Marnetegragene autotemcel (Kresladi) gene therapy
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Coverage and utilization management criteria for marnetegragene autotemcel (Kresladi) for treatment of severe leukocyte adhesion deficiency‑I (LAD‑I) and related authorization requirements affecting commercial, ICHRA, and Medicaid lines of business.
Criteria updated per FDA labeling: removed minimum age requirement, removed option for those with an HLA-matched sibling donor, and updated dose requirement; added ICHRA line of business and PDAC language.
Policy requires Precision Drug Action Committee (PDAC) Utilization Management Review for all requests.
Approval duration specified as single infusion lifetime with initial approval duration: 3 months.
Drug is now FDA approved - criteria updated per FDA labeling: removed minimum age requirement, removed option for those with an HLA-matched sibling donor, and updated dose requirement.
Coverage Criteria
Initial Therapy (LAD-I) - Covered Criteria
Covered when ALL of the following are met for Leukocyte Adhesion Deficiency Type 1 (LAD‑I):
Approval duration: 3 months (one time infusion per lifetime)
Updated Coverage Criteria (summary)
Criteria updated per FDA labeling (details summarized where present)
Full clinical criteria text not included in this document portion; refer to other policy sections for complete eligibility rules.
Non‑FDA approved indications that are not specifically addressed in this policy are not authorized unless there is adequate supporting documentation of efficacy and safety per the applicable off‑label or non‑formulary policies referenced herein (for example: CP.CPA.09 for Commercial, HIM.PA.154 for ICHRA, and CP.PMN.53 for Medicaid).
This portion of the document does not list any explicit clinical exclusions beyond standard applicability notes; no additional clinical exclusion statements are provided here.
Re‑authorization or continuation therapy is not permitted because Kresladi is indicated to be administered as a single dose for the lifetime of the patient.
Within the sections referenced, there are no explicit statements
Initial Therapy Criteria
Initial Therapy (LAD-I) - Criteria Details
Initial therapy coverage criteria for LAD‑I
Approval duration: 3 months
Initial therapy (summary)
Initial therapy rules updated per FDA approval
Exact dosing and eligibility details are contained in the Initial Therapy criteria (minimum 2.8 x 10^6 CD34+ cells/kg; single infusion per lifetime).
Continuation / Re-authorization Criteria
Continuation Therapy
Continuation/Re-authorization rules
Approval duration: Not applicable
Provider Actions & Authorization Requirements
Prior authorization and PDAC review required
Prior authorization is required for all requests for marnetegragene autotemcel (Kresladi). Every request must undergo Precision Drug Action Committee (PDAC) Utilization Management Review and include supporting documentation that demonstrates the member meets the approval criteria.
- All requests require PDAC review per CC.PHAR.21.
- Submit clinical documentation supporting each approval criterion (see coverage criteria).
Prior authorization governed by Health Plan policy
Prior authorization decisions are governed by this clinical policy (CP.PHAR.599) together with the Health Plan's administrative procedures; the policy text was updated following FDA approval and labeling-based changes.
- Refer to policy CP.PHAR.599 and Health Plan procedures for submission requirements and process.
- RT4 revision note documents alignment to FDA labeling and administrative updates.
Follow off‑label/formulary routing for non‑listed uses
If the requested use is not listed in section III (Diagnoses/Indications for which coverage is NOT authorized), route the request per the Health Plan's off‑label or formulary/non‑formulary policies referenced in this policy.
- For off‑label uses, follow CP.CPA.09 (commercial), HIM.PA.154 (ICHRA), or CP.PMN.53 (Medicaid) as applicable.
- If a recent label change (<6 months) is not reflected, follow the no-coverage or non‑formulary policies listed in section I.B.
No step therapy specifics in this document portion
This document portion does not specify any step therapy requirements for Kresladi; therefore no step therapy specifics are present here.
- If step/coverage guidance is needed for unlisted uses or recent label changes, follow the referenced formulary/off‑label policies per section I.B.
Submit required clinical documentation
Providers must submit supporting clinical documentation — such as office/chart notes, laboratory results, or other clinical information — that demonstrates the member meets all approval criteria.
- Include documentation showing diagnostic confirmation (e.g., flow cytometry results, genetic testing) and specialist attestation as required by the coverage criteria.
- Attach evidence of prior significant infections or family history when applicable.
Follow Health Plan documentation and benefit terms
Providers must follow applicable Health Plan documentation requirements, benefit terms, and state Medicaid provisions where relevant; state Medicaid rules take precedence if they conflict with this clinical policy.
- For Medicaid members, consult the state Medicaid manual for any differing coverage provisions.
- Ensure submissions conform to the Health Plan's administrative procedures and evidence requirements.
Requests not meeting coverage criteria may be denied
Requests that do not meet the listed coverage criteria for LAD‑I — or requests for non‑FDA indications that are not supported by the applicable off‑label or non‑formulary policies — may be denied.
- Coverage criteria include diagnostic confirmation, specialist prescribing/consultation, infection or family history, no HLA‑matched sibling donor, transplant specialist attestation, and minimum dose of 2.8 x 10^6 CD34+ cells/kg.
- Non‑FDA uses must be supported by the off‑label policies cited in section I.B to avoid denial.
Denial risk if inconsistent with updated labeling or medical necessity criteria
Denials may occur if coverage decisions are inconsistent with updated FDA‑approved labeling or the Health Plan's medical necessity criteria following the RT4 revisions.
- RT4 noted FDA approval and criteria updates (e.g., dosing, age, HLA‑matched sibling donor option); ensure requests align with the current labeling and policy.
Coding References
Step Therapy & Formulary Routing
| Step | Policy Instruction |
|---|---|
| 1 | If the requested use is not listed in Section III and the drug has undergone a label change within the last 6 months, follow the referenced formulary/no‑coverage or non‑formulary policies for the applicable line of business as specified in this policy (see guidance options a and b). |
| Step | Requirement |
|---|---|
| 1 | No step therapy requirements are specified in this document portion. |
Quantity Limits
Site of Care
Hospital outpatient — IV single infusion (one or two bags)
Administration is an intravenous infusion per product availability; the dose is provided as a single infusion of cell suspension in cryopreservation solution, shipped in one or two bags.
- Maximum: one dose per lifetime.
- Minimum dose requirement: 2.8 x 10^6 CD34+ cells/kg (see dosing appendix).
No site‑of‑care rules specified
No site‑of‑care restrictions or additional site rules are specified in this portion of the policy.
Background
Marnetegragene autotemcel (Kresladi) is an autologous hematopoietic stem cell‑based gene therapy that delivers a functional ITGB2 gene to patients with severe leukocyte adhesion deficiency‑I (LAD‑I). The FDA accelerated approval is based on biologic activity measures such as increased neutrophil surface expression of CD18 and CD11a/CD11b, and continued approval may depend on confirmatory trials.
Definitions
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