Elivaldogene Autotemcel (Skysona®)
IMPORTANT REMINDER
We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.
POLICY
I. INDICATIONS
The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.
FDA-Approved Indication
Skysona is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Early, active cerebral adrenoleukodystrophy refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 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 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.
All other indications are considered experimental/investigational and not medically necessary.
II. DOCUMENTATION
Submission of the following information is necessary to initiate the prior authorization review:
Chart notes, medical records, or lab results documenting all of the following:
A. Variant in the ABCD1 gene
B. Elevated very long chain fatty acids (VLCFA) values
C. Active central nervous system (CNS) disease on central radiographic review of brain magnetic resonance imaging (MRI) demonstrating:
1. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
2. Gadolinium enhancement on MRI of demyelinating lesions
D. Neurologic Function Score (NFS) less than or equal to 1
III. EXCLUSIONS
Coverage will not be provided for members with any of the following exclusions:
A. Skysona will be used to treat or prevent adrenal insufficiency.
B. Member has either of the following:
1. Full deletions of the ABCD1 transgene.
2. CALD secondary to head trauma.
IV. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a physician who specializes in the treatment of adrenoleukodystrophy (ALD).
V. CRITERIA FOR INITIAL APPROVAL
Cerebral Adrenoleukodystrophy (CALD)
Authorization of 3 months for a one-time administration may be granted for treatment of cerebral adrenoleukodystrophy (CALD) when all of the following criteria are met:
A. Member must be a male between the ages of 4 and 17 years of age
B. Member has a diagnosis of adrenoleukodystrophy confirmed by both of the following:
1. The presence of a pathogenic (or likely pathogenic) variant in the ABCD1 gene as detected by genetic testing, and
2. Elevated very long chain fatty acids (VLCFA) values per reference range of the laboratory performing the test
C. Member has early active disease as defined by all of the following:
1. Central radiographic review of brain MRI demonstrating both of the following:
a. Loes score between 0.5 and 9 (inclusive) on the 34-point scale, and
b. Gadolinium enhancement on MRI of demyelinating lesions
2. NFS of less than or equal to 1
D. Member is eligible for a hematopoietic stem cell transplant (HSCT) but is unable to find a matched sibling donor
E. Member has not received Skysona or any other gene therapy previously
F. Member has not received a prior allogeneic hematopoietic stem cell transplant (allo-HSCT)
APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS
BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily recognized standard reference compendia or in the published peer-reviewed medical literature.
ADDITIONAL INFORMATION
For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).
REFERENCES
1. Skysona [package insert]. Somerville, MA: Bluebird bio, Inc.; September 2022.
2. Raymond GV, Moser AB, Fatemi A. X-Linked Adrenoleukodystrophy. 1999 Mar 26 [Updated 2023 Apr 6]. In: Adam MP, Everman DB, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2023. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1315/. Accessed November 7, 2023.
ORIGINAL EFFECTIVE DATE: 12/31/2022
MOST RECENT REVIEW DATE: 6/11/2024
ID_CHS
Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.
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