# Axatilimab

Source: https://onco.cc/drugs/axatilimab/  
OnCo record `axatilimab` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Axatilimab targets an immune-cell signal used by macrophages. It is approved for chronic graft-versus-host disease and is being tested with olaparib in advanced breast cancer.

## Summary

FDA approval on 2024-08-14 is for chronic graft-versus-host disease after failure of at least two prior systemic treatments in adults and children weighing at least 40 kg. This is not a breast-cancer approval. The June 2026 label describes CSF1R blockade and monitoring for infusion reactions and laboratory abnormalities. The recruiting phase 1b olaparib combination, NCT06488378, studies metastatic or unresectable HER2-negative breast cancer with qualifying BRCA1/2 or germline PALB2 alterations; a history of pneumonitis or ILD, or baseline evidence of either, is excluded. No comparative or postoperative efficacy is established.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-10-10
- Also known as: axatilimab-csfr
- Brand: Niktimvo
- Modality: Monoclonal antibody
- Mechanism: Blocks colony-stimulating factor 1 receptor (CSF1R), affecting CSF1-dependent monocytes and macrophages. Its breast-cancer use with olaparib is investigational.
- Approvals: US 2024: Chronic graft-versus-host disease after failure of at least two prior systemic lines in adults and pediatric patients weighing at least 40 kg

## Sources

- FDA approval and indication: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-axatilimab-csfr-chronic-graft-versus-host-disease
- Current Niktimvo label (DailyMed): https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=bb6dba23-e7a6-4765-a1e6-b9e277ce0381
- Investigational breast combination NCT06488378: https://clinicaltrials.gov/study/NCT06488378

## Connected records

- cancers: [Breast cancer (all types)](https://onco.cc/cancers/breast-cancer/)
- targets: [CSF1R](https://onco.cc/targets/csf1r/)
- companies: [Incyte](https://onco.cc/companies/incyte/), [Syndax Pharmaceuticals](https://onco.cc/companies/syndax/)
- trials: [Axatilimab and olaparib in BRCA/PALB2-associated metastatic breast cancer](https://onco.cc/trials/nct06488378/)

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