CurrentUnitedHealthcarePolicy 2026D0135C

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

Defines UnitedHealthcare coverage criteria for Niktimvo (axatilimab-csfr) for treatment of chronic graft-versus-host disease (cGVHD) in eligible commercial and individual exchange plan members, including initial and continuation authorization requirements.

Policy Summary

PayerUnitedHealthcare

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

Policy CodePolicy 2026D0135C

Change TypeNo material change

Effective DateApr 1, 2026

Next Review DateN/A

Key ActionPrior authorization is required with documentation of cGVHD after failure of at least two prior systemic therapies and weight ≥40 kg, prescribed by or in consultation with a transplant specialist.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

≥2Required prior lines

40 kgMin patient weight

12 moMax auth period

J9038HCPCS code

75%Pivotal trial ORR

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Coverage Criteria for Niktimvo (axatilimab-csfr)

Initial Therapy — Niktimvo is medically necessary when ALL of the following are met

Initial Therapy — Niktimvo is medically necessary when ALL of the following are met:

ALL of the following

- 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.
- cGVHD is classified as moderate or severe based on a recognized grading scale (e.g., NIH disease grade, CIBMTR score).
- Corticosteroid (e.g., prednisone, methylprednisolone).
- One of the following: bortezomib; ibrutinib (Imbruvica); an immunosuppressant agent (e.g., cyclosporine, mycophenolate, tacrolimus); ruxolitinib (Jakafi); belumosudil (Rezurock); or rituximab.
Patient weighs at least 40 kg.
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 months.

Continuation of Therapy — ALL required for reauthorization

Continuation of Therapy — ALL required for reauthorization:

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.
Reauthorization will be issued for no more than 12 months.

Unproven/Not Medically Necessary Indications

Unproven / Not Medically Necessary Indications:

ALL of the following

Niktimvo (axatilimab-csfr) is unproven and not medically necessary for prevention and/or treatment of all other indications due to insufficient evidence of efficacy.
This includes, but is not limited to, use for acute graft-versus-host disease (aGVHD).

Niktimvo is proven and medically necessary only for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy when the above criteria are met. Use of Niktimvo for prevention or treatment of other indications (including acute GVHD) is considered unproven and not medically necessary.

Summary: Niktimvo is indicated and covered for moderate to severe cGVHD in patients ≥40 kg who have failed at least two prior systemic therapies (including a corticosteroid and one additional agent as listed), when prescribed by or in consultation with a transplant specialist, dosed per FDA labeling, and authorized for up to 12 months. All other uses (e.g., prevention or treatment of acute GVHD) are unproven and not medically necessary.

Provider Requirements and Authorization Workflow

Prior Authorization

Prior Authorization Required

Prior authorization is required for Niktimvo (axatilimab-csfr). Submit documentation that supports the diagnosis of chronic graft‑versus‑host disease (cGVHD) and that the patient meets all initial‑therapy clinical criteria prior to initiation.

Affected product: Niktimvo (axatilimab-csfr)

Documentation Required

Required clinical documentation for initial authorization

Initial authorization requires complete clinical documentation demonstrating the diagnosis and severity of cGVHD per NIH consensus criteria and that the patient meets weight and prescriber requirements.

Documentation must include: evidence patient underwent allogeneic hematopoietic cell transplantation (HCT); clinical signs/symptoms consistent with NIH consensus criteria for cGVHD; classification of cGVHD as moderate or severe (e.g., NIH grade or CIBMTR score); patient weight (≥ 40 kg); and documentation that Niktimvo is prescribed by or in consultation with a transplant specialist.
Dosing must follow FDA‑approved labeling.

Note

Authorization duration and reauthorization requirements

Authorization for Niktimvo is for a maximum of 12 months. Reauthorization requires documentation of continued clinical benefit.

Initial authorization duration: up to 12 months.
Reauthorization: documentation of positive clinical response to Niktimvo, continued prescribing by or in consultation with a transplant specialist, and dosing consistent with FDA labeling; reauthorizations issued for up to 12 months.

Step Therapy

Required prior systemic therapy failure

For initial approval, there must be documented failure of prior systemic therapies: failure of a corticosteroid plus at least one additional systemic agent from the listed options.

Documented history of failure with a corticosteroid (e.g., prednisone, methylprednisolone) AND one of: bortezomib; ibrutinib (Imbruvica); an immunosuppressant agent (e.g., cyclosporine, mycophenolate, tacrolimus); ruxolitinib (Jakafi); belumosudil (Rezurock); or rituximab.
Lack of documented failure of the required prior therapies may result in denial of coverage.

Denial Risk

Documentation and denial risk

Ensure all required elements are included with the prior authorization request to avoid denial or delay: diagnosis per NIH consensus, severity classification, prior therapy failure documentation, patient weight, transplant specialist involvement, and planned dosing per FDA labeling.

Missing documentation of any required element (diagnosis, NIH signs, severity, prior therapy failures, weight ≥40 kg, transplant specialist prescriber/consultation, FDA‑consistent dosing) increases risk of denial.

Regimen	Indication	Dosing / Schedule	Coverage status
Niktimvo (axatilimab-csfr) IV
Chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in patients weighing ≥40 kg
Approved dose used in AGAVE-201: 0.3 mg/kg administered intravenously every 2 weeks until disease progression, lack of efficacy by 9 months, or unacceptable toxicity
Coverage with criteria — prior authorization required; see policy for required documentation, prior therapy requirements, and authorization duration (up to 12 months)

Key Definitions

inv-15: cGVHD — definition per NIH consensus criteria in post-allogeneic HCT patient with clinical signs and severity classification

DefinitionChronic graft-versus-host disease (cGVHD) diagnosed per NIH consensus criteria in a patient who has undergone allogeneic hematopoietic cell transplantation (HCT), with clinical signs and symptoms that are components of the NIH consensus criteria and disease classified as moderate or severe.

Required transplant historyPatient has undergone allogeneic hematopoietic cell transplantation (HCT).

Diagnostic criteriaClinical symptoms and signs consistent with the NIH consensus criteria for diagnosis of cGVHD (assessment identifies affected organ systems, extent and severity).

Severity classificationcGVHD must be classified as moderate or severe based on NIH disease grade or comparable grading (e.g., CIBMTR score).

Background and Rationale

Chronic graft‑versus‑host disease (cGVHD) is a multisystem complication that occurs following allogeneic hematopoietic cell transplantation. In this policy context, Niktimvo is intended for treatment of cGVHD in patients who meet specified clinical criteria; it is not approved for prevention or treatment of other indications such as aGVHD. Per the policy instructions for use, patients considered for Niktimvo should weigh at least 40 kg, have therapy prescribed by or in consultation with a transplant specialist, and receive dosing consistent with the FDA‑approved labeling. Authorizations are limited to a maximum of 12 months for initial and reauthorization requests, and continuation requires documentation of a positive clinical response.

Policy Summary

PayerUnitedHealthcare

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

Policy CodePolicy 2026D0135C

Change TypeNo material change

Effective DateApr 1, 2026

Next Review DateN/A

SourceLink

On This Page

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

OpenPayer

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

Coverage Criteria for Niktimvo (axatilimab-csfr)

Coding and Billing References

Provider Requirements and Authorization Workflow

Dosing and Regimens

Position in Treatment Pathway

Key Definitions

Background and Rationale