Version July 2025
Dosage guidance:
Safety: Patients who previously experienced an infusion-related reaction to axatilimab should premedicate with an antihistamine and antipyretic with subsequent infusions.
Graft-versus-host disease, chronic: Note: Reserve use for patients who have failed ≥2 prior lines of systemic therapy.
Children and Adolescents, weighing ≥40 kg: IV: 0.3 mg/kg/dose every 2 weeks; maximum dose: 35 mg/dose; continue until disease progression or unacceptable toxicity (Ref).
Dosage adjustment for toxicity:
Children and Adolescents, weighing ≥40 kg: IV:
|
Adverse reaction |
Description/severity |
Axatilimab dosage modification |
|---|---|---|
|
Increases in CPK, amylase, or lipase |
≥ Grade 3, asymptomatic |
Perform diagnostic evaluation for evidence of end-organ damage. • If no evidence, continue without dose reduction. • If evidence, permanently discontinue therapy. |
|
≥ Grade 3, symptomatic |
Permanently discontinue therapy. | |
|
Other non-hematologic adverse effects |
Grade 3 |
Withhold axatilimab therapy until recovery to grade 2. • If recovery delayed therapy by ≤4 weeks from planned infusion, decrease dose to 0.2 mg/kg/dose every 2 weeks; maximum dose: 23 mg/dose. • If recovery delayed therapy by >4 weeks from planned infusion, permanently discontinue therapy. |
|
Grade 4 |
Permanently discontinue therapy. |
Children and Adolescents, weighing ≥40 kg: IV:
eGFR ≥30 mL/minute: No adjustment needed; pharmacokinetic analysis reported no clinically meaningful differences in exposure in patients with mild to moderate altered kidney impairment (eGFR 30 to 89 mL/minute) and normal kidney function.
eGFR <30 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling; has not been studied.
Children and Adolescents, weighing ≥40 kg: IV:
|
Adverse reaction |
Description/severity |
Axatilimab dosage modification |
|---|---|---|
|
Baseline liver impairment |
Mild liver impairment (total bilirubin ≤ ULN and AST > ULN or total bilirubin 1 to ≤1.5 × ULN and any AST) |
No dosage adjustment necessary; pharmacokinetic analysis reported no clinically meaningful differences in exposure; monitor closely for adverse effects. |
|
Moderate to severe liver impairment (total bilirubin >1.5 × ULN and any AST) |
Has not been studied; monitor closely for adverse effects; if adverse effects occur, adjust dose based on dosage reduction and dosage adjustment for toxicity tables. | |
|
Liver toxicity during therapy | ||
|
Elevation of AST or ALT on day of dosing |
Grade 3 with total bilirubin ≤ grade 1 |
Withhold axatilimab until recovery to grade 2 then resume at a reduced dose of 0.2 mg/kg/dose every 2 weeks; maximum dose: 23 mg/dose. |
|
Elevation of AST or ALT anytime during therapy |
ALT or AST ≥3 × ULN with total bilirubin ≥2 × ULN and alkaline phosphatase <2 × ULN |
Withhold axatilimab; evaluate for drug-induced liver injury (DILI); if confirmed, permanently discontinue therapy. |
|
Grade 4 |
Permanently discontinue therapy. | |
(For additional information see "Axatilimab: Drug information")
Dosage guidance:
Clinical considerations: Should only be administered in patients weighing ≥40 kg. For patients who experienced an infusion-related reaction with a previous dose, premedicate for subsequent doses with an antihistamine and an antipyretic.
Graft-versus-host disease, chronic: Patients weighing ≥40 kg: IV: 0.3 mg/kg (maximum dose: 35 mg) once every 2 weeks until disease progression or unacceptable toxicity.
Note: Kidney function estimated by the Cockcroft-Gault equation.
CrCl ≥30 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling; however, CrCl 30 to 89 mL/minute had no clinically meaningful difference in axatilimab pharmacokinetics.
CrCl 15 to 29 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling (effect on axatilimab pharmacokinetics is unknown).
CrCl <15 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling.
Altered liver function prior to treatment initiation:
Mild impairment (total bilirubin ≤ ULN and AST > ULN or total bilirubin 1 to ≤1.5 times ULN and any AST): There are no dosage adjustments provided in the manufacturer's labeling; however, mild impairment had no clinically meaningful difference in axatilimab pharmacokinetics.
Moderate or severe impairment (total bilirubin >1.5 times ULN and any AST): There are no dosage adjustments provided in the manufacturer's labeling (effect on axatilimab pharmacokinetics is unknown).
Acute hepatotoxicity during treatment:
AST or ALT elevation (on the day of dosing):
ALT or AST ≥3 times ULN with total bilirubin ≥2 times ULN and alkaline phosphatase <2 times ULN: Withhold axatilimab and investigate for drug-induced liver injury. If confirmed, permanently discontinue axatilimab.
Grade 3 with total bilirubin ≤ grade 1: Withhold axatilimab until recovery to grade 2, then resume at 0.2 mg/kg (maximum dose: 23 mg) once every 2 weeks.
Grade 4: Permanently discontinue axatilimab.
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Reported adverse reactions are for children, adolescents, and adults.
>10%:
Cardiovascular: Edema (13%)
Endocrine & metabolic: Decreased serum phosphate (51%), increased serum calcium (31%)
Gastrointestinal: Decreased appetite (11%), diarrhea (18%; including colitis), increased serum amylase (32%), increased serum lipase (34%), nausea (23%)
Hematologic & oncologic: Decreased hemoglobin (48%), hemorrhage (11%)
Hepatic: Increased gamma-glutamyl transferase (39%), increased serum alanine aminotransferase (51%), increased serum alkaline phosphatase (28%), increased serum aspartate aminotransferase (61%)
Hypersensitivity: Infusion-related reaction (18%; including hypersensitivity reaction [13%])
Infection: Infection (57%; including bacterial infection [15%], viral infection [43%])
Nervous system: Dizziness (11%), fatigue (32%), headache (20%)
Neuromuscular & skeletal: Increased creatine phosphokinase in blood specimen (25%), musculoskeletal pain (35%)
Respiratory: Cough (18%), dyspnea (15%)
Miscellaneous: Fever (15%)
1% to 10%:
Cardiovascular: Hypertension (<10%)
Dermatologic: Pruritus (<10%), skin rash (10%; including bullous dermatitis, exfoliative dermatitis, maculopapular rash)
Ophthalmic: Periorbital edema (<10%)
Respiratory: Respiratory failure (>3%)
Frequency not defined: Immunologic: Antibody development (including neutralizing)
There are no contraindications listed in the manufacturer's labeling.
Concerns related to adverse effects:
• Infusion-related reactions: Axatilimab may cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in nearly one-fifth of axatilimab-treated patients. Grade 3 or 4 infusion related reactions occurred in a small percentage of patients. Signs/symptoms of infusion reactions have included fever, chills, rash, flushing, dyspnea, and hypertension.
Dosage form specific issues:
• Polysorbate 80: Some dosage forms may contain polysorbate 80 (also known as Tweens). Hypersensitivity reactions, usually a delayed reaction, have been reported following exposure to pharmaceutical products containing polysorbate 80 in certain individuals (Ref). Thrombocytopenia, ascites, pulmonary deterioration, and renal and hepatic failure have been reported in premature neonates after receiving parenteral products containing polysorbate 80 (Ref). See manufacturer's labeling.
Observations from animal data suggest a possible thickening of growth plate, metaphysis, and/or femoral growth plate degeneration; monitoring of bone and growth development in pediatric patients suggested.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Solution, Intravenous [preservative free]:
Niktimvo: Axatilimab-csfr 9 mg/0.18 mL (0.18 mL); Axatilimab-csfr 22 mg/0.44 mL (0.44 mL) [contains polysorbate 80]
No
Solution (Niktimvo Intravenous)
9MG/0.18ML (per 0.18 mL): $5,670.00
22MG/0.44ML (per 0.44 mL): $13,860.00
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Note: If patient previously experienced an infusion-related reaction to axatilimab, premedicate with an antihistamine and antipyretic.
IV: Administer over 30 minutes via a dedicated IV line through a low-protein 0.2 micron inline filter or an add-on polyethersulfone filter. Flush line with NS after completion of infusion.
Rate adjustment for infusion-related reactions:
Grade 1 or 2: May either temporarily interrupt the infusion or decrease infusion rate by 50%. Initiate symptomatic treatment for the reaction.
Subsequent infusions: Starting with the next infusion, premedicate with an antihistamine and antipyretic; resume infusion at 50% of the prior infusion rate.
Grade 3 or 4: Permanently discontinue axatilimab.
IV: Infuse over 30 minutes through a dedicated infusion line that includes a sterile, low-protein binding, 0.2-micron in-line or add-on polyethersulfone filter. Do not coadminister other medications through the same infusion line. After administration, flush the infusion line with NS.
Store intact vial(s) at 2°C to 8°C (36°F to 46°F); do not freeze. Store in the original carton to protect from light. Do not shake.
Solutions diluted for infusion should be used immediately; however, if not used immediately may be stored at room temperature (up to 25°C [77°F]) for no more than 4 hours from the time of preparation to the end of the infusion or refrigerated at 2°C to 8°C (36°F to 46°F) for no more than 24 hours. If refrigerated, allow the diluted solution to come to room temperature prior to administration. The diluted solution must be completely administered (including infusion time) within 4 hours once removed from refrigerator. Do not freeze or shake diluted solution.
Treatment of chronic graft-versus-host disease (cGVHD) after failure of ≥2 prior lines of systemic therapy (FDA approved in pediatric patients weighing ≥40 kg and adults).
Axatilimab may be confused with axitinib.
None known.
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program by clicking on the “Launch drug interactions program” link above.
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program
Efgartigimod Alfa: May decrease therapeutic effects of Fc Receptor-Binding Agents. Risk C: Monitor
Nipocalimab: May decrease therapeutic effects of Fc Receptor-Binding Agents. Risk C: Monitor
Rozanolixizumab: May decrease therapeutic effects of Fc Receptor-Binding Agents. Risk C: Monitor
Verify pregnancy status prior to initiating treatment in patients who could become pregnant.
Patients who could become pregnant should use effective contraception during therapy and for 30 days after the last dose of axatilimab.
Animal reproduction studies have not been conducted. Based on the mechanism of action, in utero exposure to axatilimab may cause fetal harm.
Axatilimab is a humanized monoclonal antibody (IgG4). Human IgG crosses the placenta. Fetal exposure is dependent upon the IgG subclass, maternal serum concentrations, placental integrity, newborn birth weight, and GA, generally increasing as pregnancy progresses. The lowest exposure would be expected during the period of organogenesis and the highest during the third trimester (Ref).
AST, ALT, alkaline phosphatase, CPK, amylase, lipase (baseline, every 2 weeks for first month, and every 4 to 8 weeks thereafter); bone growth and other growth parameters.
Axatilimab is a monoclonal antibody that binds to colony stimulating factor-1 receptors (CSF-1R) expressed on monocytes and macrophages. Blocking CSF-1R reduces the levels of these circulating proinflammatory and profibrotic monocytes and monocyte-derived macrophages and inhibits the activity of pathogenic macrophages in tissues.
Note: In pediatric patients ≥40 kg, pharmacokinetic parameters were similar to adult patients.
Distribution: Vd: 6.06 L.
Metabolism: Expected to be metabolized into small peptides via catabolic pathways.
Excretion: Total clearance: 0.07 L/hour.
