Gene Therapy for the Treatment of Metachromatic Leukodystrophy

POLICY NUMBER

L.5.01.611

Lenmeldy (atidarsagene autotemcel)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Metachromatic leukodystrophy (MLD) is a rare autosomal recessive lysosomal disease that causes progressive central and peripheral nervous system demyelination and is caused by deficient activity of the lysosomal enzyme arylsulfatase A (ARSA).

Lenmeldy (atidarsagene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of children with pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ) or early symptomatic early juvenile (ESEJ) MLD.

POLICY

Lenmeldy (atidarsagene autotemcel) is considered not medically necessary as the long-term durability of treatment is unknown.

Services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary.

POLICY EXCEPTIONS

State Health Plan (State and School Employees) Participants

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

B. appropriate with regard to standards of good medical practice; and

C. not solely for the convenience of the Member, his or her Provider; and

D. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of medical necessity, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

POLICY HISTORY

01/20/2025: New policy added.

12/02/2025: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy description updated regarding metachromatic leukodystrophy and to remove the related medical policy link. Added policy statement that services related to delivery and/or administration of a medication which have not been approved through the BCBSMS PA review process will be considered not medically necessary. Sources updated. Code Reference section updated to add HCPCS code J3391.

SOURCE(S)

1. Bonkowsky JL. Metachromatic leukodystrophy. In: UpToDate, Connor RF (Ed), Wolters Kluwer. (Accessed on September 24, 2025) https://www.uptodate.com/contents/metachromatic-leukodystrophy

2. Lenmeldy prescribing information. Orchard Therapeutics (Europe) LTD. August 2025. Last accessed September 2025.

CODE REFERENCE

This may not be a comprehensive list of procedure codes applicable to this policy.

Not Medically Necessary Codes

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