Pharmacy Prior Authorization Criteria

Skysona (elivaldogene autotemcel suspension) - PA, NF

Indications for Prior Authorization

Skysona (elivaldogene autotemcel suspension)

• For diagnosis of Cerebral Adrenoleukodystrophy (CALD)
  Indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Early, active CALD refers to asymptomatic or mildly symptomatic (neurologic function score, NFS less than or equal to 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.

  Limitations of Use: SKYSONA does not treat or prevent adrenal insufficiency. An immune response to SKYSONA may cause rapid loss of efficacy of SKYSONA in patients with full deletions of the human adenosine triphosphate binding cassette, sub family D, member 1 (ABCD1) gene. SKYSONA has not been studied in CALD secondary to head trauma. Given the risk of hematologic malignancy with SKYSONA, and unclear long-term durability of SKYSONA and human adrenoleukodystrophy protein (ALDP) expression, careful consideration should be given to the timing of treatment for each boy and treatment of boys with isolated pyramidal tract disease as clinical manifestations do not usually occur until adulthood.

Criteria

Skysona

Prior Authorization

Length of Approval: 1 Time Authorization in Lifetime

• Diagnosis of early, active cerebral adrenoleukodystrophy (CALD)
AND
• Molecular genetic testing confirms mutation in the ABCD1 gene
AND
• ALL of the following:
• Patient has elevated very long chain fatty acid (VLCFA) levels
• Loes score between 0.5 and 9 (inclusive) based on brain MRI assessment [B, 4]
• Brain magnetic resonance imaging (MRI) utilizes Gadolinium enhancement (GdE +) and demonstrates demyelinating lesions [C, 5]
• Neurologic function score (NFS) less than or equal to 1
AND
• BOTH of the following:
• Patient is male sex
• Patient is 4 to 17 years of age
AND
• Patient is not eligible for an allogeneic hematopoietic stem cell transplant with an HLA-matched sibling donor
AND
• Patient has obtained a negative test result for all of the following prior to cell collection:
• Hepatitis B virus (HBV)
• Hepatitis C virus (HCV)
• Human T-lymphotropic virus 1 and 2 (HTLV-1/HTLV-2)
• Human immunodeficiency virus (HIV)
AND
• Patient does not have CALD secondary to head trauma [A, 1]
AND
• Discontinue prophylactic anti-retroviral medications (e.g., Truvada, Descovy) for at least one month prior to initiating medications for stem cell mobilization and until all cycles of apheresis are completed
AND
• Prescribed by a stem cell transplant physician from a qualified treatment center [D, 6]
AND
• Provider is aware of hematologic malignancy risk and attests they will complete adequate monitoring as indicated in prescribing information
AND
• Patient has never received Skysona treatment in their lifetime

Skysona

Non Formulary

Length of Approval: 1 Time Authorization in Lifetime

• Diagnosis of early, active cerebral adrenoleukodystrophy (CALD)
AND
• Submission of medical records (e.g., chart notes) documenting molecular genetic testing confirms mutation in the ABCD1 gene
AND
• Submission of medical records (e.g., chart notes) documenting ALL of the following:
• Patient has elevated very long chain fatty acid (VLCFA) levels
• Loes score between 0.5 and 9 (inclusive) based on brain MRI assessment [B, 4]
• Brain magnetic resonance imaging (MRI) utilizes Gadolinium enhancement (GdE +) and demonstrates demyelinating lesions [C, 5]
• Neurologic function score (NFS) less than or equal to 1
AND
• BOTH of the following:
• Patient is male sex
• Patient is 4 to 17 years of age
AND
• Submission of medical records (e.g., chart notes) documenting patient is not eligible for an allogeneic hematopoietic stem cell transplant with an HLA-matched sibling donor
AND
• Submission of medical records (e.g., chart notes) documenting patient has obtained a negative test result for all of the following prior to cell collection:
• Hepatitis B virus (HBV)
• Hepatitis C virus (HCV)
• Human T-lymphotropic virus 1 and 2 (HTLV-1/HTLV-2)
• Human immunodeficiency virus (HIV)
AND
• Patient does not have CALD secondary to head trauma [A, 1]
AND
• Discontinue prophylactic anti-retroviral medications (e.g., Truvada, Descovy) for at least one month prior to initiating medications for stem cell mobilization and until all cycles of apheresis are completed
AND
• Prescribed by a stem cell transplant physician from a qualified treatment center [D, 6]
AND
• Provider is aware of hematologic malignancy risk and attests they will complete adequate monitoring as indicated in prescribing information
AND
• Patient has never received Skysona treatment in their lifetime

P & T Revisions

2024-11-06, 2023-10-12, 2022-11-29, 2022-11-21

References

1. Skysona Prescribing Information. Bluebird Bio, Inc. Somerville, MA. April 2024.
2. Eichler, F., Duncan, C., et al. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy. N Engl J Med 2017; 377:1630-1638. Available at DOI: 10.1056/NEJMoa1700554. Accessed October 13, 2022.
3. Rare Disease Database. X-Linked Adrenoleukodystrophy. Available at https://rarediseases.org/rare-diseases/adrenoleukodystrophy/. Accesed October 13,2022.
4. Kumar, S., Sait. H., et al. Loes Score: Clinical and Radiological Profile of 22 Patients of X-Linked Adrenoleukodystrophy: Case Series from a Single Center. Indian J Radiol Imaging 2021;31:383–390. Available at https://www.thieme-connect.com/products/ejournals/pdf/10.1055/s-0041-1734366.pdf. Accessed October 18, 2022.
5. Ibrahim, M., Hazhirkarzar, B., et al. Gadolinium Magnetic Resonance Imaging. Available at https://www.ncbi.nlm.nih.gov/books/NBK482487/. Accessed October 18, 2022.
6. Bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD). Available at https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-receives-fda-accelerated-approval-skysonar-gene. Accessed October 18, 2022.
7. Per clinical consult with pediatric hematologist/oncologist. October 18, 2022.
8. Engelen, M., vanBallegoij, W., et al. International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy. Available at: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9687408/. Accessed October 12, 2023.

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End Notes

1. SKYSONA has not been studied in CALD secondary to head trauma. [1]
2. Loes is a 34-point imaging-based scoring system for patients with X-Linked Adrenoleukodystrophy to assess neuroanatomical involvement and the presence or absence of focal or global atrophy. The score helps in the prediction of disease course and selection of patients for HSCT. [4]
3. Gadolinium is a contrast-agent used to enhance MRI imaging of the CNS. [5]
4. Bluebird anticipates that commercial product will be available by the end of 2022 through a limited number of Qualified Treatment Centers (QTCs) in the United States, including Boston Children’s Hospital and Children’s Hospital of Philadelphia. [6]

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Revision History

• 2024-11-06: 2024 Annual Review. Updated guideline. Updated references.
• 2023-10-12: 2023 Annual Review
• 2022-11-29: update guideline
• 2022-11-21: New UM PA Criteria

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