About bile duct cancer known as cholangiocarcinoma (CCA)
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About cholangiocarcinoma

Learn about cholangiocarcinoma (CCA), including its subtypes, and one of the causes—an abnormal gene change called an FGFR2 fusion

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What is cholangiocarcinoma?

CCA is a rare and aggressive type of cancer that forms in bile ducts. It is also called bile duct cancer. Bile ducts are thin tubes that carry a digestive fluid called bile from your liver to other digestive organs.

Most people with CCA do not have symptoms until the disease becomes more advanced. For this reason, it is often difficult for healthcare providers (HCPs) to diagnose the disease early.

While surgery may be used to remove the tumor in some cases, this may not be an option if the tumor takes up too much of the liver, or if the disease has spread to other parts of the body.

There are different kinds of CCA, based on where it is located.

A graphic represening intraheptic cholangiocarcinoma. A graphic represening intraheptic cholangiocarcinoma.

Intrahepatic (meaning inside the liver) cholangiocarcinoma starts in bile ducts within the liver

The location of cholangiocarcinoma may affect how HCPs treat the disease.

A graphic representing extraheptic cholangiocarcinoma. A graphic representing extraheptic cholangiocarcinoma.

Extrahepatic (meaning outside the liver) cholangiocarcinoma starts in the bile ducts closer to the intestine

The location of cholangiocarcinoma may affect how HCPs treat the disease.

Causes of CCA

Cholangiocarcinoma occurs when the cells lining the bile ducts go through abnormal gene changes, which make the cells grow and divide without stopping. These cancer cells form tumors.

Over time, some cancer cells can break off and spread to other parts of the body. This is called metastasis.

There are different types of gene changes or defects that can lead to CCA, such as gene mutations, gene amplifications, or gene fusions.

Icon comparing a normal gene - all one color - to a gene that has undergone gene fusion and is represented by two colors. Icon comparing a normal gene - all one color - to a gene that has undergone gene fusion and is represented by two colors.

Gene fusions occur when parts of two different genes abnormally join together

FGFR2 fusions

One specific type of gene fusion that can cause CCA is called a fibroblast growth factor receptor 2 (FGFR2) fusion.

  • FGFR2 is a gene that makes the FGFR2 protein, which is involved in cell growth
  • An FGFR2 fusion changes the FGFR2 gene and causes the cells in your bile ducts to make an abnormal version of the FGFR2 protein
  • This abnormal FGFR2 protein can become overactive, causing these cells to grow and divide without stopping, which can lead to tumor growth in CCA

An FGFR2 fusion is one of the most common types of abnormal gene changes in people with iCCA.

Text reading "10% - 16% of patients with iCCA have an FGFR2 fusions." Text reading "10% - 16% of patients with iCCA have an FGFR2 fusions."

NGS testing for FGFR2 fusions

After you are diagnosed with CCA, your doctor may order a test called next-generation sequencing (NGS), which is a type of molecular profiling, or biomarker testing.

This type of test can help determine if you have an FGFR2 fusion and could help direct how your disease is managed.

  • In this test, HCPs examine a sample of the tumor to identify certain biomarkers, such as gene changes or defects that could drive tumor growth
  • People may be eligible for PEMAZYRE if NGS testing identifies an FGFR2 fusion

It’s a good idea to talk to your healthcare provider about biomarker testing as soon as possible after your diagnosis.

Understanding molecular profiling in cholangiocarcinoma

Watch Dr Milind Javle, a gastrointestinal oncologist, explain molecular profiling—also called biomarker testing—and how it can help guide the way cholangiocarcinoma, or bile duct cancer, is managed.

This video is intended for informational use only, and is not designed to replace the medical advice of your healthcare provider (HCP).

Watch the video
Watch the video, "Molecular Profiling in Cholangiocarcinoma".

What is the difference between “genetic” testing and “genomic” testing?

“Genetics” and “genomics” are two words that sound similar, but mean different things.

Genetics refers to the study of single genes and how they are involved in passing down traits from generation to generation. Genetic testing helps identify genetic conditions that may be inherited, like sickle cell anemia and cystic fibrosis.

Genomics is the study of how all of a person’s genes interact with each other and the environment. An FGFR2 fusion is an example of a genomic change that can cause cholangiocarcinoma.

Have you been prescribed PEMAZYRE?

Learn more about your dosing schedule, download resources to help you keep track of your treatment, and more.

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Keep learning

The National Comprehensive Cancer Network® (NCCN®) is a not-for-profit alliance of 33 leading cancer centers devoted to patient care, research, and education. You can find in-depth explanations of CCA diagnosis and treatment in the NCCN Guidelines for Patients®.*

View NCCN Guidelines for Patients

*Referenced with permission from the NCCN Guidelines for Patients® for Gallbladder and Bile Duct Cancers, 2023. © National Comprehensive Cancer Network, Inc. 2023. All rights reserved. Accessed July 22, 2024. To view the most recent and complete version of the NCCN Guidelines for Patients, go online to NCCN.org/patientguidelines. NCCN makes no warranties of any kind whatsoever regarding their content, use or application and disclaims any responsibility for their application or use in any way.

Have the conversation about PEMAZYRE today

Find out about PEMAZYRE—a therapy doctors have turned to for over 4 years to treat more than 1,500 patients with cholangiocarcinoma.†

†Commercially available in the US since 2020.

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See how PEMAZYRE works
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See how to take PEMAZYRE: one pill, once daily
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Find videos, tools, and more
 

INDICATIONS AND USAGE

PEMAZYRE is a prescription medicine that is used to treat adults with bile duct cancer (cholangiocarcinoma) that has spread or cannot be removed by surgery:

  • who have already received a previous treatment, and
  • whose tumor has a certain type of abnormal FGFR2 gene

Your healthcare provider will test your cancer for a certain type of abnormal FGFR2 gene and make sure that PEMAZYRE is right for you.

It is not known if PEMAZYRE is safe and effective in children.

PEMAZYRE is approved based on tumor response and duration of response. There are ongoing studies to show whether PEMAZYRE can improve survival or symptoms.

IMPORTANT SAFETY INFORMATION

Before you take PEMAZYRE (pemigatinib), tell your healthcare provider about all of your medical conditions, including if you:

  • have vision or eye problems
  • have kidney problems
  • have liver problems
  • are pregnant or plan to become pregnant. PEMAZYRE can harm your unborn baby or cause loss of your pregnancy (miscarriage). You should not become pregnant during treatment with PEMAZYRE.

Females who can become pregnant:

  • Your healthcare provider should do a pregnancy test before you start treatment with PEMAZYRE.
  • You should use an effective method of birth control during treatment and for 1 week after your last dose of PEMAZYRE. Talk to your healthcare provider about birth control methods that may be right for you.
  • Tell your healthcare provider right away if you become pregnant or think that you may be pregnant.

Males with female partners who can become pregnant:

  • You should use effective birth control when sexually active during treatment with PEMAZYRE and for 1 week after your last dose of PEMAZYRE.

 

  • are breastfeeding or plan to breastfeed. It is not known if PEMAZYRE passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose of PEMAZYRE.

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

What are the possible side effects of PEMAZYRE?

PEMAZYRE may cause serious side effects, including:

  • Eye problems. Certain eye problems are common with PEMAZYRE but can also be serious. Eye problems include dry eye or inflamed eyes, inflamed cornea (front part of the eye), increased tears, and a disorder of the retina (an internal part of the eye). You will need to see an eye specialist for a complete eye exam before you begin treatment with PEMAZYRE, every 2 months for the first 6 months, and then every 3 months during treatment with PEMAZYRE.
    • You should use artificial tears or substitutes, hydrating or lubricating eye gels as needed, to help prevent or treat dry eyes.
    • Tell your healthcare provider right away if you develop any changes in your vision during treatment with PEMAZYRE, including: blurred vision, flashes of light, or see black spots. You may need to see an eye specialist right away.
  • High phosphate levels in your blood (hyperphosphatemia) and buildup of minerals in different tissues in your body. Hyperphosphatemia is common with PEMAZYRE but can also be serious. High levels of phosphate in your blood may lead to buildup of minerals such as calcium, in different tissues in your body. Your healthcare provider will check your blood phosphate levels during treatment with PEMAZYRE.
    • Your healthcare provider may prescribe changes in your diet or phosphate lowering therapy, or change, interrupt or stop PEMAZYRE if needed.
    • Tell your healthcare provider right away if you develop any muscle cramps, or numbness or tingling around your mouth.

The most common side effects of PEMAZYRE for cholangiocarcinoma include:

  • hair loss
  • diarrhea
  • nails separate from the bed or poor formation of the nail
  • feeling tired
  • change in sense of taste
  • nausea
  • constipation
  • mouth sores
  • dry eyes
  • dry mouth
  • decrease in appetite
  • vomiting
  • joint pain
  • stomach-area (abdominal) pain
  • low phosphate in blood
  • back pain
  • dry skin

These are not all the possible side effects of PEMAZYRE. 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. You may also report side effects to Incyte Medical Information at 1-855-463-3463.

Keep PEMAZYRE and all medicines out of the reach of children.

General information about the safe and effective use of PEMAZYRE.

Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use PEMAZYRE for a condition for which it is not prescribed. Do not give PEMAZYRE to other people, even if they have the same symptoms you have. It may harm them. If you would like more information, talk with your healthcare provider. You can ask your pharmacist or healthcare provider for information that is written for healthcare professionals.

Please see the Full Prescribing Information, including Patient Information, which includes a more complete discussion of the risks associated with PEMAZYRE.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

 

INDICATIONS AND USAGE

PEMAZYRE is a prescription medicine that is used to treat adults with bile duct cancer (cholangiocarcinoma) that has spread or cannot be removed by surgery:

  • who have already received a previous treatment, and
  • whose tumor has a certain type of abnormal FGFR2 gene

Your healthcare provider will test your cancer for a certain type of abnormal FGFR2 gene and make sure that PEMAZYRE is right for you.

It is not known if PEMAZYRE is safe and effective in children.

PEMAZYRE is approved based on tumor response and duration of response. There are ongoing studies to show whether PEMAZYRE can improve survival or symptoms.

IMPORTANT SAFETY INFORMATION

Before you take PEMAZYRE (pemigatinib), tell your healthcare provider about all of your medical conditions, including if you:

  • have vision or eye problems
  • have kidney problems
  • have liver problems
  • are pregnant or plan to become pregnant. PEMAZYRE can harm your unborn baby or cause loss of your pregnancy (miscarriage). You should not become pregnant during treatment with PEMAZYRE.

Females who can become pregnant:

  • Your healthcare provider should do a pregnancy test before you start treatment with PEMAZYRE.
  • You should use an effective method of birth control during treatment and for 1 week after your last dose of PEMAZYRE. Talk to your healthcare provider about birth control methods that may be right for you.
  • Tell your healthcare provider right away if you become pregnant or think that you may be pregnant.

Males with female partners who can become pregnant:

  • You should use effective birth control when sexually active during treatment with PEMAZYRE and for 1 week after your last dose of PEMAZYRE.

 

  • are breastfeeding or plan to breastfeed. It is not known if PEMAZYRE passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose of PEMAZYRE.

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

What are the possible side effects of PEMAZYRE?

PEMAZYRE may cause serious side effects, including:

  • Eye problems. Certain eye problems are common with PEMAZYRE but can also be serious. Eye problems include dry eye or inflamed eyes, inflamed cornea (front part of the eye), increased tears, and a disorder of the retina (an internal part of the eye). You will need to see an eye specialist for a complete eye exam before you begin treatment with PEMAZYRE, every 2 months for the first 6 months, and then every 3 months during treatment with PEMAZYRE.
    • You should use artificial tears or substitutes, hydrating or lubricating eye gels as needed, to help prevent or treat dry eyes.
    • Tell your healthcare provider right away if you develop any changes in your vision during treatment with PEMAZYRE, including: blurred vision, flashes of light, or see black spots. You may need to see an eye specialist right away.
  • High phosphate levels in your blood (hyperphosphatemia) and buildup of minerals in different tissues in your body. Hyperphosphatemia is common with PEMAZYRE but can also be serious. High levels of phosphate in your blood may lead to buildup of minerals such as calcium, in different tissues in your body. Your healthcare provider will check your blood phosphate levels during treatment with PEMAZYRE.
    • Your healthcare provider may prescribe changes in your diet or phosphate lowering therapy, or change, interrupt or stop PEMAZYRE if needed
    • Tell your healthcare provider right away if you develop any muscle cramps, or numbness or tingling around your mouth.

The most common side effects of PEMAZYRE for cholangiocarcinoma include:

  • hair loss
  • diarrhea
  • nails separate from the bed or poor formation of the nail
  • feeling tired
  • change in sense of taste
  • nausea
  • constipation
  • mouth sores
  • dry eyes
  • dry mouth
  • decrease in appetite
  • vomiting
  • joint pain
  • stomach-area (abdominal) pain
  • low phosphate in blood
  • back pain
  • dry skin

These are not all the possible side effects of PEMAZYRE. 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. You may also report side effects to Incyte Medical Information at 1-855-463-3463.

Keep PEMAZYRE and all medicines out of the reach of children.

General information about the safe and effective use of PEMAZYRE.

Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use PEMAZYRE for a condition for which it is not prescribed. Do not give PEMAZYRE to other people, even if they have the same symptoms you have. It may harm them. If you would like more information, talk with your healthcare provider. You can ask your pharmacist or healthcare provider for information that is written for healthcare professionals.

Please see the Full Prescribing Information, including Patient Information, which includes a more complete discussion of the risks associated with PEMAZYRE.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

PEMAZYRE and the PEMAZYRE logo are registered trademarks of Incyte.

Incyte and the Incyte logo are registered trademarks of Incyte.

All other trademarks are the property of their respective owners.

© 2024, Incyte. MAT-PEM-00554  11/24