Skip to main content
 Intended for US residents only

What is Cholangiocarcinoma?

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

In cholangiocarcinoma, a gene in the cells lining the bile duct changes abnormally, causing the cells to grow and divide without stopping. These cancer cells form tumors. Over time, some cells can break off and spread to other parts of the body. This is called metastasis.

Image of the liver, stomach, gallbladder and bile duct
Image of the liver, stomach, gallbladder and bile duct

Cholangiocarcinoma is often described according to where it starts

Image of the liver, stomach, gallbladder and bile duct with the bile ducts inside the liver highlighted to show Intrahepatic cholangiocarcinoma, which starts in the bile ducts inside the liver

Intrahepatic cholangiocarcinoma

starts in the bile ducts inside the liver

Image of the liver, stomach, gallbladder, and bile duct with the bile ducts outside the liver highlighted to show Perihilar cholangiocarcinoma, which starts in the bile ducts just outside the liver

Perihilar cholangiocarcinoma

starts in the bile ducts just outside the liver

Image of the liver, stomach, gallbladder and bile duct with the bile ducts outside the liver highlighted to show Extrahepatic cholangiocarcinoma, which starts in the bile ducts outside the liver

Extrahepatic cholangiocarcinoma

(also called distal cholangiocarcinoma) starts in the bile ducts outside the liver close to the small intestine

The location of cholangiocarcinoma may affect how healthcare providers (HCPs) treat the disease.

Most people with cholangiocarcinoma do not have symptoms until the disease becomes more advanced. For this reason, it is often difficult for 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.

What Causes Cholangiocarcinoma?

As mentioned previously, cholangiocarcinoma is caused by an abnormal change to a gene that causes cells inside the bile ducts to grow and divide without stopping.

There are different types of abnormal gene changes or defects that can lead to cholangiocarcinoma. These changes may include:

Graphic showing gene fusion

Gene Fusion

This occurs when parts of two different
genes abnormally join together.

Graphic showing gene amplification

Gene Amplification

This occurs when the number of copies of a
gene is increased beyond the normal number.

Graphic showing gene mutation

Mutation

This is a change to a specific
portion of the normal gene.

Understanding Molecular Profiling in Cholangiocarcinoma

Watch Dr Milind Javle, a gastrointestinal oncologist, explain what molecular profiling, also known as biomarker testing, is and how this type of test 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 HCP.

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.

Find out more about cholangiocarcinoma and treatment recommendations from the National Comprehensive Cancer Network® (NCCN®)*

NCCN is a not-for-profit alliance of 32 leading cancer centers devoted to patient care, research, and education.

Visit the NCCN

Treatment with PEMAZYRE

Find out how PEMAZYRE works to target FGFR2 fusions in people with previously treated cholangiocarcinoma that has spread or cannot be removed surgically.

Learn more about PEMAZYRE 

* NCCN Guidelines for Patients® for Gallbladder and Bile Duct Cancers - Hepatobiliary Cancers, 2021. © 2022 National Comprehensive Cancer Network, All Rights Reserved. Accessed February 23, 2022. To view the most recent and complete version of the NCCN Guidelines for Patients, visit NCCN.org/patientguidelines.

Referenced with permission from the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) for Hepatobiliary Cancers V.2.2022. © National Comprehensive Cancer Network, Inc. 2022. All rights reserved. Accessed July 15, 2022. To view the most recent and complete version of the guideline, go online to NCCN.org. 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.

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
  • 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, or visit www.fda.gov/medwatch. 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.