CurrentBaylor Scott & White Health PlanPolicy 308

Elivaldogene autotemcel (Skysona) gene therapy for childhood cerebral adrenoleukodystrophy (CALD)

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Defines clinical coverage criteria, prior authorization requirements, and coding guidance for one-time administration of elivaldogene autotemcel (Skysona) for boys 4–17 years with early, active cerebral ALD for Baylor Scott & White Health Plan lines of business.

Policy Summary

PayerBaylor Scott & White Health Plan

PolicyElivaldogene autotemcel (Skysona) for childhood cerebral adrenoleukodystrophy (CALD)

Policy CodePolicy 308

Change TypeNo material change

Effective DateMar 1, 2026

Last ReviewFeb 26, 2026

Next Review DateFeb 26, 2027

Key ActionPrior authorization is required and requests must document eligibility, diagnostic testing, NFS ≤1, Loes 0.5–9 with gadolinium enhancement, cardiac ejection fraction >40%, negative infection screening, and that no HLA‑matched family donor is available.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

one-timeauthorized administrations per member

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CurrentBaylor Scott & White Health PlanPolicy 308

Elivaldogene autotemcel (Skysona) gene therapy for childhood cerebral adrenoleukodystrophy (CALD)

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Policy Summary

PayerBaylor Scott & White Health Plan

PolicyElivaldogene autotemcel (Skysona) for childhood cerebral adrenoleukodystrophy (CALD)

Policy CodePolicy 308

Change TypeNo material change

Effective DateMar 1, 2026

Last ReviewFeb 26, 2026

Next Review DateFeb 26, 2027

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

one-timeauthorized administrations per member

eligible patient age range (male)

Loes 0.5–9required MRI Loes score range for active disease

NFS ≤1required Neurologic Function Score

J3387listed drug billing code

Coverage Criteria for Elivaldogene Autotemcel (Skysona)

Medically Necessary — Initial (one-time) administration

BSWHP may consider elivaldogene autotemcel medically necessary when ALL of the following are met:

Eligibility and clinical criteria

Diagnostic subcriteria for early, active CALD: Documented diagnosis of early, active cerebral adrenoleukodystrophy (CALD) meeting all of the following: elevated very long chain fatty acids (VLCFA) values; confirmed ABCD1 mutation; Neurologic Function Score ≤ 1; MRI demonstrating Loes score between 0.5 and 9 on the 34‑point scale AND gadolinium enhancement (GdE+) of demyelinating lesions on central radiographic review.Loes 0.5–9; NFS ≤1
Central radiographic MRI review required

Exclusion checklist (must NOT have any): CALD secondary to head trauma; immediate family member with known or suspected familial cancer syndrome; availability of a willing HLA‑matched family donor; active bacterial, viral, fungal, or parasitic infection; prior gene therapy (including Skysona); prior allogeneic HSCT; prior or current malignancy or myeloproliferative disorder.N/A

Presence of any listed condition excludes medical necessity

Coverage is not available when any of the following exclusionary conditions are present: Cerebral ALD (CALD) secondary to head trauma; an immediate family member with a known or suspected familial cancer syndrome (for example, hereditary breast and ovarian cancer syndrome, hereditary non‑polyposis colorectal cancer syndrome, familial adenomatous polyposis); availability of a willing HLA‑matched family donor; an active bacterial, viral, fungal, or parasitic infection; any prior gene therapy for CALD (including Skysona); any prior allogeneic hematopoietic stem cell transplant (HSCT); or a prior or current malignancy or myeloproliferative disorder.

Repeat administration of elivaldogene autotemcel is considered experimental and investigational and therefore not medically necessary, because the effectiveness of repeat dosing has not been established.

Use of elivaldogene autotemcel for any indications other than early, active childhood cerebral adrenoleukodystrophy (CALD) is considered experimental, investigational, and/or unproven and therefore not medically necessary.

Coding and Key Clinical Thresholds

Covered CPT CodesCPTCovered

96365	Intravenous infusion, for therapy, prophylaxis, or diagnosis; initial, up to 1 hour
96366	Intravenous infusion, for therapy, prophylaxis, or diagnosis; each additional hour

Covered HCPCS CodesHCPCSCovered

J3387

Injection, elivaldogene autotemcel, per treatment

Applicable ICD-10 CodesICD-10

E71.520	Childhood cerebral X-linked adrenoleukodystrophy
E71.521	Adolescent X-linked adrenoleukodystrophy

inv-08: Neurologic Function Score (NFS) — threshold

Neurologic Function Score (NFS)Less than or equal to 1 (≤ 1)

ContextRequired as part of diagnostic criteria for early, active CALD — must be ≤ 1 to meet coverage eligibility

Use in prior authorizationSubmit documented NFS in prior authorization packet

inv-09: Loes score (MRI) — value and range

Loes score (MRI)Between 0.5 and 9 on the 34-point Loes scale

ContextRequired MRI finding for active CNS disease and part of diagnostic eligibility for Skysona

Prior Authorization, Documentation, and Denial Conditions

Prior Authorization

Prior authorization required — requests must meet all listed clinical criteria

Prior authorization is required; Skysona (elivaldogene autotemcel) will be considered medically necessary only when all listed clinical criteria are met and the request is for a one-time, single administration. All requests will be reviewed by a clinical pharmacist and medical director; route requests to prior authorization for clinical review per plan procedures.

Medication must meet all eligibility and diagnostic criteria in the policy (male age 4–17; prescribing specialist; confirmed CALD with elevated VLCFA and ABCD1 mutation; NFS ≤1; Loes 0.5–9 with GdE+; LVEF >40%; negative infectious disease screening).
Request must document candidate status for HSC mobilization and that no prior gene therapy or allo‑HSCT has been performed.
Request must be for a single administration (one-time).

Note

Relationship to HSCT — use Skysona only when no HLA‑matched donor is available

Allogeneic HSCT is identified in the policy as the treatment of choice for early-stage CALD when an HLA‑matched donor is available; Skysona is indicated only when there is no available HLA‑matched donor for allogeneic HSCT.

Background on Adrenoleukodystrophy and Skysona

Adrenoleukodystrophy (ALD) is a rare, X‑linked genetic disorder characterized by progressive loss of cerebral white matter and often associated adrenal insufficiency. The childhood cerebral form (CALD) is the most severe presentation and typically manifests in early childhood with rapidly progressive neurologic decline. Standard therapy for early‑stage CALD is allogeneic HSCT when an HLA‑matched donor is available; elivaldogene autotemcel (Skysona) is a one‑time gene therapy option for boys meeting strict diagnostic and radiographic criteria when no suitable HLA‑matched family donor exists.

Definitions and Outcome Measures

inv-16: Early, active cerebral adrenoleukodystrophy (CALD) — diagnostic definition

Core diagnostic elementsElevated very long chain fatty acids (VLCFA) and confirmed ABCD1 mutation

Neurologic statusNeurologic Function Score ≤ 1 required

MRI evidenceActive CNS disease demonstrated by Loes score 0.5–9 on 34-point scale AND gadolinium enhancement of demyelinating lesions

Definition summaryEarly, active cerebral adrenoleukodystrophy (CALD) = elevated VLCFA + ABCD1 mutation + NFS ≤1 + MRI evidence of active demyelination

inv-17: Major functional disability (MFD)-free survival — trial definition

DefinitionMajor functional disability (MFD)-free survival = clinical outcome used in trials indicating freedom from major functional disability events over follow-up

Policy Summary

PayerBaylor Scott & White Health Plan

PolicyElivaldogene autotemcel (Skysona) for childhood cerebral adrenoleukodystrophy (CALD)

Policy CodePolicy 308

Change TypeNo material change

Effective DateMar 1, 2026

Last ReviewFeb 26, 2026

Next Review DateFeb 26, 2027

SourceLink

On This Page

Radiology requirementCentral radiographic review of brain MRI required; must document Loes score and gadolinium enhancement

inv-10: Left ventricular ejection fraction — threshold

Left ventricular ejection fraction (LVEF)> 40%

ContextAdequate cardiac function demonstrated by LVEF >40% is required for coverage consideration

DocumentationInclude echocardiogram or equivalent report showing LVEF in prior authorization submission

Confirm and document evaluation for an HLA‑matched family donor prior to asserting Skysona as the treatment plan.
If a willing HLA‑matched family donor is available, HSCT rather than Skysona is the preferred treatment per policy.

Documentation Required

Required documentation for prior authorization

Submit complete clinical documentation with the prior authorization request to support each required eligibility element; incomplete documentation may delay or result in denial.

Patient sex and age and identity of prescribing specialist (neurologist, hematologist/oncologist, endocrinologist, or stem cell transplant specialist).
Diagnostic testing: elevated very long chain fatty acids (VLCFA) values and confirmed ABCD1 mutation.
Neurologic Function Score (NFS) demonstrating ≤ 1.
Central MRI report including Loes score (0.5–9 on 34‑point scale) and evidence of gadolinium enhancement (GdE+) of demyelinating lesions; note that central radiographic review is required.
Evidence member is a candidate for HSC mobilization and left ventricular ejection fraction > 40%.
Negative screening results for HIV-1/2, hepatitis B, hepatitis C, and HTLV‑1/2.
Documentation that the request is for a one-time administration and confirmation there is no prior gene therapy or prior allogeneic HSCT.
Supporting clinical notes and prescribing specialist identifier; include any relevant cardiac or infectious disease test reports.

Denial Risk

Grounds for denial — common reasons a request may be denied

Requests that do not meet the policy criteria or that lack required documentation are subject to denial. Common grounds for denial are explicitly listed in the policy.

Member not male or outside the 4–17 year age range.
Neurologic Function Score > 1.
Loes score outside 0.5–9 or absence of gadolinium enhancement on MRI of demyelinating lesions.
Evidence of an available willing HLA‑matched family donor.
Prior gene therapy (including Skysona) or prior allogeneic HSCT.
Active bacterial, viral, fungal, or parasitic infection.
Prior or current malignancy or myeloproliferative disorder.
Missing or incomplete documentation of required diagnostic tests, MRI central review, infectious disease screening, or cardiac function (LVEF ≤ 40%).

Trial usageUsed to compare treated (Skysona) versus untreated cohorts in ALD-102 and ALD-104 studies

Reported resultElivaldogene autotemcel–treated patients had an estimated 72% likelihood of MFD-free survival at 24 months versus 43% in untreated patients