Description

Skysona (elivaldogene autotemcel) is a lentiviral vector (LVV)-based autologous hematopoietic stem cell (HSC) gene therapy designed to add functional copies of the ABCD1 complementary deoxyribonucleic acid (DNA) into HSCs through transduction of autologous CD34+ cells with Lenti-D LVV. After Skysona (elivaldogene autotemcel) infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate into various cell types, including monocytes (CD14+) capable of producing functional human adrenoleukodystrophy protein (ALDP). Functional ALDP can then participate in the local degradation of very long chain fatty acids (VLCFAs), which is believed to slow or possibly prevent further inflammation and demyelination.

Skysona (elivaldogene autotemcel) is indicated, as a single dose per lifetime, to slow the progression of neurological dysfunction in male pediatric patients diagnosed with cerebral adrenoleukodystrophy (CALD), a rare neurologic disease caused by mutations in the ABCD1 gene that leads to a buildup of VLCFA causing inflammation and damage in the brain.

Skysona (elivaldogene autotemcel) Effective Dates

• Effective Date: 12/14/2023
• Revision Date: 12/14/2023
• Review Date: 12/14/2023

Skysona (elivaldogene autotemcel) is a cell suspension for intravenous infusion. A single dose of Skysona contains a minimum of 5.0 × 106 CD34+ cells/kg of body weight, suspended in a solution containing 5% dimethyl sulfoxide.

Individuals must undergo HSC mobilization and apheresis to obtain CD34+ cells for Skysona (elivaldogene autotemcel) manufacturing. Dosing is based on the number of CD34+ cells in the infusion bag(s) per kg of body weight. Full myeloablative and lymphodepleting conditioning must be administered prior to infusion of Skysona (elivaldogene autotemcel).

Coverage Determination

Refer all requests or questions regarding Skysona (elivaldogene autotemcel) to the Corporate Transplant Department at 1‐866‐421‐5663, Fax: 502‐508‐9300 or Email: transplant@humana.com.

Requests for Skysona (elivaldogene autotemcel) require review by a medical director.

Humana members may be eligible under the Plan for Skysona (elivaldogene autotemcel) when ALL the following criteria are met:

• Absence of contraindications; AND
• Individual has a diagnosis of CALD that is asymptomatic or mildly symptomatic confirmed by all of the following:
  1. Elevated VLCFA; AND
  2. Gadolinium enhancement on magnetic resonance imaging (MRI) of the brain demonstrating demyelinating lesions; AND
  3. Loes score of 0.5 to 9 on the 34-point scale; AND
  4. Neurologic function score (NFS) less than or equal to 1;

Refer to Medical and Pharmacy Coverage Policies to verify that this is the current version before utilizing.

• Individual is male and between 4 through 17 years of age; AND
• Individual will receive 1 dose per lifetime; AND
• Individual will receive Skysona (elivaldogene autotemcel) at a certified treatment center; AND
• Documentation that a HLA matched sibling donor is unavailable for an allogeneic HCT; AND
• Pathogenic variant in the ABCD1 gene

Coverage Limitations

Humana members may NOT be eligible under the Plan for Skysona (elivaldogene autotemcel) for any indications other than those listed above including, but may not be limited to:

• Any contraindications to hematopoietic stem cell transplant (HSCT) including, the use of granulocyte colony stimulating (G-CSF) during the mobilization of HSCs, and any contraindications to the use of busulfan or cyclophosphamide, including known hypersensitivity to the active substances or to any of the excipients in their formulations; OR
• Availability of a HLA matched sibling donor; OR
• CALD secondary to head trauma; OR
• Clinically active bacterial, fungal, parasitic, prion-associated or viral infection; OR
• Clinically significant cardiovascular, pulmonary disease or other major condition that would be contraindicated, including, but may not be limited to:
  • Cardiac compromise as evidenced by left ventricular ejection fraction less than 40 percent; OR
  • Hematological compromise as evidenced by any of the following:
    • Hemoglobin less than 10 gram per deciliter (g/dL); OR
    • Peripheral blood absolute neutrophil count (ANC) count less than 1500 cells/cubic millimeter (mm3); OR
    • Platelet count less than 100,000 cells/mm3; OR
    • Uncorrected bleeding disorder; OR
  • Hepatic compromise as evidenced by any of the following:
    • Alanine transaminase (ALT) value greater than 2.5×upper limit of normal (ULN); OR
    • Aspartate transaminase (AST) value greater than 2.5×ULN; OR
    • Total bilirubin value greater than 3.0 milligram per deciliter (mg/dL), except if there is a diagnosis of Gilbert's Syndrome and the individual is otherwise stable; OR
  • Renal compromise as evidenced by abnormal renal function (actual or calculated creatinine clearance less than 50 milliliter per minute [mL/min]); OR
• Current use of dietary regimens, Lorenzo's Oil or statins to lower VLCFA levels; OR
• Current use of investigational study drugs; OR
• First-degree relative with a known or suspected familial cancer syndrome (including, but not limited to hereditary breast and ovarian cancer syndrome, hereditary non-polyposis colorectal cancer syndrome or familial adenomatous polyposis); OR
• Positive for any of the following:
  • Hepatitis B (if individual has been vaccinated against hepatitis B, the individual must test negative for other markers of prior hepatitis B infection [eg, negative for hepatitis B core antibody].

• An individual with past exposure to hepatitis B virus must test negative for Hepatitis B DNA); OR
• Hepatitis C (if an individual is positive for anti-hepatitis C antibody, the individual must have a negative hepatitis C viral load); OR
• Human immunodeficiency virus type 1 or 2 (HIV-1, HIV-2); OR
• Human T lymphotrophic virus 1 (HTLV-1); OR
• Prevent or treat adrenal insufficiency due to adrenoleukodystrophy; OR
• Prior allogeneic HSCT or gene therapy

These are considered experimental/investigational as they are not identified as widely used and generally accepted for any other proposed uses as reported in nationally recognized peer-reviewed medical literature published in the English language.

Background

Skysona (elivaldogene autotemcel) is only available at certain centers.

Warnings and Precautions:

• Delayed platelet engraftment: monitor individuals for thrombocytopenia and bleeding until platelet engraftment and count recovery
• Hematologic malignancy, including life-threatening cases of myelodysplastic syndrome, has occurred in individuals treated with Skysona (elivaldogene autotemcel).
• Prolonged cytopenias: individuals may exhibit cytopenias greater than 1 year after treatment with Skysona (elivaldogene autotemcel). Monitor individuals for bleeding and infection
• Risk of neutrophil engraftment failure: monitor absolute neutrophil counts and if neutrophil engraftment does not occur, give rescue cells
• Serious infections: life-threatening bacterial and viral infections may occur. Monitor individuals for signs and symptoms of infection

Additional Information

Additional information about CALD may be found from the following websites:

• National Library of Medicine
• National Organization for Rare Disorders
• US Food & Drug Administration

Medical Alternatives

Alternatives to Skysona (elivaldogene autotemcel) include, but may not be limited to:

• Allogenic HSCT

Physician consultation is advised to make an informed decision based on an individual's health needs.

Humana may offer a disease management program for this condition. The member may call the number on his/her identification card to ask about our programs to help manage his/her care.

Note:

Any CPT, HCPCS or ICD codes listed on this medical coverage policy are for informational purposes only. Do not rely on the accuracy and inclusion of specific codes. Inclusion of a code does not guarantee coverage and or reimbursement for a service or procedure.