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Pharma Intelligence, Simplified

Data updated: Mar 10, 2026

NIKTIMVO

axatilimab-csfr Colony Stimulating Factor Receptor Type 1 (CSF-1R) Inhibitors
Approved 2024-08-14

NIKTIMVO (axatilimab-csfr) is a colony stimulating factor-1 receptor (CSF-1R) blocker indicated for the treatment of chronic graft-versus-host disease (cGVHD). It is specifically approved for use in adult and pediatric patients weighing at least 40 kg who have failed at least two prior lines of systemic therapy. This medication provides a therapeutic option for patients whose condition has not responded to previous systemic treatments.

Source: FDA Label • INCYTE CORP • Colony Stimulating Factor-1 Receptor Blocker

How NIKTIMVO Works

Axatilimab-csfr is a monoclonal antibody that binds to colony stimulating factor-1 receptors (CSF-1R) found on monocytes and macrophages. By blocking these receptors, the drug reduces the levels of circulating proinflammatory and profibrotic monocytes and monocyte-derived macrophages. This action inhibits the activity of pathogenic macrophages in tissues, addressing the biological drivers of the disease.

Source: FDA Label
1
Indication
--
Phase 3 Trials
1
Priority Reviews
1
Years on Market

Details

Status
Prescription
First Approved
2024-08-14
Routes
INTRAVENOUS
Dosage Forms
INJECTABLE

Companies

Active Ingredient: axatilimab-csfr

NIKTIMVO Approval History

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What NIKTIMVO Treats

1 indications

NIKTIMVO is approved for 1 conditions since its original approval in 2024. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Chronic Graft-versus-Host Disease
Source: FDA Label
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Active Pipeline

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Ongoing clinical trials by development phase

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Key Completed Trials

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Completed studies with published results, ranked by significance

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Trial Timeline

Full development history with FDA approval milestones

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

NIKTIMVO FDA Label Details

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Indications & Usage

FDA Label (PDF)

NIKTIMVO is indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. NIKTIMVO is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody indicated for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.

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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.