This site is intended for US residents only. Indications, Important Safety Information, full Prescribing Information, and Patient Information

Go to Homepage

Vertex offers several cystic fibrosis (CF) treatment options for individuals of certain ages with specific CF gene mutations. While not everyone with CF is eligible for a Vertex treatment option, we are fully committed to continuing our work in support of the CF community.

Go back to home page

Indications, Important Safety Information, full Prescribing Information, and Patient Information

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

Tell your doctor 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 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 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®)

What should I avoid while taking TRIKAFTA?

  • Avoid food or drink that contains grapefruit while you are taking TRIKAFTA

What are the possible side effects of TRIKAFTA?

TRIKAFTA can cause serious side effects, including:

  • Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease
  • High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. Your doctor will do blood tests to check your liver:
    • before you start TRIKAFTA
    • every 3 months during your first year of taking TRIKAFTA
    • every year while you are taking TRIKAFTA

    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 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), which has happened in some children and adolescents treated with TRIKAFTA. If you are a child or adolescent, your doctor 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

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 TRIKAFTA. For more information, ask your doctor 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 Patient Information.

 

TRIKAFTA is manufactured for Vertex Pharmaceuticals Incorporated.
TRIKAFTA and the TRIKAFTA logo are registered trademarks of Vertex Pharmaceuticals Incorporated.

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) 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.

 

SYMDEKO is manufactured for Vertex Pharmaceuticals Incorporated.
SYMDEKO and the SYMDEKO logo are registered trademarks of Vertex Pharmaceuticals Incorporated.

What is ORKAMBI® (lumacaftor/ivacaftor)?

ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 1 year and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. 

ORKAMBI should not be used in patients other than those who have two copies of the F508del mutation in their CFTR gene.

It is not known if ORKAMBI is safe and effective in children under 1 year of age.

 

IMPORTANT SAFETY INFORMATION

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

  • have or have had liver problems
  • are allergic to ORKAMBI or any ingredients in ORKAMBI. See the Patient Information for a list of ingredients
  • have kidney problems
  • have lung problems
  • have had an organ transplant
  • are using birth control (hormonal contraceptives, including oral, injectable, transdermal, or implantable forms). Hormonal contraceptives should not be used as a method of birth control when taking ORKAMBI. Talk to your doctor about the best birth control method you should use while taking ORKAMBI
  • are pregnant or plan to become pregnant. It is not known if ORKAMBI will harm your unborn baby. You and your doctor should decide if you will take ORKAMBI while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if ORKAMBI passes into your breast milk. You and your doctor should decide if you will take ORKAMBI while you are breastfeeding

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

ORKAMBI may affect the way other medicines work, and other medicines may affect how ORKAMBI works. The dose of ORKAMBI 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: rifampin (RIFAMATE®, RIFATER®) or rifabutin (MYCOBUTIN®)
  • seizure medicines: phenobarbital, carbamazepine (TEGRETOL®, CARBATROL®, and EQUETRO®), or phenytoin (DILANTIN®, PHENYTEK®)
  • sedatives and anti-anxiety medicines: triazolam (HALCION®) or midazolam (DORMICUM®, HYPNOVEL®, and VERSED®)
  • immunosuppressant medicines: cyclosporine, everolimus (ZORTRESS®), sirolimus (RAPAMUNE®), or tacrolimus (ASTAGRAF XL®, ENVARSUS XR®, PROGRAF®, and PROTOPIC®)
  • St. John’s wort
  • antifungal medicines including ketoconazole, itraconazole (such as SPORANOX®), posaconazole (such as NOXAFIL®), or voriconazole (such as VFEND®)
  • antibiotics including telithromycin, clarithromycin (such as BIAXIN®), or erythromycin (such as ERY-TAB®)

What should I avoid while taking ORKAMBI?

  • Do not eat or drink grapefruit products during your first week of treatment with ORKAMBI. Eating or drinking grapefruit products can increase the amount of ORKAMBI in your blood

What are the possible side effects of ORKAMBI?

ORKAMBI can cause serious side effects, including:

  • Worsening of liver function in people with severe liver disease. The worsening of liver function can be serious or cause death. Talk to your doctor if you have been told you have liver disease as your doctor may need to adjust the dose of ORKAMBI
  • High liver enzymes in the blood, which can be a sign of liver injury in people receiving ORKAMBI. Your doctor will do blood tests to check your liver:
    • before you start ORKAMBI
    • every 3 months during your first year of taking ORKAMBI
    • every year while you are taking ORKAMBI
    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
    • confusion
  • Serious allergic reactions have happened to people who are treated with ORKAMBI. 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
    • swelling of the face, lips, and/or tongue, or difficulty swallowing
    • light-headedness or dizziness
  • Breathing problems such as trouble breathing, shortness of breath or chest tightness in patients when starting ORKAMBI, especially in patients who have poor lung function. Call your doctor right away if you experience these symptoms
  • An increase in blood pressure in some people receiving ORKAMBI. Call your doctor right away if you have an increase in blood pressure
  • Abnormality of the eye lens (cataract) in some children and adolescents receiving ORKAMBI. If you are a child or adolescent, your doctor should perform eye examinations before and during treatment with ORKAMBI to look for cataracts

The most common side effects of ORKAMBI include:

  • breathing problems such as shortness of breath and chest tightness
  • nausea
  • diarrhea
  • fatigue
  • increase in a certain blood enzyme called creatine phosphokinase
  • rash
  • gas
  • common cold, including sore throat, stuffy or runny nose
  • flu or flu-like symptoms
  • irregular, missed, or abnormal periods (menses) and increase in the amount of menstrual bleeding

Side effects seen in children are similar to those seen in adults and adolescents. Additional common side effects seen in children include:

  • cough with sputum
  • stuffy nose
  • headache
  • stomach pain
  • increase in sputum

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 ORKAMBI. 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.

 

ORKAMBI is manufactured for Vertex Pharmaceuticals Incorporated.
ORKAMBI and the ORKAMBI logo are registered trademarks of Vertex Pharmaceuticals Incorporated.

What is KALYDECO® (ivacaftor)?

KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in people aged 1 month and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to KALYDECO.

Talk to your doctor to learn if you have an indicated CF gene mutation.

It is not known if KALYDECO is safe and effective in children under 1 month of age.

IMPORTANT SAFETY INFORMATION

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

  • have liver or kidney problems
  • are allergic to KALYDECO or any ingredients in KALYDECO. See the Patient Information for a list of ingredients
  • are pregnant or plan to become pregnant. It is not known if KALYDECO will harm your unborn baby. You and your doctor should decide if you will take KALYDECO while you are pregnant
  • are breastfeeding or planning to breastfeed. It is not known if KALYDECO passes into your breast milk. You and your doctor should decide if you will take KALYDECO while you are breastfeeding

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

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Ask your doctor or pharmacist for a list of these medicines if you are not sure.

Especially tell your doctor if you take:

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

What should I avoid while taking KALYDECO?

  • KALYDECO 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 containing grapefruit while you are taking KALYDECO

What are the possible side effects of KALYDECO?

KALYDECO can cause serious side effects, including:

  • High liver enzymes in the blood, which have happened in people receiving KALYDECO. Your doctor will do blood tests to check your liver:
    • before you start KALYDECO
    • every 3 months during your first year of taking KALYDECO
    • every year while you are taking KALYDECO

    For people who have had high liver enzymes in the past, your doctor may do blood tests to check the liver more often.

    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 KALYDECO. Call your healthcare provider or go to the emergency room right away if you have 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), which has happened in some children and adolescents receiving KALYDECO. Your doctor should perform eye examinations before and during treatment with KALYDECO to look for cataracts.

The most common side effects of KALYDECO include:

  • headache
  • upper respiratory tract infection (common cold), including:
    • sore throat
    • nasal or sinus congestion
    • runny nose
  • stomach (abdominal) pain
  • diarrhea
  • rash
  • nausea
  • 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 KALYDECO. For more information, ask your doctor or pharmacist.

Use of KALYDECO in children aged 1 month to less than 6 months born from a pregnancy lasting (gestational age) less than 37 weeks has not been evaluated.

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.

 

KALYDECO is manufactured for Vertex Pharmaceuticals Incorporated.
KALYDECO and the KALYDECO logo are registered trademarks of Vertex Pharmaceuticals Incorporated.