CurrentCignaPolicy N/A

Skysona (gene therapy) prior authorization / coverage criteria

This form governs prior authorization requirements and documentation for Skysona gene therapy requests submitted to Cigna, describing clinical eligibility, required labs/tests, administration/setting, and attestations for pediatric patients with cerebral adrenoleukodystrophy (CALD). It applies to providers submitting requests to Cigna's Gene Therapy Program.

Policy Summary

PayerCigna

PolicySkysona (gene therapy) prior authorization / coverage criteria

Policy CodePolicy N/A

Change TypeNo material changes

Effective DateN/A

Next Review DateN/A

Key ActionComplete and fax the Gene Therapy Prior Authorization form with supporting clinical documentation to Fax# 833-910-1625.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

age 4-17Eligible patient age range

Loes 0.5-9MRI Loes score range

score ≤1Neurologic function

AST/ALT ≤2.5xLiver thresholds

ANC ≥1500Hematologic thresholds

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Coverage Criteria for Skysona (Initial and Limitations)

inv-01: Initial therapy — Skysona is considered medically necessary when ALL of the following are met

Skysona is considered medically necessary when ALL of the following are met:

Age and prior receipt: Patient is male AND patient is ≥ 4 and < 18 years of age AND patient has not received Skysona previously (verification in claims history required or prescriber attestation)

Documentation required: claims check or prescriber confirmation

Disease state (early, active CALD): Patient has early, active cerebral adrenoleukodystrophy demonstrated by: (i) neurologic function score ≤ 1; AND (ii) gadolinium enhancement on brain MRI; AND (iii) Loes score between 0.5 and 9Loes 0.5-9; neurologic score ≤1

Documentation required for each element (MRI report with Loes score and enhancement, neurologic function score)

Genetic and biochemical confirmation: Patient has a pathogenic ABCD1 variant AND elevated very long chain fatty acids per laboratory reference

Documentation required: genetic test results and VLFC/biochemical lab report

Donor status: Either patient does not have an HLA-matched donor OR has an HLA-matched donor who is not able or willing to donate

Infection and malignancy exclusions: Patient does not currently have an active bacterial, viral, fungal, or parasitic infection AND does not have prior or current hematologic malignancy or myeloproliferative disorder AND no familial cancer syndrome or history of such in the immediate family

Documentation required: infection screening and medical history

Transplant suitability: Prescribing physician considers hematopoietic stem cell transplantation appropriate (if applicable)

Documentation required: physician attestation

Laboratory and organ function thresholds (within 30 days): AST and ALT ≤ 2.5 × ULN AND total bilirubin ≤ 3.0 mg/dL AND (estimated creatinine clearance ≥ 50 mL/min OR eGFR ≥ 70 mL/min/1.73 m2) AND peripheral blood ANC ≥ 1,500 cells/mm3 AND platelet count ≥ 100,000 cells/mm3 AND hemoglobin ≥ 10 g/dLAST/ALT ≤2.5×ULN; bilirubin ≤3.0 mg/dL; CrCl ≥50 mL/min or eGFR ≥70; ANC ≥1500; platelets ≥100,000; Hgb ≥10 g/dL

All labs must be within the past 30 days and documented

Planned procedures and prescribing specialists: Patient will undergo mobilization, apheresis, myeloablative conditioning, and lymphodepletion; a granulocyte‑colony stimulating factor product will be used for mobilization; busulfan will be used for myeloablative conditioning; cyclophosphamide or fludarabine will be used for lymphodepletion; and medication is prescribed by a hematologist, neurologist, and/or stem cell transplant specialist

Provider must attest planned procedures and agents on the prior authorization form

Recent weight: Current patient body weight has been obtained within the past 30 days

Documentation required

Prior hematopoietic stem cell transplantation and prior receipt of gene therapy (including Skysona) were exclusion criteria in the pivotal studies and are listed on the prior authorization form. Providers must attest the patient has not had a prior allogeneic hematopoietic stem cell transplant and, if either prior transplant or prior gene therapy applies, submit clinical rationale and supporting documentation explaining why Skysona is still medically necessary for this patient.

A Full ABCD1 gene deletion is called out as a limitation of use on the form. In one clinical trial patient with a full ABCD1 deletion, radiologic disease progression and declining peripheral blood vector copy number suggested loss of product efficacy—potentially immune mediated—so providers must document and provide clinical rationale if this applies.

Any use of Skysona outside the specified coverage criteria is considered experimental, investigational, or unproven. Examples explicitly listed include prior allogeneic hematopoietic stem cell transplantation, prior receipt of gene therapy, and full ABCD1 gene deletion; if such circumstances exist, the provider must supply clinical support and rationale with the authorization request.

Provider Action Items and Submission Requirements

Prior Authorization

Prior Authorization Required — Gene Therapy

Complete and fax the Cigna Gene Therapy Prior Authorization form with ALL required supporting documentation to expedite review. Fax completed form to 833-910-1625. Failure to complete the form in its entirety or to include required documentation may delay processing or result in an adverse determination for insufficient information.

Fax number: 833-910-1625
Contact: Gene Therapy Program 855.678.0051 or GeneTherapyProgram@Cigna.com
Form must include all asterisked (*) fields to permit fax response

Documentation Required

Conditioning and Mobilization Attestation

Providers must attest to planned mobilization, apheresis, myeloablative conditioning, and lymphodepletion and specify the agents to be used. This attestation is required as part of the authorization submission.

Provider must confirm patient will undergo mobilization, apheresis, myeloablative conditioning, and lymphodepletion
Mobilization agent: granulocyte‑colony stimulating factor product (specify agent)
Myeloablative conditioning agent: busulfan (specify dosing plan)
Lymphodepletion agent: cyclophosphamide or fludarabine (specify agent and dosing)

Documentation Required

Required Supporting Documentation

Submit the Gene Therapy Prior Authorization form accompanied by complete supporting clinical documentation. Include chart notes and specific test reports referenced on the form so clinical reviewers can verify eligibility criteria.

Chart notes and recent history & physical
Genetic test results confirming pathogenic ABCD1 variant (documentation required)
Brain MRI reports demonstrating gadolinium enhancement and Loes score (documentation required)
Neurologic function score documentation (score ≤ 1) and clinical assessment
Laboratory reports within 30 days: AST, ALT, total bilirubin, CBC with ANC, platelet count, hemoglobin, creatinine clearance or eGFR
Infectious disease screening results: Hepatitis B, Hepatitis C, HTLV‑1/2, HIV‑1/2
Documentation of very long chain fatty acid levels (elevated per lab reference)
Proof of no prior Skysona claim or prescribing physician attestation that patient has not previously received Skysona
Documentation of current body weight within 30 days
If applicable, clinical rationale and supporting documentation when any standard requirement is not met

Denial Risk

Incomplete Submission Risk

Incomplete submissions — including missing form fields, absent supporting test reports, or lack of the required attestations — may delay processing or result in an adverse determination for insufficient information.

All asterisked (*) fields on the prior authorization form must be completed for Cigna to respond via fax
Omittted genetic, MRI, laboratory, or infectious screening documentation may lead to denial or request for additional information
If provider believes Skysona is medically necessary despite unmet criteria, submit detailed clinical rationale and supporting evidence

Relevant CPT/HCPCS/J-Codes and Clinical Thresholds

Cell collection / administration CPT/HCPCSmixed

96372	Therapeutic, prophylactic, or diagnostic injection; subcutaneous or intramuscular
38206	Blood-derived hematopoietic progenitor cell harvesting for transplantation, per collection; autologous

Mobilization / G-CSF and plerixafor J/Q codesHCPCS

J2562	Injection, plerixafor, 1 mg (Mozobil)
J1442	Injection, filgrastim, excludes biosimilar, 1 mcg
J1447	Injection, tbo-filgrastim, 1 mcg
Q5101	Injection, filgrastim-sndz, biosimilar (Zarxio), 1 mcg
Q5110	Injection, filgrastim-aafi, biosimilar (Nivestym), 1 mcg

Conditioning regimen J-codesHCPCS

J0594

Injection, busulfan, 1 mg

inv-08: Creatinine clearance / eGFR thresholds

Creatinine clearance thresholdEstimated creatinine clearance ≥ 50 mL/min (within past 30 days)

eGFR thresholdEstimated glomerular filtration rate (eGFR) ≥ 70 mL/min/1.73 m2 (within past 30 days)

Documentation timingResult must be documented within the past 30 days

Attestation item	Provider response
Provider attests that the patient plans to undergo mobilization, apheresis, myeloablative conditioning and lymphodepletion
Provider attests that a granulocyte‑colony stimulating factor product (G‑CSF) and Mozobil (plerixafor) will be used for mobilization when applicable

OpenPayer

Skysona (gene therapy) prior authorization / coverage criteria

Coverage Criteria for Skysona (Initial and Limitations)

Initial Therapy Specific Criteria

Provider Action Items and Submission Requirements

Relevant CPT/HCPCS/J-Codes and Clinical Thresholds

Key Definitions and Scores

Site of Care and Acquisition Options

Step Therapy / Attestations

Background on Skysona and Indication