Niktimvo (axatilimab)
An approved treatment for Chronic Graft-versus-Host Disease.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Axatilimab wherever you encounter it, plus the key facts at a glance.
- Generic name
- Axatilimab
- Brand name
- Niktimvo
- Development code
- SNDX-6352
- Drug class
- Anti-CSF-1R monoclonal antibody
- Manufacturer
- Syndax Pharmaceuticals / Incyte Corporation
- How it's taken
- Given as an intravenous (IV) infusion, 0.3mg/kg (up to 35mg maximum) over 30 minutes every 2 weeks.
The first anti-CSF-1R antibody approved for any indication.
Axatilimab targets monocytes and macrophages, the immune cells that drive both inflammation and fibrosis in late-stage cGVHD, and demonstrates efficacy even in patients who have failed other novel agents.
We'll email you when Niktimvo's FDA label changes, when the FDA acts on it, and when new trials for Chronic Graft-versus-Host Disease open. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
We never share your email. Unsubscribe anytime. Privacy policy · Check any health site
Where Axatilimab fits
The most recently approved cGVHD agent (August 2024), positioned for patients who have failed two or more prior systemic therapies.
- Distinguished by efficacy in patients refractory to all other approved agents (ruxolitinib, belumosudil, ibrutinib).
- The anti-macrophage, anti-fibrotic mechanism addresses a previously untargeted driver of late-stage disease.
- Combination trials with ruxolitinib for frontline use are underway.
How Axatilimab works
In severe, treatment-resistant chronic GVHD, monocytes and macrophages become overactivated and drive both the inflammatory tissue attack and the progressive scarring (fibrosis) that defines late-stage disease. Two growth factors, CSF-1 and IL-34, keep these cells activated by binding to the CSF-1 receptor on their surface.
Axatilimab is an antibody that blocks CSF-1R, cutting off the fuel supply to inflammatory monocytes and macrophages. When this receptor is blocked, the population of overactivated macrophages drops, their production of fibrosis-promoting factors decreases, and the immune balance shifts back toward health.
This mechanism is particularly valuable because it targets macrophages, which play a central role in the chronic scarring that makes late-stage cGVHD so difficult to treat. In the AGAVE-201 trial, 74% of heavily pretreated patients responded, including many who had already failed ruxolitinib, belumosudil, and ibrutinib.
Side effects and safety
Common side effects include infections (both viral and bacterial), musculoskeletal pain, fatigue, nausea, headache, diarrhea, cough, fever, and shortness of breath. Liver enzyme elevations can occur and are monitored with regular blood tests. Infusion-related reactions are possible and are managed with premedication (antihistamines and antipyretics) in patients who have had prior reactions.
Serious adverse events occurred in 44% of patients in clinical trials, most commonly infections. Niktimvo may harm an unborn baby, so people who can become pregnant should use effective birth control during treatment and for 30 days after the last dose. Blood tests for liver enzymes, CPK (a muscle enzyme), amylase, and lipase are checked before starting, every 2 weeks for the first month, and every 1 to 2 months after that.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Axatilimab
Given as an intravenous (IV) infusion, 0.3mg/kg (up to 35mg maximum) over 30 minutes every 2 weeks. Patients must weigh at least 40kg. Treatment continues until the disease gets worse or side effects become too hard to manage. Premedication with antihistamines and fever-reducing medication is recommended for patients with prior infusion reactions.
Availability and cost
Only available as the brand-name product.
Help paying for Niktimvo
With private insurance, the drugmaker's copay card can lower what you pay for Niktimvo. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE, but charity funds often can. Of the 3 charity funds for Chronic Graft-versus-Host Disease, 1 was open on October 26, 2026. People on Medicare may still qualify for the drugmaker's free medicine program. Without insurance, you can apply to the drugmaker's free medicine program, which sets its own eligibility rules. Pick your insurance below to see which offers fit you.
- Copay help
Eligible commercially insured patients may pay as little as $15, subject to limits. Medicare, Medicaid, TRICARE and uninsured patients not eligible.
For: private insurance · source - Free medicine program
Free Niktimvo for uninsured, Medicare, or denied-coverage patients who meet household income criteria. Not for patients in Alternate Funding Programs.
For: no insurance, underinsured, Medicare · source - Insurance and case manager help
IncyteCARES reviews insurance coverage, checks eligibility for assistance, and refers to independent foundations for drug, travel or lodging costs.
The official page does not say who qualifies. Ask the program. · source
Good to know: Proof of household income required for free drug. Program phone hours Mon-Fri 8 AM-8 PM ET.
Charity funds for Chronic Graft-versus-Host Disease
- From a charity · TotalAssist (formerly PAN Foundation)Graft-vs-Host Disease (GVHD) fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
- From a charity · NMDP (formerly Be The Match / National Marrow Donor Program)NMDP Patient Financial Assistance Grants fundApply directly
Pays for: Out-of-pocket costs before and after blood or marrow transplant, ongoing GVHD treatment costs, clinical-trial travel and financial-crisis grants.
The foundation says: “Status not shown on page”
Every program and charity fund for Chronic Graft-versus-Host Disease →
Axatilimab treatment timeline for Chronic Graft-Versus-Host Disease
Tap how long you have been on Axatilimab to drop your pin on the road. Your answers stay on this device.
Stage 1 of 5 · Start to 1 mo
Starting axatilimab
You are starting axatilimab (Niktimvo) after your chronic GVHD has not responded to at least two prior therapies. You may have already tried ruxolitinib, ibrutinib, belumosudil, or other agents. Axatilimab works through a completely different mechanism: it targets CSF-1R on monocytes and macrophages, blocking these cells from driving inflammation and fibrosis. It was the first anti-CSF-1R antibody ever approved for any condition (August 2024). The drug is given as an IV infusion every 2 weeks at your infusion center. Axatilimab may harm an unborn baby, so the label advises birth control during treatment and for 30 days after. Before your first infusion, your team established baseline labs. If you had an infusion-related reaction during your first dose, you may receive premedication (antihistamines and acetaminophen) for subsequent infusions. Because you are heavily immunosuppressed from prior therapies, practical infection precautions matter: frequent handwashing, avoiding close contact with people who are sick, staying current on non-live vaccinations, and contacting your team immediately if you develop fever or feel unwell rather than waiting for your next appointment. Your team should also review your current medications for interactions, though axatilimab has fewer drug interaction concerns than oral therapies like ruxolitinib or ibrutinib.
Coming up
During the first month (first 2 infusions), your team will closely monitor how you tolerate the drug. Infusion-related reactions can occur but are generally manageable. Between infusions, you will have labs checked. Your team will watch for early side effects including infections (the most common serious adverse event), liver enzyme changes, and any new symptoms. Symptom improvement can begin within the first few weeks. In the AGAVE-201 trial, the median time to a first response was about 1.5 months. If you are still on corticosteroids, your team will observe your initial response before starting a taper.
Get in touch with your transplant team if you notice
- Infusion reaction: fever, chills, rigors, hypotension, or rash during or shortly after infusion (tell your infusion team immediately)
- Fever, severe cough, or difficulty breathing between infusions (infections were the most common side effect in the trial, and serious side effects of any kind occurred in 44% of people)
- Right upper abdominal pain, nausea, dark urine, or yellowing of skin (possible liver injury)
- Severe fatigue, pallor, or bleeding (blood count effects)
- Rapid worsening of GVHD symptoms before the drug has had time to work
- What is the infusion schedule, and how long does each infusion take?
- Will I be premedicated before infusions to prevent reactions?
- How will this drug work differently from the ones I already tried?
- What infection precautions should I take (I have been on multiple immunosuppressive drugs)?
- How soon might I start noticing improvement in my symptoms?
- Are there any medications I should avoid while on axatilimab?
Every stage of Axatilimab treatment at a glance
Starting axatilimab Start to 1 mo
You are starting axatilimab (Niktimvo) after your chronic GVHD has not responded to at least two prior therapies. You may have already tried ruxolitinib, ibrutinib, belumosudil, or other agents. Axatilimab works through a completely different mechanism: it targets CSF-1R on monocytes and macrophages, blocking these cells from driving inflammation and fibrosis. It was the first anti-CSF-1R antibody ever approved for any condition (August 2024). The drug is given as an IV infusion every 2 weeks at your infusion center. Axatilimab may harm an unborn baby, so the label advises birth control during treatment and for 30 days after. Before your first infusion, your team established baseline labs. If you had an infusion-related reaction during your first dose, you may receive premedication (antihistamines and acetaminophen) for subsequent infusions. Because you are heavily immunosuppressed from prior therapies, practical infection precautions matter: frequent handwashing, avoiding close contact with people who are sick, staying current on non-live vaccinations, and contacting your team immediately if you develop fever or feel unwell rather than waiting for your next appointment. Your team should also review your current medications for interactions, though axatilimab has fewer drug interaction concerns than oral therapies like ruxolitinib or ibrutinib.
During the first month (first 2 infusions), your team will closely monitor how you tolerate the drug. Infusion-related reactions can occur but are generally manageable. Between infusions, you will have labs checked. Your team will watch for early side effects including infections (the most common serious adverse event), liver enzyme changes, and any new symptoms. Symptom improvement can begin within the first few weeks. In the AGAVE-201 trial, the median time to a first response was about 1.5 months. If you are still on corticosteroids, your team will observe your initial response before starting a taper.
Early response evaluation 1 mo to 3 mo
You have received several infusions of axatilimab and your transplant team is evaluating your initial response. In the AGAVE-201 trial, the overall response rate at the approved dose was 75%, and at least a 7-point drop on the Lee Symptom Scale occurred in 56% of people at this dose. These numbers are especially notable because these were patients who had failed multiple prior therapies, including many who had tried all three other approved agents. Your team is tracking your organ scores, symptom burden, and functional status. If you are responding, you may be noticing less skin tightness, easier eating, better range of motion, or improvement in other symptoms that have been limiting you.
If you are responding: your team will continue infusions on the every-2-week schedule and may begin tapering other immunosuppressive medications (steroids in particular). In the trial, 60% of people who responded did not need a new cGVHD treatment for at least 12 months. If you are partially responding: some patients continue to improve over the next several months, particularly in fibrotic manifestations. Your team will likely continue treatment. If you are not responding: this is a difficult situation since axatilimab is typically used after other approved agents have failed. Your team may discuss clinical trials testing newer mechanisms, combination approaches, or other investigational agents. If side effects are problematic: dose adjustments or supportive care measures may help, and your team will weigh the benefits against the side effect burden.
Response assessment 3 mo to 6 mo
This is the formal assessment period. By 3-6 months, your transplant team has a clear picture of how you are responding to axatilimab. Your organ scores, symptom burden, steroid dose, and functional status are all being compared to where you started. In the trial, responses were typically evident within the first few months, so by this point your trajectory is fairly well-established. If you have achieved a meaningful response and are tolerating the infusions, axatilimab is working. If you have made modest improvement but still have significant disease burden, your team is weighing whether the level of improvement justifies continuing versus exploring other options.
For patients responding well: you will continue biweekly infusions. There is limited data on how long patients need to stay on axatilimab, so the current approach is to continue as long as it is working and tolerated. The label gives only an every 2 weeks schedule. Spacing infusions further apart is not part of the label, so any change is a question for your team. Steroid tapering should be well underway or complete. For patients with partial response: your team may continue several more months to see if improvement continues. Some patients respond slowly, particularly in fibrotic organs. For patients not responding: the conversation shifts to investigational approaches and clinical trials. Your team may also revisit whether palliative or supportive strategies can improve your quality of life even without achieving GVHD control.
Sustained treatment 6 mo to 18 mo
You have been on axatilimab for 6 months or more and are in sustained treatment. The every-2-week infusion schedule means you have developed a routine with your infusion center. If your GVHD is well-controlled, you can ask your team about the schedule, though the label lists only every 2 weeks. Your monitoring continues with pre-infusion labs and periodic organ assessments. At this stage, the focus extends beyond GVHD control to overall survivorship care: managing chronic organ damage, infection prevention, cancer screening, and quality of life optimization.
The key question during this phase is how long to continue infusions. Since axatilimab is the newest approved agent (August 2024), there is less long-term experience compared to the other approved drugs. Your team will make individualized decisions about treatment duration based on your disease severity, depth of response, and whether stopping treatment is likely to lead to a flare. If your team does try extending the interval between infusions or stopping, they will monitor you closely for relapse. If your GVHD flares, your team will talk with you about restarting regular infusions or other options.
Long-term management 18 mo on
You are in long-term management, either continuing axatilimab infusions, having transitioned to an extended schedule, or having completed a course of treatment. As the newest cGVHD agent, long-term data with axatilimab is still being generated by real-world experience and ongoing clinical trials. Your care now focuses on maintaining GVHD control, managing chronic effects of the disease and its treatment, and comprehensive survivorship monitoring. You may be working with multiple specialists: your transplant team, pulmonologist (if lung involvement), ophthalmologist (for dry eyes), dermatologist (for skin monitoring), and physical or occupational therapists (for mobility).
Long-term follow-up continues with your transplant team. If you are still on axatilimab infusions, periodic reassessment of whether to continue, reduce frequency, or attempt discontinuation will occur. The cGVHD treatment landscape is evolving rapidly, and combination studies pairing axatilimab with earlier-line agents are generating data that may change how the drug is used in the future. Regardless of your treatment status, continued surveillance for secondary malignancies, infection management, and functional rehabilitation remain priorities.
This timeline shows typical patterns from the prescribing information, guidelines and published studies. Your own treatment may differ, and your transplant team is the source of truth for your plan. Last checked against the FDA label on .
Clinical trial results
The Phase 2 AGAVE-201 trial tested 3 dose levels in 241 patients with active, recurrent, or refractory cGVHD after two or more prior therapies. At the selected dose (0.3mg/kg every 2 weeks), the overall response rate was 74%. Clinically meaningful symptom improvement (at least 30% reduction on the Lee Symptom Scale) occurred in 55% of patients. Responses were durable, with 60% maintaining response at 12 months. Notably, axatilimab worked even in patients who had previously failed all other approved cGVHD therapies.
Development history
Axatilimab was developed by Syndax Pharmaceuticals (originally licensed from UCB) and advanced in partnership with Incyte Corporation under a co-development agreement from 2021. The AGAVE-201 trial demonstrated strong efficacy in the most difficult-to-treat cGVHD patients. FDA granted Priority Review and approved axatilimab on August 14, 2024, making it the newest approved cGVHD agent and the first anti-CSF-1R antibody approved for any indication.
Explore Chronic Graft-versus-Host Disease trials
Other Chronic Graft-versus-Host Disease treatments
Common questions about Axatilimab
▸What is Axatilimab (Niktimvo)?
The first anti-CSF-1R antibody approved for any indication. Axatilimab targets monocytes and macrophages, the immune cells that drive both inflammation and fibrosis in late-stage cGVHD, and demonstrates efficacy even in patients who have failed other novel agents.
▸How does Axatilimab work?
In severe, treatment-resistant chronic GVHD, monocytes and macrophages become overactivated and drive both the inflammatory tissue attack and the progressive scarring (fibrosis) that defines late-stage disease. Two growth factors, CSF-1 and IL-34, keep these cells activated by binding to the CSF-1 receptor on their surface.
Axatilimab is an antibody that blocks CSF-1R, cutting off the fuel supply to inflammatory monocytes and macrophages. When this receptor is blocked, the population of overactivated macrophages drops, their production of fibrosis-promoting factors decreases, and the immune balance shifts back toward health.
This mechanism is particularly valuable because it targets macrophages, which play a central role in the chronic scarring that makes late-stage cGVHD so difficult to treat. In the AGAVE-201 trial, 74% of heavily pretreated patients responded, including many who had already failed ruxolitinib, belumosudil, and ibrutinib.
▸What are the side effects of Axatilimab?
Common side effects include infections (both viral and bacterial), musculoskeletal pain, fatigue, nausea, headache, diarrhea, cough, fever, and shortness of breath. Liver enzyme elevations can occur and are monitored with regular blood tests. Infusion-related reactions are possible and are managed with premedication (antihistamines and antipyretics) in patients who have had prior reactions. Serious adverse events occurred in 44% of patients in clinical trials, most commonly infections. Niktimvo may harm an unborn baby, so people who can become pregnant should use effective birth control during treatment and for 30 days after the last dose. Blood tests for liver enzymes, CPK (a muscle enzyme), amylase, and lipase are checked before starting, every 2 weeks for the first month, and every 1 to 2 months after that.
▸How is Axatilimab taken?
Given as an intravenous (IV) infusion, 0.3mg/kg (up to 35mg maximum) over 30 minutes every 2 weeks. Patients must weigh at least 40kg. Treatment continues until the disease gets worse or side effects become too hard to manage. Premedication with antihistamines and fever-reducing medication is recommended for patients with prior infusion reactions.
▸Is Axatilimab FDA approved?
Yes, Axatilimab (Niktimvo) is FDA approved (2024) for the treatment of Chronic Graft-versus-Host Disease.
▸What is Niktimvo used for?
Niktimvo (axatilimab) is an anti-CSF-1R antibody approved by the FDA on August 14, 2024 for chronic GVHD after failure of at least two prior systemic therapies. It targets monocytes and macrophages, addressing both inflammation and fibrosis in late-stage disease, and is the first anti-CSF-1R antibody approved for any indication. [1]
▸Does axatilimab work after other GVHD drugs have failed?
Yes. In the Phase 2 AGAVE-201 trial of 241 patients, axatilimab achieved a 74% response rate at the approved dose even in patients who had previously failed ruxolitinib, belumosudil, and ibrutinib, and 60% of responders maintained their response at 12 months. This makes it a valuable option for treatment-resistant cGVHD. [2]
▸How is Niktimvo given?
Niktimvo (axatilimab) is given as an intravenous infusion of 0.3mg/kg, up to a maximum of 35mg, over 30 minutes every 2 weeks. Patients must weigh at least 40kg. Treatment continues until the disease gets worse or side effects become too hard to manage, and premedication with antihistamines and fever-reducing medication is recommended for patients who have had prior infusion reactions. [1]
▸What are the side effects of axatilimab?
Common side effects of axatilimab (Niktimvo) include viral and bacterial infections, musculoskeletal pain, fatigue, nausea, headache, diarrhea, cough, fever, and shortness of breath. Serious adverse events occurred in 44% of patients in clinical trials, most commonly infections, and liver enzyme elevations can occur. Blood tests for liver enzymes, CPK, amylase, and lipase are checked before starting, every 2 weeks for the first month, and every 1 to 2 months after that, and people who can become pregnant should use effective birth control during treatment and for 30 days after the last dose. [1]
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. Food and Drug Administration · August 14, 2024. FDA approves axatilimab-csfr for chronic graft-versus-host disease. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease
- Syndax / Incyte. Axatilimab for Chronic Graft-versus-Host Disease After Failure of at Least Two Prior Therapies. https://investor.incyte.com/news-releases/news-release-details/incyte-and-syndax-announce-us-fda-approval-niktimvotm-axatilimab
- Incyte Corporation. IncyteCARES Patient Support, Niktimvo. https://www.incytecares.com/oncology-hematology-niktimvo/