Study design
Study design
78 children age 6-11 years participated in a study evaluating the safety and tolerability of ALYFTREK™.
Study design
The purpose of this study was to look at the safety and tolerability of ALYFTREK in children with cystic fibrosis (CF) age 6-11 years.
Children in this safety study had at least one mutation responsive to TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor). The study also evaluated changes in lung function and sweat chloride.
Everyone in the study took TRIKAFTA twice a day with fat-containing food for at least 4 weeks, and then they all started taking ALYFTREK once a day with fat-containing food. Each participant’s dose of ALYFTREK was based on their weight.
Because no one took placebo in the safety study, it is not known if changes seen in the study were due to ALYFTREK.
Keep in mind that all results shown are an average of all people studied and differed among individuals and mutations. Your loved one may have a different experience.
Safety
Through 24 weeks of treatment:
Keep in mind that all results shown are an average of all people studied and differed among individuals and mutations. Your loved one may have a different experience.
Lung function
Through 24 weeks, there was no change in lung function after participants switched from TRIKAFTA to ALYFTREK.
At least 4 weeks on TRIKAFTA
Baseline lung function (FEV1) was measured after everyone took TRIKAFTA for at least 4 weeks.
Then all participants were switched to ALYFTREK, and their lung function was measured at 24 weeks (0.0% change).
24 weeks
on ALYFTREK
This study was not designed to determine whether these results were because of ALYFTREK. These results are not included in the full Prescribing Information for ALYFTREK.
Because no one took placebo in the safety study, it is not known if changes seen in the study were due to ALYFTREK.
Keep in mind that all results shown are an average of all people studied and differed among individuals and mutations. Your loved one may have a different experience.
Sweat chloride
The clinical importance of these differences in sweat chloride has not been shown in clinical trials.
Because no one took placebo in the safety study, it is not known if changes seen in the study were due to ALYFTREK.
Keep in mind that all results shown are an average of all people studied and differed among individuals and mutations. Your loved one may have a different experience.
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.
