Axatilimab-Csfr
Axatilimab-csfr is a targeted antibody therapy used to treat chronic graft-versus-host disease (cGVHD) in adults and certain pediatric patients when at least two prior treatments have not worked.
🧬 Where this information comes from Click to expand
The clinical label records used on this page are FDA-filed Structured Product Labeling (SPL) retrieved through openFDA. We identified 1 clinical label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. Forms, routes, brands, labelers, strengths, and NDC product-record counts are built separately from matching FDA National Drug Code Directory records. The linked DailyMed document is a representative official source for verification, not the page’s only clinical source. View the linked label on DailyMed →
📖 Axatilimab-Csfr: Patient Information Guide
A plain-language walkthrough of Axatilimab-Csfr — what it treats, how it works, how it’s taken, side effects, warnings and interactions. Tap any section to expand it.
🎯What it's used for▾
Axatilimab-csfr is a targeted antibody used to treat a specific immune complication following stem cell or bone marrow transplant.
- Niktimvo (intravenous injection) is approved for the treatment of chronic graft-versus-host disease (cGVHD) in adults and pediatric patients weighing at least 40 kg, after at least two prior lines of systemic therapy have not worked.
- Niktimvo has not been shown to be safe or effective in patients weighing less than 40 kg.
⚙️How it works▾
Axatilimab-csfr is a monoclonal antibody — a targeted lab-made protein — that locks onto a receptor called CSF-1R (colony stimulating factor-1 receptor) found on the surface of monocytes and macrophages (two types of immune cells). In cGVHD, these cells become overactive and cause damaging inflammation and scarring in organs and tissues. By blocking CSF-1R, axatilimab-csfr reduces the levels of these harmful immune cells in the bloodstream and suppresses their destructive activity in body tissues, helping to ease the damage caused by cGVHD.
💊How it's taken▾
Niktimvo is given as an intravenous (IV) infusion — directly into a vein — once every two weeks. Each infusion takes 30 minutes and is administered by a trained healthcare professional in a clinical setting. It is not taken by mouth and is not a self-administered medication.
- Lab tests (liver enzymes, muscle enzymes, and others) are checked before starting and regularly throughout treatment
- Your dose may be delayed, reduced, or stopped depending on how you respond or if side effects occur — follow your care team's guidance
- Do not miss scheduled infusions without talking to your doctor first
🤒Side effects — in detail▾
The most common side effects reported in the clinical trial (occurring in 15% or more of patients) include:
- Infections — bacterial, viral, or unspecified (such as pneumonia, urinary tract infections, herpes zoster, or COVID-19)
- Elevated liver enzymes (AST, ALT, GGT, ALP) — usually detected on blood tests
- Low phosphate levels in the blood
- Low hemoglobin (anemia)
- Musculoskeletal pain — joint pain, muscle pain, back pain
- Fatigue or weakness
- Nausea or diarrhea
- Headache
- Cough or shortness of breath
- Fever
- Elevated lipase, amylase, and CPK levels (detected on blood tests)
Serious reactions to watch for and act on immediately include severe infusion reactions (fever, chills, rash, difficulty breathing), signs of serious infection (high fever, worsening illness, difficulty breathing), and signs of liver injury (yellowing of the skin or eyes, severe abdominal pain, dark urine).
⚠️Warnings & precautions▾
- Infusion-related reactions: About 18% of patients experienced a reaction during or after the infusion, including fever, chills, rash, flushing, shortness of breath, and high blood pressure. Severe reactions (Grade 3–4) occurred in about 1.3% of patients. Your care team will monitor you closely during infusions. If you've had a reaction before, you'll be pre-medicated with an antihistamine and a fever reducer. Severe reactions may require permanently stopping Niktimvo.
- Fetal harm (embryo-fetal toxicity): Based on how axatilimab-csfr works — blocking pathways involved in fetal immune development — it has the potential to harm an unborn baby. Women who could become pregnant must use effective contraception during treatment and for 30 days after the last dose. A pregnancy test is required before starting treatment.
- Breastfeeding risk: Because the drug could potentially pass into breast milk and harm a nursing infant, breastfeeding is not recommended during treatment or for 30 days after the last dose.
- Bone growth monitoring in children: In animal studies, axatilimab-csfr was associated with changes in bone growth plates. Pediatric patients receiving Niktimvo should have their bone growth and development monitored.
🔀What it interacts with▾
The FDA label for Niktimvo does not identify specific drug, food, or supplement interactions.
- Always tell your doctor and pharmacist about all medications, supplements, and vitamins you take before starting Niktimvo
This is not a complete list of interactions. Always talk with your doctor or pharmacist before starting, stopping, or changing any medication.
📋Before taking it▾
- Pregnancy: Niktimvo may cause fetal harm. Tell your doctor if you are pregnant or could become pregnant. A pregnancy test is required before starting treatment, and effective contraception must be used during and for 30 days after treatment.
- Breastfeeding: Do not breastfeed during treatment or for 30 days after the last dose.
- Pediatric patients: Niktimvo is approved for children and adolescents weighing at least 40 kg. It has not been shown to be safe or effective in those weighing less than 40 kg. Bone growth should be monitored in pediatric patients.
- Older adults (65+): No meaningful differences in safety or effectiveness were observed in patients 65 and older compared to younger patients in the clinical trial.
- Kidney impairment: Mild to moderate kidney impairment does not appear to meaningfully affect how the body handles Niktimvo. The effect of severe kidney impairment is not known.
- Liver impairment: Mild liver impairment does not appear to meaningfully affect Niktimvo. The effect of moderate to severe liver impairment is not known — discuss this with your doctor.
- Active infections: Because Niktimvo affects immune cells, tell your doctor about any current or recent infections before starting treatment.
📡Pharmacodynamics — effects in the body▾
Axatilimab-csfr produces measurable changes in the immune system that can be tracked in the blood. It causes a dose-related reduction in the levels of nonclassical monocytes — a subset of inflammatory immune cells — circulating in the bloodstream. At the same time, it causes dose-related increases in the levels of CSF-1 and interleukin-34 (IL-34), two proteins that normally signal through the CSF-1R receptor that axatilimab-csfr is blocking. These changes reflect the drug's active engagement with its target in the body.
🔬Pharmacokinetics — how your body processes it▾
Axatilimab-csfr is given intravenously, so it enters the bloodstream directly. It distributes into a relatively small volume in the body and is broken down (metabolized) into small peptides (protein fragments) through normal protein-breakdown pathways — not through the liver enzyme system. The drug's clearance from the body is complex: it clears more slowly at higher doses, and its half-life (the time for blood levels to drop by half) increases substantially with dose. After the recommended every-two-week dosing schedule, drug does not meaningfully build up in the body over time. Mild to moderate kidney impairment and mild liver impairment do not appear to significantly change how the body handles this medication; the impact of more severe organ impairment is not yet known.
📦How to store it▾
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.
📄 Written from Axatilimab-Csfr’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Every point is grounded in that labeling — nothing is invented. How we use AI →
🩺 Pharmacist Counseling Corner
Quick, plain-English answers to the questions patients ask most about Axatilimab-Csfr — the kind of thing our pharmacy team would talk through with you at the counter.
What exactly is chronic graft-versus-host disease, and why is Niktimvo used for it? ▾
How is Niktimvo given, and how often will I need to go in for treatment? ▾
What side effects should I actually watch out for? ▾
Is it safe to get pregnant or breastfeed while on Niktimvo? ▾
What happens if I have a reaction during the infusion? ▾
Can my child receive Niktimvo? ▾
Is Axatilimab-Csfr available over the counter? ▾
When was Axatilimab-Csfr first available? ▾
Who makes Axatilimab-Csfr? ▾
💬 Answers drafted from Axatilimab-Csfr’s FDA-approved label information, summarized in plain language with AI, and reviewed by our pharmacy team before publication. Grounded in that labeling — nothing is made up. How we use AI →
🧰 Keep exploring
💊 How Axatilimab-Csfr comes
Axatilimab-Csfr is available in 2 forms. Different forms and brands can have different approved uses, doses, schedules, and directions. Follow the instructions for your exact product, and do not switch forms without guidance from your prescriber or pharmacist.
Injection
How this form is used
- Niktimvo is used to treat chronic graft-versus-host disease (cGVHD) in adults and pediatric patients weighing at least 40 kg after at least two prior systemic therapies have failed
- Niktimvo is administered as an intravenous infusion over 30 minutes, once every two weeks
- Niktimvo must be diluted before administration and given through a dedicated infusion line with a sterile 0.2-micron in-line filter
- Niktimvo should not be mixed or co-administered with other drugs through the same infusion line
- Niktimvo comes in different vial concentrations — healthcare staff should verify the vial label carefully before preparation to avoid dosing errors
Oral solution
📊 Axatilimab-Csfr across the U.S. market
How Axatilimab-Csfr appears in the FDA’s National Drug Code Directory — including its dosage forms, strengths, brand names, manufacturers, and FDA-listed product records. These are directory records, not sales or prescription volume.
FDA-listed product records by dose form (tablet variants merged) — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
Share of FDA-listed product records by manufacturer — FDA National Drug Code Directory. Record counts are not sales or prescription volume.
📈 How widely Axatilimab-Csfr is used
A general measure of how commonly Axatilimab-Csfr is prescribed, based on Medicaid — one of the largest public drug programs. The figure below is prescriptions filled in Q1–Q4 2025, summed across every form and brand of the drug.
Think of this as a proxy for how widely Axatilimab-Csfr is used, drawn from freely available public data. Medicaid is just one program — these numbers don’t include Medicare, other government coverage, or commercial and cash-pay prescriptions, so real-world use is higher than what’s shown here.
How commonly is Axatilimab-Csfr prescribed? Of the 1,596 medications we measure, Axatilimab-Csfr ranks #1,407 by Medicaid prescriptions — more than 12% of them.
Utilization by Axatilimab-Csfr product
Pick a product to see its own Medicaid numbers. Each chip is one clinical product — a specific strength & dosage form as defined by RxNorm — with brand-name and generic versions combined.
Other Axatilimab-Csfr products — combination packs and listings not yet matched to a specific clinical product (kept visible so every prescription is accounted for)
Summed across the 2 of 5 listed NDC products with Medicaid activity.
Shares are of the Medicaid prescriptions shown here. Product grouping follows RxNorm (the National Library of Medicine’s drug terminology), so “one product” means one strength & dosage form regardless of manufacturer. Dollar figures are gross Medicaid reimbursement before mandatory rebates — rebates (often large, especially for brand-name drugs) are confidential, so actual net cost to Medicaid is lower than shown.
Source: Medicaid State Drug Utilization Data (CMS), aggregated by generic ingredient — summed across every NDC (all brands, strengths, salt forms and dosage forms) of Axatilimab-Csfr, and across all states and both fee-for-service and managed-care claims. A general popularity signal; it excludes Medicare, commercial insurance and cash prescriptions, so it is not total U.S. use. Based on the 2 of 5 listed Axatilimab-Csfr NDCs with Medicaid activity.
🔍 Compare Axatilimab-Csfr products
A representative set of FDA-listed product records for Axatilimab-Csfr — across manufacturers, strengths, and dosage forms, grouped by form with each product’s manufacturer and how the FDA approved it. The same medicine may be made and packaged by different companies, so a single drug can have many directory entries. (It’s also why a refill can look different — a new shape, color, or box — even though it contains the same medication.)
| Strength | Route | Manufacturer / Labeler | Category ⓘ | Details |
|---|---|---|---|---|
| 💊Injection | ||||
| 9 mg/0.9 mL | Intravenous | Incyte Corporation | BLA | View NDC → |
| 9 mg/0.18 mL | Intravenous | Incyte Corporation × 2 listings | BLA | View NDC → |
| 22 mg/0.44 mL | Intravenous | Incyte Corporation × 2 listings | BLA | View NDC → |
| 22 mg/2.2 mL | Intravenous | Incyte Corporation | BLA | View NDC → |
| 50 mg/mL | Intravenous | Incyte Corporation × 2 listings | BLA | View NDC → |
| 💊Oral solution | ||||
| 50 mg/mL | — | AGC Biologics, Inc. | DRUG FOR FURTHER PROCESSING | View NDC → |
Showing 9 of 9 products — FDA National Drug Code Directory. See every product, package size and price: browse all 9 Axatilimab-Csfr NDCs →
📈 What people report — real-world data (FDA FAERS)
After a medicine reaches the market, patients, doctors and drugmakers can send the FDA reports of problems they think may be linked to it. Here’s what’s been reported for Axatilimab-Csfr — a signal of what to watch for, not proof the drug caused it.
Source: openFDA, from the FDA Adverse Event Reporting System (FAERS). These are voluntary reports — they don’t prove the drug caused the effect, and counts reflect how often something was reported, not how often it actually happens. Reports also often name the very problem the medicine is taken for — a common reason to file one is that the drug didn’t help — so for a pain reliever you may see “pain” high on the list; that points to why the report was filed, not to the drug causing the symptom. This counts every report that lists Axatilimab-Csfr in any role, so the total runs higher than the FDA’s official dashboard, which counts only cases where the drug is the suspect. For the authoritative figures, see the FDA FAERS Public Dashboard. Always talk to your pharmacist or doctor.
🏛️ The road to your pharmacy
How Axatilimab-Csfr went from FDA review to the pharmacy shelf — the key milestones in its regulatory journey.
Source: Drugs@FDA.
🚨 Recall history
A recall is when a specific batch of a medicine is pulled from the market — usually a manufacturing issue, not a problem with the drug itself. Class I is most serious, Class III least.
Source: openFDA drug enforcement (recall) reports.
RxNorm Concept Web — From Ingredient to Every Form and Strength
This page starts with one ingredient concept (IN). The branches below show its dose-form concepts (SCDF), then the available clinical drug and strength concepts (SCD). Combination products remain separate concepts with their own pages.
RxCUI 2691526 1 dose form · 5 strengths
-
Dose form (SCDF) Injection RxCUI 2691531In current FDA listingsClinical drug / strength (SCD) 0.18 ML axatilimab-csfr 50 MG/MLRxCUI 27030250.44 ML axatilimab-csfr 50 MG/MLRxCUI 27030230.9 ML axatilimab-csfr 10 MG/MLRxCUI 27465511 ML axatilimab-csfr 50 MG/MLRxCUI 26915342.2 ML axatilimab-csfr 10 MG/MLRxCUI 2746546
Look up RxCUI 2691526 in the RxCUI Atlas →
RxNorm is the U.S. National Library of Medicine’s normalized drug vocabulary. Its concept hierarchy can include forms that are not currently marketed; the status on each branch compares that concept with current FDA listings. RxCUIs identify vocabulary concepts, not individual packages or manufacturers.
📦 Supply & shortage status
Whether Axatilimab-Csfr is in short supply in the U.S. right now, plus any shortage history the FDA has on record. A shortage usually reflects a manufacturing or demand issue — not a safety problem with the drug.
Source: openFDA, from the FDA Drug Shortages database.
🏷️ Sold under these brand names
🏭 Manufacturers & labelers
🔬 Where this comes from
Core label, product, RxNorm, safety, and utilization data on this page come from the sources identified below. AI-written, editorial, and licensed sections are labeled separately.
How we combine label and product data
HelloPharmacist combines public FDA and NLM records; we don’t author the underlying clinical facts. We identified 1 FDA-filed SPL label set within the page’s selected clinical scope after exact ingredient or ingredient-combination matching and applicable route/form matching. The representative label is the starting point for the displayed reference monograph; the 12 largest stored in-scope SPL records, which may include that representative, are compared section by section, and the longest available version of each section is retained. Forms, routes, brands, labelers, product-listed strengths, and product-record counts are aggregated separately from ingredient-matched FDA NDC Directory records; package records listed above are associated with the linked SPL; and the normalized concept hierarchy comes from NLM RxNorm.
For canonical ingredient pages, bounded product-attributed indication and administration excerpts may be added to the AI evidence, and the generator is instructed to preserve available product, brand, route, and form qualifiers instead of treating one label as universal. Where a section is identified as AI-written, its plain-language text is generated from bounded label evidence. New draft drug pages remain in the admin review queue before first publication. Incyte Corporation is the representative DailyMed label linked for verification and dates; it is not the sole source used for the page.
This is general education, not medical advice — always consult your doctor or pharmacist about your medications and any medical condition. For the exact wording of the linked representative label, view that label on DailyMed; some displayed composite sections may come from other in-scope SPL labels. Learn how we use AI and our data sources & update cadence.