Niktimvo™ (axatilimab-csfr) works differently to treat chronic GVHD

How does Niktimvo work?

Niktimvo works differently to block the CSF-1 receptor, helping reduce inflammation and fibrosis (scarring of tissue) in chronic graft-versus-host disease (GVHD).

What makes Niktimvo different?

Other treatments you may have tried target different immune cells, such as T cells and/or B cells.

Niktimvo is the FIRST AND ONLY treatment to block the CSF-1 receptor in chronic GVHD.

Without Niktimvo

animation of proteins bouncing off a CSF-1 receptor that has been blocked by Niktimvo, preventing entrance into an immune cell, resulting in reduced inflammation and fibrosis
Increased immune cell activity leads to inflammation and fibrosis

With Niktimvo

animation of proteins bouncing off a CSF-1 receptor that has been blocked by Niktimvo, preventing entrance into an immune cell, resulting in reduced inflammation and fibrosis
Niktimvo blocks signaling proteins, potentially addressing both inflammation and fibrosis

This is how Niktimvo was shown to work in lab studies.

The CSF-1 receptor is located on the surface of certain immune cells. The receptor and the immune cells play an important role in the development of inflammation and fibrosis in chronic GVHD.

Why does the CSF-1 receptor matter?

When a signaling protein binds to the CSF-1 receptor, it sends signals that increase the activity of immune cells, leading to inflammation and fibrosis—two key contributors to chronic GVHD symptoms.

Talk to your doctor about a different treatment approach and see if Niktimvo may be right for you

What are the possible side effects of Niktimvo?

Niktimvo may cause serious side effects, including:

Infusion-related reactions. Infusion-related reactions are common with Niktimvo and can be serious. Your healthcare provider will monitor you for infusion-related reactions during your treatment. If you have a reaction, your healthcare provider may temporarily or completely stop your treatment with Niktimvo. Tell your healthcare provider right away if you have fever, chills, rash, flushing, shortness of breath, trouble breathing, nausea, vomiting, or symptoms of high blood pressure such as chest pain, headaches, or blurred vision during an infusion of Niktimvo.

The most common side effects of Niktimvo include:

  • Infections
  • Increased blood level of liver enzymes
  • Decreased blood level of phosphate
  • Low red blood cell count (anemia)
  • Muscle, bone, or joint pain
  • Increased blood level of pancreatic enzymes
  • Low energy
  • Increased blood level of calcium
  • Increased blood level of a muscle enzyme
  • Increased blood level of a bone enzyme
  • Nausea
  • Headache
  • Diarrhea
  • Cough
  • Fever
  • Shortness of breath

These are not all the possible side effects of Niktimvo. Call your doctor for medical advice about side effects.

Inflammation: Part of the body’s immune response to fight off infection or heal damaged tissue. Chronic inflammation is an abnormal immune response that can damage cells and organs over time.

Fibrosis: The growth of scar tissue in the body.

CSF-1=colony stimulating factor-1.

Discover how Niktimvo may help

Explore Study Results

Explore possible side effects

Learn About Safety

Indications and Usage

NiktimvoTM (axatilimab-csfr) is a prescription medicine used to treat adults and children who weigh at least 88.2 pounds (40 kg) with chronic graft-versus-host disease (cGVHD) after receiving at least 2 prior treatments (systemic therapy) and they did not work.

It is not known if Niktimvo is safe and effective in adults and children weighing less than 88.2 pounds (40 kg).

IMPORTANT SAFETY INFORMATION

What are the possible side effects of Niktimvo?

Niktimvo may cause serious side effects, including:

Infusion-related reactions. Infusion-related reactions are common with Niktimvo and can be serious. Your healthcare provider will monitor you for infusion-related reactions during your treatment. If you have a reaction, your healthcare provider may temporarily or completely stop your treatment with Niktimvo. Tell your healthcare provider right away if you have fever, chills, rash, flushing, shortness of breath, trouble breathing, nausea, vomiting, or symptoms of high blood pressure such as chest pain, headaches, or blurred vision during an infusion of Niktimvo.

The most common side effects of Niktimvo include:

  • infections
  • increased blood level of liver enzymes
  • decreased blood level of phosphate
  • low red blood cell count (anemia)
  • muscle, bone, or joint pain
  • increased blood level of pancreatic enzymes
  • low energy
  • increased blood level of calcium
  • increased blood level of a muscle enzyme
  • increased blood level of a bone enzyme
  • nausea
  • headache
  • diarrhea
  • cough
  • fever
  • shortness of breath

These are not all the possible side effects of Niktimvo. Call your doctor for medical advice about side effects.

Before receiving Niktimvo, tell your healthcare provider about all of your medical conditions, including if you:

  • have or have had liver problems.
  • are pregnant or plan to become pregnant. Niktimvo may harm your unborn baby.

    Females who are able to become pregnant:

    • Your healthcare provider should do a pregnancy test before you start treatment with Niktimvo.
    • You should use an effective method of birth control during your treatment and for 30 days after your last dose of Niktimvo. Talk to your healthcare provider about birth control methods that you can use during this time.
    • Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Niktimvo.
  • are breastfeeding or plan to breastfeed. It is not known if Niktimvo passes into your breast milk. Do not breastfeed during treatment and for 30 days after your last dose of Niktimvo.

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

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

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.

You may also report side effects to Incyte Medical Information at 1-855-463-3463.

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Indications and Usage

NiktimvoTM (axatilimab-csfr) is a prescription medicine used to treat adults and children who weigh at least 88.2 pounds (40 kg) with chronic graft-versus-host disease (cGVHD) after receiving at least 2 prior treatments (systemic therapy) and they did not work.

It is not known if Niktimvo is safe and effective in adults and children weighing less than 88.2 pounds (40 kg).

IMPORTANT SAFETY INFORMATION

What are the possible side effects of Niktimvo?

Niktimvo may cause serious side effects, including:

Infusion-related reactions. Infusion-related reactions are common with Niktimvo and can be serious. Your healthcare provider will monitor you for infusion-related reactions during your treatment. If you have a reaction, your healthcare provider may temporarily or completely stop your treatment with Niktimvo. Tell your healthcare provider right away if you have fever, chills, rash, flushing, shortness of breath, trouble breathing, nausea, vomiting, or symptoms of high blood pressure such as chest pain, headaches, or blurred vision during an infusion of Niktimvo.

The most common side effects of Niktimvo include:

  • infections
  • increased blood level of liver enzymes
  • decreased blood level of phosphate
  • low red blood cell count (anemia)
  • muscle, bone, or joint pain
  • increased blood level of pancreatic enzymes
  • low energy
  • increased blood level of calcium
  • increased blood level of a muscle enzyme
  • increased blood level of a bone enzyme
  • nausea
  • headache
  • diarrhea
  • cough
  • fever
  • shortness of breath

These are not all the possible side effects of Niktimvo. Call your doctor for medical advice about side effects.

Before receiving Niktimvo, tell your healthcare provider about all of your medical conditions, including if you:

  • have or have had liver problems.
  • are pregnant or plan to become pregnant. Niktimvo may harm your unborn baby.

    Females who are able to become pregnant:

    • Your healthcare provider should do a pregnancy test before you start treatment with Niktimvo.
    • You should use an effective method of birth control during your treatment and for 30 days after your last dose of Niktimvo. Talk to your healthcare provider about birth control methods that you can use during this time.
    • Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Niktimvo.
  • are breastfeeding or plan to breastfeed. It is not known if Niktimvo passes into your breast milk. Do not breastfeed during treatment and for 30 days after your last dose of Niktimvo.

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

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

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.

You may also report side effects to Incyte Medical Information at 1-855-463-3463.

Niktimvo and the Niktimvo logo are trademarks of Incyte.

Incyte and the Incyte logo are registered trademarks of Incyte.

Niktimvo (axatilimab) is licensed to Incyte from Syndax.

© 2025, Incyte. MAT-AXA-00050  09/25