CurrentUnitedHealthcarePolicy CS2026D0135E

Niktimvo (Axatilimab-Csfr) Medical Benefit Drug Policy

Defines UnitedHealthcare Medical Benefit Drug Policy coverage and requirements for Niktimvo (axatilimab-csfr) use in chronic graft-versus-host disease (cGVHD), including initial and continuation criteria, applicability by state, coding, dosing alignment with FDA label, and exclusions for other indications.

Policy Summary

PayerUnitedHealthcare

PolicyNiktimvo (Axatilimab-Csfr) Medical Benefit Drug Policy

Policy CodePolicy CS2026D0135E

Change TypeApplication / supporting information revised

Effective DateApr 1, 2026

Next Review Date

Key ActionSubmit prior authorization documenting moderate/severe cGVHD per NIH criteria, prior failure of corticosteroid plus one additional listed agent, weight ≥ 40 kg, and that prescribing is by or in consultation with a transplant specialist.

POLICY UPDATE CHANGES

Application section updated to remove Louisiana and adjust state-specific guidance (Indiana, Ohio, supporting information updated).

1Indication covered (cGVHD)

>=40 kgMinimum patient weight

12 monthsMax authorization duration

Coverage Summary

This UnitedHealthcare Medical Benefit Drug Policy defines coverage and requirements for Niktimvo (axatilimab-csfr) for chronic graft-versus-host disease (cGVHD), including initial and continuation criteria, linkage to the FDA-approved dosing/labeling, applicability by state (see state-specific guidance), coding, and explicit exclusion of use for other indications.

Initial Therapy (Medically Necessary) Criteria

Initial Therapy (Medically Necessary)

Covered when ALL of the following are met:

ALL of the following

ALL of the following
- Patient has undergone allogenic 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 grading scale (e.g., NIH disease grade, CIBMTR score)
ALL of the following
- Corticosteroid (e.g., prednisone, methylprednisolone)
- AND one of the following
  - Bortezomib
  - Imbruvica (ibrutinib)
  - Immunosuppressant agent (e.g., cyclosporine, mycophenolate, tacrolimus)
  - Jakafi (ruxolitinib)
  - Rezurock (belumosudil)
  - Rituximab
Patient weighs at least 40 kg>=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 for initial approval

Continuation of Therapy (Reauthorization) Criteria

Continuation of Therapy (Reauthorization)

Covered when ALL of the following are 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
Reauthorization will be issued for no more than 12 months

Not Medically Necessary / Unproven

Not covered when ANY of the following apply:

ANY of the following

Use of Niktimvo for prevention and/or treatment of indications other than chronic graft-versus-host disease (cGVHD), including but not limited to acute graft-versus-host disease (aGVHD)
Considered unproven and not medically necessary due to insufficient evidence of efficacy

Provider Actions & Authorization

Prior Authorization

Prior authorization required with documentation

Initial requests must document diagnosis of moderate or severe chronic graft-versus-host disease per NIH criteria, prior failure of a corticosteroid plus at least one additional listed agent, patient weight of at least 40 kg, and that the therapy is prescribed by or in consultation with a transplant specialist. Authorization will be issued for up to 12 months.

HCPCS: J9038
Authorization issued for up to 12 months

Prior Authorization

Reauthorization requirements

For continuation of therapy, submit documentation of a positive clinical response to Niktimvo and confirm dosing is in accordance with the FDA-approved labeling. Reauthorization will be issued for up to 12 months.

HCPCS: J9038
Reauthorization issued for up to 12 months

Documentation Required

Specialist involvement

Therapy must be prescribed by or in consultation with a transplant specialist; retain documentation of the consultation in the medical record.

Clinical Evidence Summary

75%ORR within first 6 months (n=79)

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)

AGAVE-201 was a randomized, open-label study in adults and pediatric patients with recurrent or refractory cGVHD who had received at least two prior lines of systemic therapy; patients received Niktimvo at the approved dose of 0.3 mg/kg every two weeks, and the results (75% ORR within the first six months at this dose, n=79) are cited in the policy as the clinical evidence supporting coverage for the specified population.

Background

Chronic graft-versus-host disease (cGVHD) is a multisystem disorder that can develop after allogeneic hematopoietic cell transplantation (HCT) and is delineated by NIH consensus criteria into mild, moderate, or severe, which guides initial management and therapy. Niktimvo is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody indicated for the treatment of cGVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.

cGVHD — a multisystem disorder after allogeneic HCT defined by NIH consensus criteria.

Failure — history of inadequate response or intolerance to prior listed therapy (corticosteroid plus at least one other agent) as required by policy (exact clinical definition per treating clinician and documentation).