Niktimvo (axatilimab)
An approved treatment for Chronic Graft-versus-Host Disease.
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.
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.
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.
Mechanism: Humanized monoclonal antibody targeting colony-stimulating factor-1 receptor (CSF-1R) on monocytes and macrophages, blocking their activation and reducing both inflammation and fibrosis in chronic GVHD
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, 0.3 mg/kg (up to 35 mg maximum) over 30 minutes every 2 weeks. Patients must weigh at least 40 kg. 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
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- 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.
- 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”
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 (0.3 mg/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, 0.3 mg/kg (up to 35 mg maximum) over 30 minutes every 2 weeks. Patients must weigh at least 40 kg. 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 for chronic GVHD after failure of at least two prior therapies. It targets monocytes and macrophages, addressing both inflammation and fibrosis in late-stage disease.
▸Does axatilimab work after other GVHD drugs have failed?
Yes. In the AGAVE-201 trial, axatilimab achieved a 74% response rate even in patients who had previously failed ruxolitinib, belumosudil, and ibrutinib, making it a valuable option for treatment-resistant cGVHD.
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/