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PEMAZYRE is the first FDA-approved treatment option for adults with 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

PEMAZYRE is not chemotherapy; it is a targeted-treatment for cholangiocarcinoma.

How do healthcare professionals know if a person has an abnormal FGFR2 gene?

After a person is diagnosed with cholangiocarcinoma, their healthcare team may order a type of genomic test called molecular profiling.

  • In this test, healthcare professionals examine a sample of the tumor to identify unique gene changes or defects that could drive tumor growth.
  • People may be eligible for PEMAZYRE if molecular profiling identifies a specific abnormal gene change in his or her tumor called an FGFR2 fusion.

Click here to learn more about FGFR2 fusions in cholangiocarcinoma.

How does PEMAZYRE work?

  • An FGFR2 fusion causes your bile duct cells to make an abnormal version of the FGFR2 protein.
  • This abnormal protein causes these cells to grow and divide without stopping, leading to tumor growth.

PEMAZYRE helps stop the activity of the FGFR2 protein

  • By doing this, PEMAZYRE may help reduce the size of the targeted tumor(s) by a certain amount or cause it to disappear* in people with cholangiocarcinoma.


One specific type of molecular profiling test is known as next generation sequencing. This type of test can identify a wide range of abnormal changes in cholangiocarcinoma, including FGFR2 fusions.

FoundationOne® CDx is one example of next generation sequencing and is the FDA-approved companion diagnostic for PEMAZYRE.


Watch Dr Milind Javle, a gastrointestinal oncologist, discuss treatment with PEMAZYRE, including how it works, how it was studied, and important safety considerations.

This video is intended for informational use only, and is not designed to replace the medical advice of your Healthcare Professional.

How has PEMAZYRE been studied?

PEMAZYRE has been studied in a clinical trial involving 107 people with cholangiocarcinoma who:

  • Tested positive for an FGFR2 gene fusion or rearrangement
  • Had disease that was metastatic or could not be removed surgically
  • Had progressed following at least one previous treatment

The main point of the study was to find how many people saw their targeted tumor(s) shrink in size by a certain amount or disappear* while taking PEMAZYRE, as well as how long the response lasted (duration of response).

*This does not always mean the cancer has been cured.

What were the results of this trial?

Graphic of a circle with text – 36% (38 of 107) of people in this clinical trial responded to treatment; and a small circle with 33% and another small circle with 3%

33% (35 of 107) experienced a partial response, meaning the size of their targeted tumors shrunk by a certain amount following treatment.

3% (3 of 107) experienced a complete response, meaning their targeted tumors disappeared* following treatment.

For patients who had a complete or partial response to treatment, this response lasted for a median of 9.1 months: This means that the response to treatment lasted 9.1 months or longer for 50% of patients, while the other 50% had a response that lasted shorter than 9.1 months.

  • 24 of the 38 patients who responded to treatment (63%) had a response that lasted for 6 months or longer
  • 7 of the 38 patients who responded to treatment (18%) had a response that lasted for 12 months or longer

*This does not always mean the cancer has been cured.

Image of a doctor and a patient looking at a clipboardImage of a doctor and a patient looking at a clipboard
Your results with PEMAZYRE may vary.

Talk to your healthcare team if you have any questions.

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 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, or call 1-800-FDA-1088.

Talking with your Healthcare Team

It’s important to tell your healthcare team about any side effects you may experience, even if you are not sure they are related to your treatment.

It’s important to frequently talk to your healthcare team about how you are feeling, and how your cholangiocarcinoma and treatment are affecting you. Be sure to tell your healthcare team about any new or worsening symptoms you may have, even if you are not sure if they are related to your disease or its treatment.

Learn more about taking PEMAZYRE.

Learn more about how to take PEMAZYRE, and what you should tell your healthcare team before starting treatment with PEMAZYRE.

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