CurrentBlue Cross Blue Shield - IowaPolicy 05.04.84

Skysona (elivaldogene autotemcel) — coverage criteria for cerebral adrenoleukodystrophy (CALD)

Policy governs prior authorization, coverage criteria, and clinical use of Skysona for pediatric patients with early, active cerebral adrenoleukodystrophy (CALD) under Wellmark/Blue Cross Blue Shield Iowa benefit plans.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicySkysona (elivaldogene autotemcel) — coverage criteria for CALD

Policy CodePolicy 05.04.84

Change TypeRoutine review — no material changes

Effective DateJul 25, 2025

Next Review DateJun 1, 2025

Key ActionSubmit prior authorization with documentation of ABCD1 mutation, elevated VLCFA, Loes score 0.5–9 with gadolinium enhancement, and NFS ≤ 1.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4-17 yrsFDA-indicated patient age range

0.5–9Loes score eligibility

NFS ≤1Neurologic function score

1 lifetimeQuantity limit

15 yearsLong-term safety monitoring

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J3387Assigned HCPCS code

Coverage Criteria for Skysona (elivaldogene autotemcel)

Initial Approval — Covered when ALL of the following are met

Covered when ALL of the following are met:

Initial Approval Criteria for Skysona: Member is 4–17 years of age at time of infusion; documented diagnosis of active cerebral X-linked adrenoleukodystrophy (CALD) defined by ALL of the following: (a) confirmed ABCD1 gene mutation; (b) elevated very-long-chain fatty acids (VLCFA) per the performing laboratory's reference range; and (c) presence of active CNS disease with Loes score between 0.5 and 9 (inclusive) AND gadolinium enhancement on MRI of demyelinating lesions; documented neurologic function score (NFS) ≤ 1; and member has NONE of the following exclusion criteria: history of prior gene therapy or allogeneic hematopoietic stem cell transplant; peripheral blood absolute neutrophil count (ANC) < 1500 cells/mm3; platelet count < 100,000 cells/mm3; uncorrected bleeding disorder; hemoglobin < 10 g/dL; AST or ALT > 2.5 x ULN; total bilirubin > 3.0 mg/dL (except Gilbert's syndrome if otherwise stable).

Continuation/Repeat Therapy — Not covered / Investigational

Not covered/Investigational:

Repeat Treatment: Repeat treatment of Skysona for any indication is considered investigational and is not covered because safety and efficacy beyond a single dose have not been established; approvals are limited to one lifetime treatment.

Coverage is limited to a single lifetime administration of Skysona; repeat dosing is considered investigational due to insufficient evidence of safety and efficacy beyond one dose. Requests will be evaluated against required baseline clinical and laboratory criteria — patients with the specified hematologic, hepatic, renal, cardiac, infectious, prior-gene-therapy/HSCT, or familial cancer-syndrome findings are excluded from coverage. Examples of exclusionary laboratory/clinical conditions include: ANC < 1500 cells/mm3, platelets < 100,000 cells/mm3, hemoglobin < 10 g/dL, AST/ALT > 2.5 × ULN, total bilirubin > 3.0 mg/dL (except Gilbert’s syndrome), LVEF < 40%, eGFR < 70 mL/min/1.73 m2 or creatinine clearance < 50 mL/min, and any clinically significant uncontrolled active infection. Authorization, when criteria are met, may be granted for a one-time treatment and approvals will be limited to one lifetime treatment.

Coverage is not provided for patients who have one or more Major Functional Disabilities (MFD). MFDs include loss of communication, cortical blindness, requirement of tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement; the presence of any of these denotes advanced disease status and is exclusionary for Skysona.

Skysona is considered not medically necessary for any patient who does not meet the policy’s initial approval criteria (including age, confirmed ABCD1 mutation and elevated VLCFA, MRI evidence of active CNS disease with Loes score within the required range and gadolinium enhancement, NFS ≤ 1, and acceptable baseline labs and cardiac/renal assessments). In addition, repeat treatment of Skysona for any indication is considered investigational and therefore not medically necessary because safety and efficacy beyond a single dose have not been established. Approvals, when granted, are limited to one lifetime treatment.

Coding, Scoring, and Dose Requirements

Procedures and billing codesHCPCS

J3387	Injection, elivaldogene autotemcel, per treatment (effective 1/1/26)
C9399	Unclassified drugs or biologicals
J3590	Unclassified biologicals

Loes score — eligibility

Required MRI Loes score rangeLoes score between 0.5 and 9 (inclusive) on the 34-point scale

Documentation sourceActive CNS disease must include Loes 0.5–9 AND gadolinium enhancement on MRI per prior auth requirements

Scale maximum referenceLoes scoring uses a 34-point scale (see Loes components)

Neurologic Function Score (NFS) — threshold

Required NFS thresholdNeurologic Function Score (NFS) ≤ 1

Population describedAsymptomatic or mildly symptomatic boys with NFS ≤ 1 qualify as early, active CALD per FDA indication

Provider Requirements, Authorization, and Billing Guidance

Prior Authorization

Prior Authorization and Approval Duration

Prior Authorization Required: Prior authorization is required for Skysona (elivaldogene autotemcel). Authorization may be granted as a one-time treatment for a duration of 3 months when initial approval criteria are met. Approvals are limited to one treatment per lifetime.

Prior authorization required and approval duration: one-time treatment authorization for 3 months; Quantity limit: one treatment per lifetime.

Billing Rule

Drug Coding and Reporting

Provider must report drug administration using the appropriate HCPCS/alpha‑numeric codes. Effective 2026-01-01 report elivaldogene autotemcel with J3387. Until that effective date or if an appropriate product-specific code is not available, report using unclassified drug/biological codes as appropriate.

Background and Clinical Context

Cerebral adrenoleukodystrophy (CALD) is an X‑linked, childhood‑onset form of adrenoleukodystrophy caused by mutations in the ABCD1 gene that lead to accumulation of very‑long‑chain fatty acids (VLCFA) and progressive central nervous system demyelination. Early, active CALD presents in boys with minimal or no neurologic impairment but with MRI evidence of inflammatory demyelination; disease progression results from VLCFA‑mediated neuroinflammation and loss of myelin, ultimately producing severe neurologic dysfunction if untreated. Skysona is an autologous CD34+ cell gene therapy (Lenti‑D) that introduces functional ABCD1 cDNA into hematopoietic stem cells to restore ALDP expression and address the underlying biochemical defect.

Definitions and Scoring Systems

Early, active CALD — definition

Definition of early, active CALDAsymptomatic or mildly symptomatic boys with NFS ≤ 1, gadolinium enhancement on brain MRI, and Loes score between 0.5 and 9

Clinical elements requiredMust have confirmed ABCD1 mutation and elevated VLCFA in addition to MRI and NFS findings for active disease designation

FDA indication contextThis population (boys 4–17 years) is the FDA‑approved indication for Skysona to slow neurologic progression

Major Functional Disability (MFD) — definition

Definition of Major Functional Disability (MFD)Functional endpoints including loss of communication, cortical blindness, requirement of tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement

Relevance to coverage

Policy Revision History

2025-07-25Policy effectiveLatest

Policy effective date established for Skysona coverage criteria and prior authorization requirements.

Review scheduled

Next policy review scheduled for 2025-06-01.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicySkysona (elivaldogene autotemcel) — coverage criteria for CALD

Policy CodePolicy 05.04.84

Change TypeRoutine review — no material changes

Effective DateJul 25, 2025

Next Review DateJun 1, 2025

Key ActionSubmit prior authorization with documentation of ABCD1 mutation, elevated VLCFA, Loes score 0.5–9 with gadolinium enhancement, and NFS ≤ 1.

SourceLink

On This Page

DocumentationNFS must be documented in prior authorization submission

Minimum CD34+ cell dose

Minimum recommended CD34+ dose≥ 5.0 x 10^6 CD34+ cells per kg of body weight

Relation to dosing limitsApprovals may be subject to dosing limits consistent with FDA-approved labeling

Source sectionDosing and Administration (minimum recommended dose)

ANC — lab exclusion threshold

ANC exclusion thresholdPeripheral blood absolute neutrophil count (ANC) < 1500 cells/mm3 excludes coverage

RequirementBaseline hematologic assessment required prior to treatment

ContextListed as a NONE-of-the-following exclusion in initial approval criteria

Platelets — lab exclusion threshold

Platelet exclusion thresholdPlatelet count < 100,000 cells/mm3 excludes coverage

Related exclusionUncorrected bleeding disorder is also an exclusion

DocumentationBaseline hematologic assessment must be submitted for prior authorization

Hemoglobin — lab exclusion threshold

Hemoglobin exclusion thresholdHemoglobin < 10 g/dL excludes coverage

RequirementBaseline hematologic assessment required prior to infusion

Placement in criteriaListed among NONE-of-the-following exclusions for initial approval

AST/ALT — lab exclusion threshold

AST/ALT exclusionAST > 2.5 x ULN or ALT > 2.5 x ULN excludes coverage

Baseline testingBaseline hepatic assessments required and must be documented

Criteria placementIncluded in the NONE-of-the-following exclusion list for initial approval

Total bilirubin — threshold and exception

Total bilirubin exclusionTotal bilirubin > 3.0 mg/dL excludes coverage

ExceptionException: patients diagnosed with Gilbert's Syndrome may be eligible if otherwise stable

DocumentationBaseline hepatic assessment and diagnosis of Gilbert's (if claimed) must be provided

LVEF — cardiac function exclusion

LVEF exclusion thresholdLeft ventricular ejection fraction (LVEF) < 40% excludes coverage

Baseline requirementBaseline cardiac assessment required prior to treatment

Criteria placementListed among exclusion conditions in initial approval criteria

eGFR / CrCl — renal function thresholds

Renal function exclusionEstimated GFR (eGFR) < 70 mL/min/1.73 m2 OR creatinine clearance (CrCl) < 50 mL/min excludes coverage

Baseline requirementBaseline renal assessment required prior to infusion

Criteria placementListed among NONE-of-the-following exclusions for initial approval

Loes score total — maximum

Maximum possible Loes total scoreLoes score total up to 34 points

Component subscoresIncludes parieto-occipital, temporal-anterior, frontal/periventricular/central/subcortical/local atrophy, corpus callosum, optic and auditory pathways, projection fibers, cerebellum, basal ganglia, global atrophy

ReferenceLoes components and totals detailed in Appendix B

Effective 1/1/26 use HCPCS J3387 — Injection, elivaldogene autotemcel, per treatment.
If J3387 is not yet effective or not applicable, use C9399 or J3590 as unclassified drug/biological codes as appropriate.

Denial Risk

Coding-related Denial Risk

Claims using codes other than the listed HCPCS (J3387 when effective) or the unclassified codes (C9399, J3590) may be misclassified or denied. Providers should ensure the correct code is used to reduce risk of claim denial or misprocessing.

Coding-related denial risk: use J3387 (effective 1/1/26) or C9399/J3590 when applicable; incorrect or alternate codes may result in denial or misclassification.

Documentation Required

Required Billing Documentation

Required billing documentation must accompany claims. Use appropriate CPT, HCPCS (including J3387 effective 1/1/26), revenue, and ICD codes when reporting services. Submit supporting clinical documentation used for prior authorization with claims as needed.

Use appropriate CPT, HCPCS (include J3387 effective 1/1/26), Revenue, and ICD-10 diagnosis codes when billing.
Include prior authorization documentation and pertinent clinical records when submitting claims to support medical necessity.

Note

Alternate Treatments Note

Alternate treatments: Hematopoietic stem cell transplant (HSCT) is a potential alternative for some patients with CALD; however, only an estimated ~30% of patients have a matched sibling donor and may be eligible for HSCT. Consider donor availability when discussing treatment options.

HSCT is an alternative treatment option; approximately 30% of patients have a matched sibling donor making them eligible for HSCT.

Presence of one or more MFD items denotes advanced disease and is relevant to coverage determinations (exclusion from initial approval)

Source appendixListed explicitly in Appendix C (Major Functional Disability)

Loes Score — description

Loes Score descriptionAn MRI-based scoring system with component subscores (parieto‑occipital, temporal‑anterior, frontal/periventricular/central/subcortical/local atrophy, corpus callosum regions, optic and auditory pathways, projection fibers, cerebellum, basal ganglia, global atrophy) aggregated to a total up to 34

Component score rangesIndividual region scores range (e.g., parieto‑occipital 0–4, corpus callosum 0–5, basal ganglia 0–1, total = 34)

ReferenceSee Appendix B for full component breakdown and scoring

Major Functional Disability — alternate clinical entry

Alternate MFD entry (clinical states)Major Functional Disability includes loss of communication, cortical blindness, requirement of tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement

Use in determinationsThese clinical states are used to identify advanced disease and may affect coverage eligibility

Appendix referenceAppendix C lists the six MFD items verbatim