Skysona (elivaldogene autotemcel) — Prior authorization and coverage criteria
Customize your policy alerts
Sign up for all CareSource policy alerts
Know when CareSource releases new policies or updates existing guidance.
Monitor payer policy activity
Defines prior authorization and medical necessity criteria for Skysona (elivaldogene autotemcel) as a one-time gene therapy for boys 4–17 years with early, active cerebral adrenoleukodystrophy (CALD); applies to the payer's pharmacy/medical benefit in Arkansas PASSE and affects prescribers and prior authorization reviewers.
Changed 'matched sibling donor' to 'an available human leukocyte antigen (HLA)-matched donor' to match updated indication wording.
Added restriction for family history of a familial cancer syndrome and added specific qualifying values to CBC from clinical trial due to increased reporting of hematologic malignancy.
Coverage Criteria for Skysona (elivaldogene autotemcel)
Initial Therapy
Covered when ALL of the following are met:
If all requirements are met, approve for 3 months
Reauthorization / Continuation
Reauthorization
CareSource considers Skysona (elivaldogene autotemcel) not medically necessary for use in any condition not expressly listed in this policy. In addition, Skysona is not indicated for members who have an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant (HSCT); such members should be evaluated for HSCT instead of gene therapy. Requests that do not meet the specified eligibility criteria (including age, confirmatory testing, disease activity, infectious disease screening, CBC thresholds, absence of immediate family familial cancer syndrome, and no prior allogeneic transplant or gene therapy) will be considered not medically necessary and may be denied.
If all initial authorization criteria are met, prior authorization will be approved for 3 months; Skysona is a one-time therapy and ongoing/repeated authorizations are not applicable for continued treatment.
Skysona is considered not medically necessary for any use not described in this policy. For any indication outside this document, refer to the payer's Off‑Label policy for coverage determination. Additional exclusions include members with a prior allogeneic transplant or prior gene therapy and those with an immediate family member with a known or suspected familial cancer syndrome; such situations preclude coverage under this policy.
Initial Authorization Criteria
Initial Therapy
Initial authorization criteria for Skysona
If all requirements are met, approve for 3 months
Continuation / Reauthorization Criteria
Continuation Therapy
Continuation/Reauthorization
Key Clinical Thresholds and Coding Values
Provider Actions, Documentation, and Denial Triggers
Prior authorization required; approvals valid 3 months
Prior authorization is required for Skysona (elivaldogene autotemcel). If all initial authorization criteria are met, the request will be approved for a 3-month duration.
No step therapy specified
No step therapy is specified for Skysona. Prior authorization must still be obtained and all initial criteria met for approval.
Required supporting documentation to verify all initial criteria
Submit documentation that demonstrates the member meets all initial authorization criteria, including diagnostic, laboratory, imaging, and screening records.
- Member age (male 4–17 years)
- Prescription by or documentation of consultation with a neurologist, endocrinologist, or hematologist/oncologist
- Laboratory report showing elevated very long‑chain fatty acids (VLCFA)
- Genetic testing report confirming ABCD1 mutation
- Brain MRI report documenting gadolinium-enhancing demyelinating lesions and Loes score (0.5–9)
- Neurologic Function Score (NFS) showing ≤ 1
- Documentation that no available HLA‑matched donor exists for allogeneic HSCT
- Infectious disease screening results: negative for HBV, HCV, HIV‑1/2, HTLV‑1/2
- Complete blood count (CBC) with ANC ≥ 1,500 cells/mm3, platelets ≥ 100,000 cells/mm3, hemoglobin ≥ 10 g/dL
- Family history assessment confirming no immediate family member with known or suspected familial cancer syndrome
- History confirming no prior allogeneic transplant or prior gene therapy
- Dose/quantity documentation (single dose; minimum 5.0 × 10^6 CD34+ cells/kg; QL: two 20 mL bags)
Denial triggers if initial criteria are not met
Deny requests that fail to meet any of the initial authorization requirements, including age, specialty prescriber/consult, diagnostic confirmation (elevated VLCFA plus ABCD1 mutation), early active disease findings (NFS ≤1, gadolinium enhancement, Loes 0.5–9), absence of an available HLA‑matched donor, negative infectious disease screens, CBC values below specified thresholds, presence of an immediate family familial cancer syndrome, or prior allogeneic transplant/gene therapy.
- Age not within 4–17 years (male)
- Medication not prescribed by or in consultation with required specialists
- Missing or negative VLCFA elevation or absent ABCD1 mutation report
- MRI lacking gadolinium-enhancing demyelinating lesions or Loes score outside 0.5–9
- Neurologic Function Score > 1
- An available HLA‑matched donor exists for allogeneic HSCT
- Positive HBV, HCV, HIV‑1/2, or HTLV‑1/2 screen
- CBC values below ANC < 1,500 cells/mm3, platelets < 100,000 cells/mm3, or hemoglobin < 10 g/dL
- Immediate family member with known or suspected familial cancer syndrome
- History of prior allogeneic transplant or prior gene therapy
Definitions and Product Notes
Background
Cerebral adrenoleukodystrophy (CALD) is an X‑linked disorder caused by mutations in the ABCD1 gene that lead to accumulation of very long‑chain fatty acids (VLCFA) and an inflammatory demyelinating process in the brain. CALD typically affects boys and can progress rapidly from asymptomatic or mildly symptomatic disease to irreversible neurologic decline. Early, active CALD is characterized by NFS ≤ 1, gadolinium‑enhancing demyelinating lesions on brain MRI, and a Loes score of 0.5–9. Hematopoietic stem cell transplant (HSCT) can halt progression when an appropriate HLA‑matched donor is available; when no such donor exists, autologous gene therapy with Skysona (elivaldogene autotemcel) adds functional copies of ABCD1 to patient stem cells and is indicated as a one‑time treatment for boys aged 4–17 with early, active CALD.
Skysona carries important safety considerations, including a boxed warning for hematologic malignancy observed in trials and the need for baseline infectious disease screening and specific CBC thresholds (absolute neutrophil count ≥ 1,500 cells/mm3, platelets ≥ 100,000 cells/mm3, hemoglobin ≥ 10 g/dL) prior to administration.
Step Therapy
| Step | Requirement / Note |
|---|---|
| 1 | No step therapy is specified for Skysona (elivaldogene autotemcel). Prior authorization is required; requests must meet the program's initial authorization criteria to be approved (see coverage criteria). If all initial criteria are met, authorization is approved for 3 months. |
Quantity Limits and Dosing
Site of Care
Site of care not specified
Policy does not specify a required site of care for Skysona; hospital outpatient is listed but no site restriction is mandated.
OpenPayer is powered by Trek Health's payer performance platform. Trek continuously ingests, validates, and normalizes Transparency in Coverage data alongside payer policies and other commercial payer data to create a structured payer intelligence foundation. OpenPayer uses this foundation to deliver personalized search results, dynamically generated policy pages, and tailored policy monitoring based on each user's payers, specialties, billing codes, and areas of interest. The same intelligence powers broader payer performance workflows, including reimbursement benchmarking, contract evaluation, payer negotiations, and financial decision-making.