Niktimvo

Pronunciation: nik tim voe
Generic name: axatilimab-csfr
Dosage form: injection (50 mg/mL)

Medically reviewed by Melisa Puckey, BPharm. Last updated on Aug 15, 2024.

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 is 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 treatment 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 clinical trial AGAVE-201 75% of patients achieved an overall response rate (ORR) within the first six months of treatment, with a median time to response of 1.5 months, using the approved dose of 0.3 mg/kg every two weeks. Also, 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 binds and blocks 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

The most common Niktimvo side effects are muscle and joint pain, tiredness (fatigue), infection (pathogen unspecified), nausea, headache, diarrhea, cough, bacterial infection, viral infection, fever (pyrexia), and difficulty breathing (dyspnea). 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), increased alkaline phosphatase (ALP). The common side effects and changes in laboratory results occurred in 15% or more patients.

Clinically relevant Niktimvo side effects that occurred in 10% or less 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.

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 Injection: 50 mg/mL NDC 50881-012-10

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.