CurrentUnitedHealthcarePolicy CSOH2O2500135.A

Niktimvo (Axatilimab-Csfr) (for Ohio Only)

UnitedHealthcare Ohio-only medical benefit drug policy defining coverage criteria for Niktimvo (axatilimab-csfr) for treatment of chronic graft-versus-host disease (cGVHD), continuation/reauthorization criteria, exclusions for other indications, and applicable HCPCS/ICD-10 codes.

Policy Summary

PayerUnitedHealthcare

PolicyNiktimvo (Axatilimab-Csfr) (for Ohio Only)

Policy CodePolicy CSOH2O2500135.A

Change TypeNew policy

Effective DateJun 1, 2025

Next Review Date

Key ActionAuthorization must document diagnosis of cGVHD, failure of at least two prior systemic therapies, patient weight ≥40 kg, dosing per FDA label, and prescriber or consult with a transplant specialist; authorizations issued for up to 12 months.

POLICY UPDATE CHANGES

New Medical Benefit Drug Policy created effective 06/01/2025.

1Covered Indication (cGVHD)

>=40 kgMinimum patient weight

12Max authorization (months)

Coverage Summary

UnitedHealthcare Ohio-only medical benefit drug policy (Policy Number: CSOH2O2500135.A, Effective Date: 2025-06-01) establishes coverage for Niktimvo (axatilimab-csfr) as covered with criteria for the treatment of chronic graft-versus-host disease (cGVHD). The policy defines specific medical necessity criteria including prior authorization requirements, documentation of prior systemic therapy failures (at least two prior lines), patient weight threshold, dosing consistent with the FDA label, transplant specialist involvement, and authorization duration limits.

Initial Therapy Criteria (Medically necessary)

Medically necessary (initial therapy)

Niktimvo is medically necessary for treatment of cGVHD when ALL of the following are met:

ALL of the following

cGVHD definition components
- Patient has undergone allogenic hematopoietic cell transplantation (HCT)
- cGVHD is classified as moderate or severe based on grading scale (e.g., NIH disease grade, CIBMTR score)
- Clinical symptoms and signs that are components of the National Institutes of Health (NIH) consensus criteria for diagnosis of cGVHD
Patient weighs at least 40 kg>= 40 kg
Niktimvo dosing is in accordance with the United States Food and Drug Administration approved labeling
Prescribed by or in consultation with a transplant specialist
Prior therapy requirements
- Patient has history of failure with both of the following:
- At least two prior line examples (ONE of the following listed agents may constitute lines of therapy; policy lists these as examples to meet prior therapy requirement)
  - Imbruvica (ibrutinib)
  - Bortezomib
  - Immunosuppressant agent (e.g., cyclosporine, mycophenolate, tacrolimus)
  - Rezurock (belumosudil)
  - Jakafi (ruxolitinib)

Continuation / Reauthorization Criteria

Medically necessary (continuation / reauthorization)

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

ALL of the following

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

Provider Actions & Requirements

Prior Authorization

Prior authorization required

Authorization must document diagnosis of chronic graft-versus-host disease (cGVHD); history of failure with at least two prior systemic therapies; patient weight >= 40 kg; dosing per the FDA-approved labeling; and that the prescriber is a transplant specialist or that there is transplant specialist consultation. Authorizations will be issued for up to 12 months.

J9038
D89.811
D89.812

Documentation Required

Documentation of prior therapies and response

Document failure of prior lines of systemic therapy. Examples of prior systemic therapies to document include ibrutinib, bortezomib, cyclosporine, mycophenolate, tacrolimus, belumosudil, ruxolitinib, and rituximab. For continuation/reauthorization, document a positive clinical response to Niktimvo.

Billing Rule

Use specified HCPCS code

Report axatilimab-csfr using HCPCS code J9038 (Injection, axatilimab-csfr, 0.1 mg).

Clinical Evidence

75%Overall response rate within first 6 months (AGAVE-201, N=79)

1.5 moMedian time to response

60%Proportion of responders maintaining response at 12 months (Kaplan-Meier)

The approval and coverage rationale are supported by AGAVE-201, a clinical study described in the policy involving N = 79 adult and pediatric patients with recurrent or refractory cGVHD who had received at least two prior systemic therapies; these trial results (including a 75% overall response rate within the first six months at the approved dose) underpin the evidence supporting Niktimvo's approval and coverage.

Background & Definitions

Chronic graft-versus-host disease (cGVHD) is a multisystem disorder that can occur after allogeneic hematopoietic cell transplantation (HCT); severity is classified as mild, moderate, or severe and is assessed using NIH consensus criteria to guide therapy. Niktimvo is a colony-stimulating factor-1 receptor (CSF-1R)–blocking antibody indicated for treatment of cGVHD after failure of at least two prior lines of systemic therapy in patients weighing at least 40 kg. The policy requires that patients have undergone allogeneic HCT and that cGVHD be classified (e.g., NIH disease grade or CIBMTR score) as part of the coverage criteria.

Term	Definition
cGVHD	Chronic graft-versus-host disease diagnosed per NIH consensus criteria and graded (mild, moderate, severe) by NIH disease grade or CIBMTR score.
Failure of prior therapy	History of inadequate response or intolerance to prior systemic therapies; policy requires failure with at least two prior lines of systemic therapy.