CurrentUnitedHealthcarePolicy CS2025D0135D

Niktimvo (axatilimab-csfr) for chronic graft-versus-host disease

Defines UnitedHealthcare Medical Benefit Drug Policy coverage criteria for Niktimvo (axatilimab-csfr) for treatment of chronic graft-versus-host disease (cGVHD), including initial and continuation authorization criteria, applicable codes, exclusions, and state-specific applicability exceptions.

Policy Summary

PayerUnitedHealthcare

PolicyNiktimvo (axatilimab-csfr) for chronic graft-versus-host disease

Policy CodePolicy CS2025D0135D

Change TypeApplication update; formatting correction

Effective DateAug 1, 2025

Next Review Date

Key ActionObtain prior authorization and document prior corticosteroid use and failure of required prior agents; ensure dosing follows FDA labeling and involve a transplant specialist.

POLICY UPDATE CHANGES

Application section updated to add Louisiana instruction to refer to state's Medicaid clinical policy and to indicate the policy does not apply to Texas; refer to Texas Medicaid Provider Procedures Manual.

Corrected formatting issue in References section.

1Indication covered

>=40 kgMinimum patient weight

12Max auth duration (months)

5Prior therapies required (examples)

Coverage Summary

Defines UnitedHealthcare Medical Benefit Drug Policy coverage criteria for Niktimvo (axatilimab-csfr) for treatment of chronic graft-versus-host disease (cGVHD). Coverage stance: covered_with_criteria. Effective date: 2025-08-01; Last review: 2025-09-01. Policy subject: Niktimvo (axatilimab-csfr) for chronic graft-versus-host disease; status: CURRENT.

Surface thresholds and key stats

Weight threshold>= 40 kg

Authorization duration (initial & reauthorization)<= 12 months per authorization / reauthorization

Minimum patient weight (stat)>=40 kg

Max authorization duration (stat)12 months

Prior therapy examples required (stat)At least one of: ibrutinib, bortezomib, ruxolitinib, rituximab, or belumosudil (5 examples listed)

Initial Therapy Criteria (Medically Necessary)

Medically Necessary (Initial Therapy)

Niktimvo is medically necessary for treatment of chronic graft-versus-host disease (cGVHD) when ALL of the following are met:

ALL of the following

cGVHD diagnostic requirements
- Patient has undergone allogeneic hematopoietic cell transplantation (HCT)
- Clinical symptoms and signs that are components of the National Institutes of Health (NIH) consensus criteria for diagnosis of cGVHD
Prior systemic therapy failure
- ALL of the following
  - At least one of the listed additional agents

Continuation Therapy Criteria (Reauthorization)

Medically Necessary (Continuation of Therapy / Reauthorization)

For continuation of therapy, ALL of the following must be met:

ALL of the following

Authorization will be issued for no more than 12 months<= 12 months per authorization / reauthorization
Documentation of a positive clinical response to Niktimvo therapy
Niktimvo dosing is in accordance with the United States Food and Drug Administration approved labeling
Prescribed by or in consultation with a transplant specialist

Unproven / Not Medically Necessary

Not Medically Necessary / Unproven

Niktimvo is unproven and not medically necessary for prevention and/or treatment of all other indications including, but not limited to:

ANY of the following

Acute graft-versus-host disease (aGVHD)
Any other indication not listed in the medically necessary criteria due to insufficient evidence of efficacy

Applicable Codes

Drug HCPCSHCPCS

J9038

Injection, axatilimab-csfr; 0.1 mg

Diagnosis Codes (relevant)ICD-10

D89.811	Chronic graft-versus-host disease
D89.812	Acute on chronic graft-versus-host disease

Provider Actions & Billing Notes

Note

Prior authorization, documentation, and dosing requirements

Actionable requirements for prescribing and authorizing Niktimvo.

Prior authorization required; initial and continuation approvals limited to no more than 12 months (Affected code: J9038)
Document prior corticosteroid use and history of failure with required agents (ibrutinib, bortezomib, ruxolitinib, rituximab, belumosudil) and document positive clinical response for reauthorization
Dosing must follow FDA-approved labeling (Affected code: J9038)

Prescribed by or in consultation with a transplant specialist is required for both initial and continuation authorization; this requirement is specified in the clinical criteria and prior authorization/provider action instructions.

Clinical Evidence

75%ORR within first 6 months (n=79) at 0.3 mg/kg q2w

1.5 moMedian time to response

60%Maintained response at 12 months (Kaplan-Meier)

1.9 moMedian duration of response (95% CI: 1.6, 3.5)

Niktimvo is indicated for patients with chronic graft-versus-host disease (cGVHD), a multisystem disorder after allogeneic hematopoietic cell transplantation assessed by NIH consensus criteria. The policy notes the indication follows FDA approval for use after failure of at least two lines of systemic therapy in patients weighing at least 40 kg.

Background & Definitions

Niktimvo (axatilimab-csfr) 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 lines of systemic therapy in patients weighing at least 40 kg.

cGVHD is a multisystem disorder that arises after allogeneic hematopoietic cell transplantation and is assessed by the National Institutes of Health (NIH) consensus criteria; treatment goals include reducing symptom burden and disease activity. Clinical trial data (AGAVE-201) supporting Niktimvo demonstrated a 75% overall response rate within the first six months at the approved dosing regimen.

Term	Definition
cGVHD	Chronic graft-versus-host disease, diagnosed per NIH consensus criteria after allogeneic hematopoietic cell transplantation.
FDA-approved labeling	Dosing and administration instructions as specified in the U.S. Food and Drug Administration approved package insert for Niktimvo.

Revision History

2025-08-01revisionLatest

Revision archived previous policy version CS202500135C and updated Application to add Louisiana instruction to refer to the state's Medicaid clinical policy and to indicate the policy does not apply to Texas (refer to Texas Medicaid Provider Procedures Manual).

2025-09-01formatting_correction

Corrected formatting issue in References section.