Blue Cross MA Skysona Coverage Prior Auth

Coverage Criteria for Elivaldogene Autotemcel (Skysona)

inv-01: Medically Necessary — Initial Eligibility — Covered when ALL of the following are met

Covered when ALL of the following are met:

Age: Individual is 4 to 17 years of age at the time of infusion of elivaldogene autotemcel.4-17 years

Genetic confirmation: Confirmed pathogenic mutation in the ABCD1 gene.

Evidence of active CNS disease: Presence of active central nervous system disease evidenced by gadolinium-enhancing demyelinating lesions on MRI and elevated very-long-chain fatty acids (VLCFAs).

Loes score must be between 0.5 and 9 (inclusive) and NFS <1

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

Neurologic function: Documented Neurologic Function Score (NFS) < 1.NFS <1

inv-02: Contraindications / NOT eligible (any one excludes coverage) — Not covered if ANY of the following are present

Not covered if ANY of the following are present:

Prior gene therapy or allo-HSCT: History of receiving prior gene therapy or allogeneic hematopoietic stem cell transplant.

Hematologic compromise: Peripheral blood absolute neutrophil count (ANC) <1500 cells/mm^3, platelet count <100,000 cells/mm^3, or hemoglobin <10 g/dL.ANC <1500; platelets <100,000; Hgb <10 g/dL

Uncorrected bleeding disorder that contraindicates procedures or imaging.

Hepatic compromise: Alanine transaminase (ALT) >2.5 x ULN, aspartate transaminase (AST) >2.5 x ULN, or total bilirubin >3.0 mg/dL (except stable Gilbert's syndrome).ALT >2.5x ULN; AST >2.5x ULN; bilirubin >3.0 mg/dL

inv-03: Medically Necessary (trial-based) criteria — Covered when ALL of the following are met (trial enrollment-based criteria)

Covered when ALL of the following are met

Trial-defined eligibility (interventional studies): Patient is assigned male at birth AND age 4 to 17 years AND has early, active CALD defined by Loes score 0.5–9 inclusive AND has gadolinium enhancement on MRI AND has neurologic function score (NFS) <1.Loes 0.5-9; NFS <1; age 4-17 years

Based on single-arm interventional studies with post-hoc analyses versus historical controls; evidence limited by small sample sizes, use of historical controls, baseline differences, and limited follow-up.

Repeat administration of elivaldogene autotemcel is not supported. The policy explicitly states one injection per lifetime, and any attempt at repeat gene‑therapy dosing is considered investigational and thus not covered.

Coverage is limited to the trial‑defined early, active CALD population. Use may be denied when the member’s disease or demographic characteristics do not match the studied population — for example, a Loes score outside 0.5–9, absence of gadolinium enhancement on MRI, a Neurologic Function Score (NFS) ≥1, or age outside the 4–17 years range are not supported by the evidence cited in this policy.

When an individual does not meet the specified medical‑necessity criteria (including genetic confirmation of an ABCD1 mutation, required MRI findings, Loes score 0.5–9 inclusive, NFS <1, and age 4–17 years), elivaldogene autotemcel is considered investigational and therefore not covered.

The evidence base has important limitations that affect certainty. Available data come from small, single‑arm interventional studies with post‑hoc analyses against non‑concurrent historical controls, with short and variable follow‑up. These limitations — including small sample sizes, baseline differences between treated and historical cohorts, and limited duration to detect long‑term outcomes such as later hematologic malignancy — contribute to uncertainty about net health benefit and durability of effect.

Covered Indications

Eligibility Requirements

Eligibility for elivaldogene autotemcel requires documentation of active cerebral X‑linked adrenoleukodystrophy and fulfillment of trial‑based enrollment criteria. At a minimum, the individual must have a confirmed ABCD1 mutation, be within the 4–17 years age range at infusion, demonstrate active CNS disease with elevated VLCFAs and gadolinium enhancement on MRI, have a Loes score between 0.5 and 9 (inclusive), and a documented NFS <1. Refer to the policy checklists and required documentation when submitting prior authorization.

Additional pre‑treatment eligibility considerations include screening laboratory and clinical parameters addressed in the policy. Contraindications that would exclude coverage include prior gene therapy or allogeneic hematopoietic stem cell transplant, hematologic compromise (e.g., ANC <1500 cells/mm3, platelets <100,000 cells/mm3, hemoglobin <10 g/dL), significant hepatic or renal impairment, left ventricular ejection fraction <40%, uncontrolled active infection, and contraindications to mobilization or conditioning agents (G‑CSF, plerixafor, busulfan, fludarabine). Adequate contraception for fertile patients is also required.

Provider Actions and Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required for elivaldogene autotemcel. Providers must submit initial authorization requests using Authorization Manager and include the servicing provider's NPI (surgeon or servicing provider) and the facility's NPI where services will be performed. For out-of-network providers, requests should still be faxed to 888-973-0726. Precertification/preauthorization is required for inpatient services; prior authorization is also required for outpatient services across Commercial Managed Care (HMO and POS), Commercial PPO and Indemnity, and Medicare products as specified by the payer.

Use Authorization Manager (available 24/7) to submit requests, upload clinical documentation, check case status, and view/print decision letters.
Enter the servicing provider's NPI (surgeon's NPI or provider ID) — not the billing group.
Enter the facility's NPI or provider ID for the service location.
Out-of-network providers: fax requests to 888-973-0726.

Billing and Coding

HCPCS CodesHCPCS

C9399	Unclassified drugs or biologicals.
J3490	Unclassified drugs.
J3590	Unclassified biologics.

ICD-10 Procedure CodesICD-10

XW043F3	Percutaneous ApproachNew Technology Group 3 (no specific code for this drug).
XW043FS	Introduction of Other New Technology Therapeutic Substance into Central Vein, Percutaneous Approach_New Technology Group 5

Loes score

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

Scale referenced34-point Loes MRI scoring scale used in eligibility assessment.

Context of useRequired component of documented active CNS disease for medical necessity.

Neurologic Function Score (NFS)

NFS requirementDocumented neurologic function score (NFS) <1 for eligibility.

Role in trialsNFS <1 indicates limited changes in neurologic function in interventional studies.

Not Covered / Investigational Use

Consistent with the policy exclusions, any use that does not meet the stated medical‑necessity criteria is considered investigational and not covered. In particular, repeat administration is explicitly listed as investigational and is not a covered benefit.

Use of elivaldogene autotemcel outside the trial‑defined early, active CALD population — for example in patients who do not meet the Loes, MRI enhancement, NFS, age, or genetic criteria described for the interventional studies — is not supported by the provided evidence and is therefore not covered.

Background

Cerebral adrenoleukodystrophy (CALD) is an X‑linked neurodegenerative disorder caused by pathogenic variants in the ABCD1 gene leading to accumulation of very‑long‑chain fatty acids (VLCFAs) and inflammatory cerebral demyelination. The childhood, cerebral form predominantly affects individuals assigned male at birth and can progress rapidly after symptom onset. Allogeneic hematopoietic stem cell transplantation has been used historically for early disease but carries significant risks. Elivaldogene autotemcel is an autologous CD34+ hematopoietic stem‑cell based lentiviral gene therapy intended to deliver functional ABCD1 cDNA to restore ALDP expression and reduce VLCFA accumulation.

Definitions and Key Terms

CALD (definition)

Definition of CALDCerebral adrenoleukodystrophy (CALD) is an X-linked genetic neurodegenerative disease caused by ABCD1 mutations leading to impaired or loss of ALDP expression and accumulation of very-long-chain fatty acids (VLCFAs) with inflammatory cerebral demyelination.

Population most affectedMost severely affects individuals assigned male at birth in childhood; female carriers are rare for childhood CALD.

PathophysiologyVLCFA accumulation initiates an inflammatory cascade resulting in cerebral demyelination and rapid clinical decline after symptom onset.

Minimum dose

Minimum recommended doseMinimum recommended dose of elivaldogene autotemcel is 5.0 x 10^6 CD34+ cells/kg of body weight.

Dose unit

Revision History

2024-01clarified

Clarified coverage and updated internal criteria to align with 118E MGL S S1A.

2023-07reformatted

Policy reformatted (layout and presentation updates).

2023-02-01addedLatest

New medical policy created describing medically necessary indications for elivaldogene autotemcel with literature review through October 24, 2022; policy effective 02/01/2023.

OpenPayer

Elivaldogene autotemcel (Skysona) for cerebral X‑linked adrenoleukodystrophy (CALD) — coverage criteria