Pronunciation: nik tim voe
Generic name: axatilimab-csfr
Dosage form: injection (9 mg/0.18 mL, 22 mg/0.44 mL, 50 mg/mL)
Drug class: Miscellaneous antineoplastics

Medically reviewed by Melisa Puckey, BPharm. Last updated on Mar 6, 2025.

What is Niktimvo?

Niktimvo (axatilimab-csfr) is a prescription medicine used to treat chronic graft-versus-host disease (cGVHD) if you have received at least 2 prior treatments and they have not worked.  Niktimvo works by targeting the drivers of inflammation and fibrosis that are seen in chronic GVHD, it is the first approved anti-CSF-1R antibody. It can be used in patients who weigh at least 88.2 pounds (40 kg).

Chronic GVHD is a serious condition that can occur after a transfer of stem cells from a donor (allogeneic stem cell transplant) when there is an immune response against the donated cells, which attacks the transplant recipient’s organs.

Niktimvo FDA approval was received on August 14, 2024, for the Incyte Corporation for the indication:

• of chronic graft-versus-host disease (GVHD)
• after failure of at least two prior lines of systemic therapy
• in adult and pediatric patients weighing at least 40 kg (88.2 lbs). 

Approval was based on positive results from the Niktimvo clinical trial AGAVE-20: 

• 75% of patients achieved an overall response rate (ORR) within the first six months of treatment,
• Median time to response of 1.5 months
• 60% of patients maintained a response at 12 months when measured from the first response to new systemic therapy or death.  

How does Niktimvo work? 

Niktimvo is a monoclonal antibody that works by binding and blocking a receptor called colony-stimulating factor-1 receptors (CSF-1R) on cells in the immune system (monocytes and macrophages). By blocking CSF-1R, Niktimvo reduces the levels of monocytes and monocyte-derived macrophages, which are proinflammatory and profibrotic, and inhibits the activity of pathogenic macrophages in tissues. 

Niktimvo is from a class of medicines called colony-stimulating factor-1 receptor (CSF-1R)-blocking antibodies.

Niktimvo side effects 

Common Niktimvo side effects

The most common Niktimvo side effects included:

• Muscle and joint pain (35%)
• Tiredness, fatigue (32%)
• Infection, pathogen unspecified (57%)
• Nausea (23%)
• Headache (20%)
• Diarrhea (18%)
• Cough (18%)
• Bacterial infection (43%)
• Viral infection (15%) 
• Fever (15%)
• Difficulty breathing (15%).

Laboratory abnormality side effects were increased aspartate aminotransferase (AST), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, increased gamma glutamyl transferase (GGT), increased lipase increased amylase, increased calcium, increased creatine phosphokinase (CPK), and increased alkaline phosphatase (ALP). 

The common side effects and changes in laboratory results occurred in 15% of the Niktimvo patients in the AGAVE-201 clinical trial (NCT04710576).

Clinically relevant Niktimvo side effects that occurred in 10% or fewer patients included:

• Eye disorders: Swelling around the eyes (periorbital edema)
• Skin and subcutaneous skin disorders: Itchy skin (pruritus)
• Vascular disorders: High blood pressure (hypertension)

Serious Niktimvo side effects

Niktimvo may cause serious side effects, including Infusion-related reactions. Infusion-related reactions are common with this medicine and can be serious. You will be monitored for infusion-related reactions during your treatment. If you have a reaction, your healthcare provider may temporarily or completely stop your treatment. Tell your healthcare provider right away if you get 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 your Niktimvo infusion.

This is not a complete list of side effects, and others may occur. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Related/similar drugs

Warnings

Infusion-Related Reactions: If you have an infusion-related reactions your treatment may be interrupted, slowed, or permanently discontinued depending on the severity of the reaction.

Embryo-Fetal Toxicity: This medicine may cause fetal harm. Females of reproductive potential should be advised of the potential risk to a fetus and to use effective contraception.

Before taking this medicine

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

• have or have had liver problems. 

Pregnancy

Tell your healthcare provider if you are pregnant or plan to become pregnant as 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. 

Breastfeeding

Tell your healthcare provider if you are breastfeeding or plan to breastfeed as 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 this medicine. 

How will I receive this medicine?

Niktimvo is given through an intravenous (IV) infusion into a vein over 30 minutes, every  2 weeks.

Your healthcare provider will decide how many treatments you will need. 

Before you receive each Niktimvo infusion, your healthcare provider may give you medicines called diphenhydramine (antihistamine) and acetaminophen (antipyretic) to help prevent infusion-related reactions. 

Your healthcare provider will do blood tests to check you for side effects. 

Niktimvo Dosing Information

The recommended Niktimvo dosage is 0.3 mg/kg (maximum 35 mg) every 2 weeks in adult and pediatric patients weighing 40 kg and above. 

Niktimvo is available as:

• 9 mg/0.18 mL solution in a single-dose vial
• 22 mg/0.44 mL solution in a single-dose vial
• 50 mg/mL solution in a single-dose vial

Interactions

Other drugs may interact with Niktimvo. Tell your healthcare provider about including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible drug interactions are listed here.

Niktimvo PI 

HCPs and patients often use the Niktimvo PI (Package Insert) for more detailed information about this medicine. The Package Insert (prescribing information) contains more comprehensive information on Indications and Usage, Dosage and Administration, Clinical Pharmacology, Clinical Studies, Drug Interaction, and more. Discuss any medical questions you have with your HCP (health care professional). This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

The Package Insert is sometimes called the Niktimvo Prescribing Information (PI) or FDA label.

Storage

• Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light.
• Do not freeze or shake. 

Ingredients

Active ingredient: axatilimab-csfr

Inactive ingredients: citric acid monohydrate, glycine, polysorbate 80, sodium citrate, sucrose, and Water for Injection. 

Niktimvo manufacturer

Niktimvo manufacturer Incyte Corporation, Wilmington, DE 19803

Niktimvo Biosimilars

Biosimilar and interchangeable products are biological products that are highly similar to and have no clinically meaningful differences from the reference product.

Reference products

These are biological products that have already been approved by the FDA, against which biosimilar products are compared. There is 1 for Niktimvo.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer