Currentblue cross blue shield - tennesseePolicy N/A

Skysona (gene therapy) for cerebral adrenoleukodystrophy (CALD)

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Covers medical necessity criteria, prior authorization, documentation, exclusions, and monitoring requirements for use of Skysona in boys with early, active cerebral adrenoleukodystrophy (CALD) for BlueCross BlueShield of Tennessee members.

Policy Summary

Payerblue cross blue shield - tennessee

PolicySkysona (gene therapy) for cerebral adrenoleukodystrophy (CALD)

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 31, 202611d

Next Review DateN/A

Key ActionObtain prior authorization and submit documentation including VLCFA results and a pathogenic or likely pathogenic ABCD1 variant, MRI central review (Loes score and gadolinium enhancement), and Neurologic Function Score.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

4–17eligible patient age range

0.5–9Loes score range

NFS ≤1Neurologic Function Score

3 months, one-timeAuthorization

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Full ABCD1 deletions

Coverage Criteria for Skysona (CALD)

Initial authorization for CALD

Covered when ALL of the following are met:

Patient: Member is male between 4 and 17 years of ageage 4-17

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Genetic and biochemical confirmation: Presence of a pathogenic or likely pathogenic ABCD1 variant AND elevated very long chain fatty acids (VLCFA) per laboratory reference rangegenetic test + VLCFA

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Disease activity and severity: Central radiographic MRI review demonstrating gadolinium enhancement of demyelinating lesions AND Loes score between 0.5 and 9 inclusive; Neurologic Function Score ≤ 1Loes 0.5-9; NFS ≤ 1; gadolinium enhancement

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Treatment history: No prior Skysona or other gene therapy AND member has not received prior allogeneic HSCTno prior gene therapy or allo-HSCT

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HSCT donor status: Member is eligible for hematopoietic stem cell transplant (HSCT) but unable to find an HLA-matched donorunable to find HLA-matched donor

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Safety labs and organ function: No hematologic compromise (ANC ≥ 1500 cells/mm3; platelets ≥ 100,000 cells/mm3; hemoglobin ≥ 10 g/dL; no uncorrected bleeding disorder); no hepatic compromise (AST and ALT ≤ 2.5 × ULN; total bilirubin ≤ 3.0 mg/dL unless Gilbert's and stable); no cardiac compromise (LVEF ≥ 40%); no renal compromise (CrCl ≥ 50 mL/min or eGFR ≥ 70 mL/min/1.73 m2); negative serology for HIV, HBV, HCV, HTLV-1/2; no active infectionsee lab thresholds module

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Cancer risk and monitoring: No immediate family member with known or suspected familial cancer syndrome or history of such; member will be monitored for evidence of malignancy per the prescribing information following infusionfamily history negative; monitoring per PI

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The following scenarios are excluded from coverage for Skysona: use to treat or prevent adrenal insufficiency; genetic variants consisting of full deletions of the ABCD1 transgene; and Cerebral Adrenoleukodystrophy (CALD) secondary to head trauma. Coverage will not be provided for members meeting any of these exclusion criteria.

Any use of Skysona beyond the FDA‑approved indication and recognized compendial uses is considered experimental/investigational and not medically necessary and therefore is not covered under this policy.

Laboratory and Code Thresholds

Laboratory and physiologic thresholds

Absolute neutrophil count (ANC)≥ 1500 cells/mm3 (no hematologic compromise)

Platelet count≥ 100,000 cells/mm3 (no hematologic compromise)

Hemoglobin≥ 10 g/dL (no hematologic compromise)

AST/ALTAST and ALT ≤ 2.5 × upper limit of normal (no abnormal hepatic function)

Total bilirubin≤ 3.0 mg/dL unless diagnosed Gilbert's syndrome and otherwise stable

Cardiac functionLeft ventricular ejection fraction ≥ 40% (no cardiac compromise)

Renal functionCreatinine clearance ≥ 50 mL/min or eGFR ≥ 70 mL/min/1.73 m2 (no abnormal renal function)

Provider Actions and Prior Authorization Requirements

Prior Authorization

Prior authorization required; 3-month one-time authorization

Prior authorization is required. When all coverage criteria are met, authorization of 3 months for a one-time administration may be granted.

Step Therapy

HSCT eligibility and donor-unavailability required

Member must be eligible for hematopoietic stem cell transplant (HSCT) but unable to find a human leukocyte antigen (HLA)-matched donor; member must not have received a prior allogeneic HSCT or prior gene therapy.

Documentation Required

Documentation required to initiate prior authorization

Background

Skysona is an autologous hematopoietic stem cell gene therapy indicated to slow neurologic progression in boys aged 4–17 years with early, active cerebral adrenoleukodystrophy (CALD). Early, active disease is characterized by gadolinium enhancement on brain MRI, Loes scores between 0.5 and 9, and minimal clinical symptoms (Neurologic Function Score ≤ 1). Authorization is for a one‑time administration and prior authorization is required.

Key Definitions

Definition: Early, active CALD

Age and sexMale between 4 and 17 years of age

Clinical severity (NFS)Neurologic Function Score (NFS) ≤ 1 (asymptomatic or mildly symptomatic)

MRI activityGadolinium enhancement on brain MRI of demyelinating lesions

Loes scoreLoes score between 0.5 and 9 inclusive on the 34-point scale

Definition summaryThese features define early, active cerebral adrenoleukodystrophy (CALD) appropriate for Skysona under the FDA indication

Exclusion: Full ABCD1 gene deletions

Genetic exclusion

Policy Summary

Payerblue cross blue shield - tennessee

PolicySkysona (gene therapy) for cerebral adrenoleukodystrophy (CALD)

Policy CodePolicy N/A

Change TypeNo material clinical or coverage changes

Effective DateJul 31, 202611d

Next Review DateN/A

SourceLink

On This Page

Submit chart notes, medical records, or laboratory results documenting elevated very long chain fatty acids (VLCFA) per the laboratory reference range and a pathogenic or likely pathogenic ABCD1 gene variant. Include baseline hematologic, hepatic, renal, and cardiac assessments, MRI central radiographic review (Loes score and gadolinium enhancement), and Neurologic Function Score as part of the prior authorization package.

Elevated VLCFA values (lab reference range).
Pathogenic or likely pathogenic ABCD1 variant (genetic testing).
Baseline labs and assessments: ANC, platelets, hemoglobin, AST/ALT, total bilirubin, creatinine/CrCl or eGFR, LVEF.
Central radiographic MRI review showing gadolinium enhancement and Loes score; Neurologic Function Score (≤1).

Denial Risk

Triggers for denial and active exclusions

Coverage will not be provided for members with full deletions of the ABCD1 transgene, for use of Skysona to treat or prevent adrenal insufficiency, or for CALD secondary to head trauma. Additional denial risks include active infection, hematologic/hepatic/renal/cardiac compromise, or immediate family history of a familial cancer syndrome per the exclusions.

Full deletions of the ABCD1 transgene are excluded.
Use to treat or prevent adrenal insufficiency is excluded.
CALD secondary to head trauma is excluded.
Active bacterial, viral, fungal, or parasitic infection or abnormal labs/organ dysfunction can trigger denial.

Full deletions of the ABCD1 gene (complete ABCD1 gene deletions) are associated with limited persistence/efficacy of Skysona and are listed as an exclusion

ImplicationAn immune response in patients with full ABCD1 deletions may limit persistence of descendent cells and cause rapid loss of efficacy

Policy actionCoverage will not be provided for members with full ABCD1 deletions

Catch payer policy changes before they impact reimbursement