Axatilimab-Csfr
Axatilimab-csfr is an investigational monoclonal antibody targeting the colony stimulating factor-1 receptor (CSF-1R). It is being studied for the treatment of chronic graft-versus-host disease (cGVHD) in patients who have not responded adequately to prior therapies. By inhibiting CSF-1R signaling, it aims to modulate the immune cells involved in GVHD pathophysiology.
Axatilimab-csfr is an experimental monoclonal antibody being developed to treat chronic graft-versus-host disease by targeting immune cells through CSF-1R inhibition.
Key facts at a glance
- RxNorm
- 2691528
- Generic
- Axatilimab-Csfr
- Class
- pin-variant
- Brands
- Niktimvo
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
10 groupspregnancy
first trimesteravoid if possible
use only if potential benefits outweigh potential risks to fetus; discuss with healthcare provider
regular fetal monitoring recommended
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
use only if potential benefits outweigh potential risks to fetus
regular fetal monitoring and ultrasound assessments
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
use only if potential benefits outweigh potential risks; consider timing of last dose before delivery
regular fetal monitoring; assess need for vaccination of newborn
Use with caution and monitor for adverse effects.
lactation
breastfeedingnot recommended
breastfeeding not recommended during treatment and for at least 6 months after last dose
monitor infant for signs of infection or immune effects
Use with caution and monitor for adverse effects.
geriatric
elderly over 65no specific adjustment
no dose adjustment required; monitor for adverse effects
routine monitoring of renal and hepatic function
pediatric_age
adolescents 12 17weight-based dosing
approved for use in pediatric patients 12 years and older with cGVHD; follow pediatric dosing guidelines
monitor growth, development, and immune function
Use with caution and monitor for adverse effects.
renal_impairment
mild to moderateno adjustment needed
no dose adjustment required for mild to moderate renal impairment
routine renal function monitoring
renal_impairment
severeuse with caution
limited data in severe renal impairment; use with caution
close monitoring of renal function and adverse effects
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno adjustment needed
no dose adjustment required for mild hepatic impairment
routine hepatic function monitoring
hepatic_impairment
moderate to severeuse with caution
limited data in moderate to severe hepatic impairment; use with caution
close hepatic function monitoring
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval for treatment of chronic graft-versus-host disease
Drug class
Biologic therapeutic antibody targeting CSF-1R
Therapeutic indication
Approved for cGVHD after failure of at least two prior lines of systemic therapy
Mechanism of action
Inhibits colony-stimulating factor 1 receptor to modulate macrophage biology
Axatilimab-csfr is an investigational agent being evaluated for chronic graft-versus-host disease (cGVHD), particularly in patients who have failed at least two prior lines of systemic therapy.
This monoclonal antibody binds to the colony stimulating factor-1 receptor (CSF-1R) on target cells, blocking CSF-1 ligand binding and inhibiting downstream signaling. This modulation affects macrophage and monocyte populations implicated in immune-mediated tissue damage characteristic of cGVHD.
As an investigational agent, axatilimab-csfr is not FDA-approved. Clinical trials are ongoing to evaluate its safety and efficacy. Common adverse events in studies have included transaminase elevations, fatigue, and musculoskeletal pain. Patients should only receive this medication through clinical trial participation or expanded access programs under physician supervision.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed August 24, 2026. · Read our editorial policy