Atidarsagene Autotemcel (Lenmeldy™)
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
Lenmeldy is indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD).
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. PSLI, PSEJ, or ESEJ classification of MLD.
B. Variant(s) in the ARSA gene.
C. Deficiency of arylsulfatase A (ARSA) on biochemical testing.
D. Elevated sulfatide levels based on 24-hour urine collection, if applicable.
III. PRESCRIBER SPECIALTIES
This medication must be prescribed by or in consultation with a physician who specializes in the treatment of metachromatic leukodystrophy (MLD).
IV. CRITERIA FOR INITIAL APPROVAL
Metachromatic Leukodystrophy (MLD)
Authorization of 3 months for a one-time administration may be granted for treatment of metachromatic leukodystrophy (MLD) when all of the following criteria are met:
A. Member must have one of the following types of MLD:
1. Pre-symptomatic late infantile (PSLI).
2. Pre-symptomatic early juvenile (PSEJ).
3. Early symptomatic early juvenile (ESEJ).
B. The diagnosis was confirmed by all of the following:
1. Biochemical testing documenting ARSA activity below the normal range for the laboratory performing the test.
2. The presence of two disease-causing ARSA alleles, either known or novel mutations, identified on genetic testing.
3. If novel mutations are identified, a 24-hour urine collection showing elevated sulfatide levels.
C. Member has not received Lenmeldy or any other gene therapy previously.
D. Member does not have evidence of residual cells of donor origin if the member has 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. Lenmeldy [package insert]. Boston, MA: Orchard Therapeutics North America.; March 2024.
2. Gomez-Ospina N. Arylsulfatase A Deficiency. 2006 May 30 [Updated 2024 Feb 8]. In: Adam MP, Feldman J, Mirzaa GM, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1130/. Accessed March 19, 2024.
ORIGINAL EFFECTIVE DATE: 7/2/2024
MOST RECENT REVIEW DATE: 7/2/2024
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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