CurrentBlue Cross Blue Shield - MassachusettsPolicy 241

Gene Therapy for Cerebral Adrenoleukodystrophy (Skysona® / elivaldogene autotemcel) — Prior Authorization

This document governs prior authorization submission requirements and medical necessity criteria for Skysona (elivaldogene autotemcel) for treatment of cerebral X-linked adrenoleukodystrophy in pediatric patients, and applies to providers submitting requests to Blue Cross Blue Shield Massachusetts.

Policy Summary

PayerBlue Cross Blue Shield - Massachusetts

PolicyGene Therapy for Cerebral Adrenoleukodystrophy (Skysona® / elivaldogene autotemcel) — Prior Authorization

Policy CodePolicy 241

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit initial authorization requests via Authorization Manager for commercial members (out-of-network providers may fax to 888-973-0726).

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

Age 4–17eligible patient age at time of infusion

Loes 0.5–9required MRI Loes score range (34-point scale)

C9399 / J3490 / J3590billing codes listed for Skysona

ANC ≥1500; Plt ≥100k; Hgb ≥10hematologic minimums

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cardiac function threshold

Coverage and Medical Necessity Criteria

Initial Therapy

Covered when ALL of the following are met:

Age: Individual is 4 to 17 years of age at the time of infusion of elivaldogene autotemcel4 to 17 years

Diagnosis: Documented diagnosis of active cerebral X-linked adrenoleukodystrophy with confirmed ABCD1 (ABCDI) gene mutation and elevated very-long-chain fatty acids (VLCFAs)

Loes score between 0.5 and 9 (inclusive) on the 34-point MRI scaleLoes 0.5–9

Active CNS disease: Presence of active central nervous system disease documented by gadolinium enhancement on MRI of demyelinating lesions

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

Patients with any of the listed contraindications are excluded from coverage for Skysona (elivaldogene autotemcel). Key exclusions include a history of prior gene therapy or allogeneic hematopoietic stem cell transplant; significant hematologic compromise such as ANC < 1500 cells/mm3, platelet count < 100,000 cells/mm3 or hemoglobin < 10 g/dL, or an uncorrected bleeding disorder; hepatic compromise with AST or ALT > 2.5 × ULN and total bilirubin > 3.0 mg/dL (except stable Gilbert’s syndrome); left ventricular ejection fraction < 40%; baseline renal impairment with eGFR < 70 mL/min/1.73 m2 or CrCl < 50 mL/min; a first‑degree relative with a known familial cancer syndrome as specified on the form; and clinically significant uncontrolled active infections (including but not limited to HIV, HTLV‑1, active hepatitis B or C).

Therapy is not supported when the documented eligibility criteria are not fully met. Coverage requires that the patient be 4 to 17 years of age at the time of infusion, have a confirmed ABCD1 mutation with elevated very‑long‑chain fatty acids (VLCFAs), a Loes score between 0.5 and 9 (inclusive), evidence of active CNS disease demonstrated by gadolinium enhancement on MRI of demyelinating lesions, and a documented Neurologic Function Score (NFS) < 1. Additionally, listed contraindications (see exclusions) must be absent; presence of any contraindication renders the use not supported.

Initial Therapy — Detailed Required Elements

Initial therapy coverage criteria

Initial coverage is granted when all of the following are documented:

Age: 4 to 17 years at time of infusion4–17 years

Genetics: Confirmed ABCD1 (ABCDI) gene mutation documented

VLCFA: Elevated very-long-chain fatty acids documented

Loes score 0.5 to 9 inclusive on the 34-point scale0.5–9

Coding and Clinical Thresholds

HCPCS Codes listedHCPCS

C9399	Unclassified drugs or biologicals
J3490	Unclassified drugs
J3590	Unclassified biologics

inv-05: Loes score — required MRI Loes score range

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

Scale described34-point MRI-based scale used to quantify cerebral demyelination

Context for eligibilityLoes score requirement is part of initial therapy criteria for active cerebral X-linked adrenoleukodystrophy

inv-06: ANC — hematologic minimum

ANC minimum>= 1500 cells/mm3 (absolute neutrophil count)

RelevancePart of hematologic criteria to exclude patients with hematologic compromise

Authorization Submission and Documentation Requirements

Prior Authorization

Submission route and contact

Providers must use Authorization Manager to submit initial authorization requests for commercial members. Authorization documentation, case status checks, and decision letters are available in Authorization Manager. Out-of-network providers should fax requests to 888-973-0726.

Submit initial authorization requests via Authorization Manager for commercial members.
Out-of-network providers: fax requests to 888-973-0726.

Denial Risk

Missing medical necessity criteria

Clinical documentation that supports the medical necessity criteria for Skysona must be submitted. If the patient does not meet all listed medical necessity criteria, submit a letter of medical necessity requesting Clinical Exception (Individual Consideration) that explains why an exception is justified.

Include documentation demonstrating the patient meets all medical necessity criteria in the policy.

Clinical Background

Cerebral X‑linked adrenoleukodystrophy (CALD) is a progressive demyelinating disorder caused by mutations in the ABCD1 gene that result in accumulation of very‑long‑chain fatty acids (VLCFAs) and active central nervous system disease. Skysona (elivaldogene autotemcel) is a gene therapy indicated for pediatric patients with active CALD who meet specific genetic, biochemical, MRI, and neurologic criteria.

Key Definitions and Scoring

inv-17: Loes score definition — MRI-based 34-point scale; required range 0.5 to 9 inclusive

DefinitionLoes score: MRI-based 34-point scale used to quantify cerebral demyelination

Required range for eligibilityRequired Loes score between 0.5 and 9 (inclusive)

Use in criteriaLoes score requirement is part of initial therapy coverage criteria for active cerebral X-linked adrenoleukodystrophy

inv-18: Neurologic Function Score (NFS) — clinical scoring; document NFS < 1 for eligibility

DefinitionNeurologic Function Score (NFS): clinical scoring of neurologic function

Eligibility requirementDocumented NFS < 1 is required for eligibility

Place of Service Requirements

Billing Rule

Document place of service (outpatient or inpatient)

Document place of service on the prior authorization form as outpatient or inpatient; the form includes fields for Place of Service.

Select 'Outpatient' or 'Inpatient' in the Place of Service field on form #242

Policy Summary

PayerBlue Cross Blue Shield - Massachusetts

PolicyGene Therapy for Cerebral Adrenoleukodystrophy (Skysona® / elivaldogene autotemcel) — Prior Authorization

Policy CodePolicy 241

Change TypeNo material change

Effective Date

Next Review Date

Key ActionSubmit initial authorization requests via Authorization Manager for commercial members (out-of-network providers may fax to 888-973-0726).

SourceLink

On This Page

MRI enhancement: Gadolinium enhancement on MRI of demyelinating lesions indicating active disease

NFS: Documented Neurologic Function Score < 1NFS <1

Related contraindication note

Hematological compromise requires ANC <1500, platelets <100,000 OR Hgb <10 g/dL to be considered compromised

inv-07: Platelet count — hematologic minimum

Platelet count minimum>= 100,000 cells/mm3

Hematologic panel contextPlatelet threshold used with ANC and hemoglobin to define hematologic suitability for therapy

Contraindication tie-inPlatelet count <100,000 is listed as hematologic compromise and excludes therapy

inv-08: Hemoglobin — hematologic minimum

Hemoglobin minimum>= 10 g/dL

Role in eligibilityHemoglobin <10 g/dL constitutes hematologic compromise and is a contraindication

Combined criteriaHgb threshold is considered with ANC and platelet criteria to assess hematologic compromise

inv-09: LVEF — cardiac function threshold

LVEF threshold>= 40% (Left ventricular ejection fraction)

ContraindicationLeft ventricular ejection fraction <40% is listed as cardiac compromise and excludes therapy

AssessmentDocument LVEF to confirm cardiac eligibility prior to therapy

inv-10: Baseline eGFR / CrCl — renal function thresholds

eGFR thresholdeGFR >= 70 mL/min/1.73 m2

CrCl thresholdCreatinine clearance (actual or calculated) >= 50 mL/min

Most actionable renal requirementBaseline renal function must meet either eGFR >=70 mL/min/1.73 m2 OR CrCl >=50 mL/min

If criteria are not met, include a detailed letter of medical necessity requesting Clinical Exception (Individual Consideration).

Note

Incorrect provider/facility identifiers

Ensure the servicing provider and facility identifiers are entered correctly in Authorization Manager. Enter the appropriate surgeon's NPI or provider ID as the servicing provider (not the billing group) and enter the facility's NPI or provider ID where services will be performed. Incorrect identifiers may delay processing.

Enter surgeon's NPI or provider ID as the servicing provider (do not use the billing group).
Enter the facility's NPI or provider ID for the location where services are performed.
Incorrect provider/facility NPI entries may delay authorization processing.

Documentation Required

Required documentation

Required documentation must be submitted with the prior authorization request. Complete the Prior Authorization Request Form #242 and include patient and facility information and all clinical records that support medical necessity.

Complete Prior Authorization Request Form for Cerebral Adrenoleukodystrophy Skysona (Form #242) using Authorization Manager.
Patient information: name, BCBSMA ID#, date of birth, date of treatment, place of service.
Facility information: facility name, address, phone, fax, NPI.
All clinical documentation supporting medical necessity criteria.

Note

Provider action

Provider action highlight: follow Authorization Manager submission steps and verify identifiers and documentation to avoid processing delays.

Use Authorization Manager and follow submission guidance and resources.
Refer to Authorization Manager resources for tips, guides, and video demonstrations.

Placement in criteriaNFS <1 is one of the documented initial therapy criteria for treatment