inflamed sinuses
- increase in blood bilirubin
- constipation
Tell your healthcare provider if you have any side effect that bothers you or that does not go away.
These are not all the possible side effects of TRIKAFTA. For more information, ask your healthcare provider or pharmacist. 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 Medication Guide with Important Warning.
