WHA

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) without an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. 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. This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)-free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

    Limitations of Use: SKYSONA does not treat or prevent the development of or treat adrenal insufficiency due to adrenoleukodystrophy. An immune response to SKYSONA may limit the persistence of descendent cells of SKYSONA, causing 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 appropriateness and timing of treatment for each boy, especially for boys with isolated pyramidal tract disease since their clinical symptoms 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 does not have an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant
    • 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
  • Both of the following:
    • Prescribed by a stem cell transplant physician from a qualified treatment center [D, 6]
      • AND
    • Submission of medical records (e.g., chart notes) confirming that prescriber completed a shared decision-making conversation regarding the potential risks of treatment with Skysona, including but not limited to hematologic malignancy
    AND
  • Provider is aware of hematologic malignancy risk and attests they will complete adequate baseline hematologic assessments [e.g., complete blood count with differential, hematopathology review of peripheral blood smear, and bone marrow biopsy (core and aspirate) with flow cytometry] and 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 does not have an available human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant
    • 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
  • Both of the following:
    • Prescribed by a stem cell transplant physician from a qualified treatment center [D, 6]
      • AND
    • Submission of medical records (e.g., chart notes) confirming that prescriber completed a shared decision-making conversation regarding the potential risks of treatment with Skysona, including but not limited to hematologic malignancy
    AND
  • Provider is aware of hematologic malignancy risk and attests they will complete adequate baseline hematologic assessments [e.g., complete blood count with differential, hematopathology review of peripheral blood smear, and bone marrow biopsy (core and aspirate) with flow cytometry] and monitoring as indicated in prescribing information
    • AND
  • Patient has never received Skysona treatment in their lifetime

  • Guideline ID
    GL-374413

  • Formulary
    premium

  • Effective date
    Nov 1, 2025

  • P&T approval date
    Nov 17, 2022

P & T Revisions

2025-09-05, 2024-11-06, 2023-10-12, 2022-11-29, 2022-11-21

  1. Skysona Prescribing Information. Bluebird Bio, Inc. Somerville, MA. August 2025.
  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.
  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]
  • 2025-09-05: Updated criteria in line with latest prescribing information updates.
  • 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