CurrentBlue Cross Blue Shield - IowaPolicy 05.05.47

Lenmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy

Defines prior authorization, coverage criteria, dosing limits, and billing for Lenmeldy (atidarsagene autotemcel) for treatment of specific subtypes of metachromatic leukodystrophy (MLD) in pediatric patients when benefit exists under member contract.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyLenmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy

Policy CodePolicy 05.05.47

Change TypeNo material changes

Effective DateAug 26, 2024

Next Review Date

Key ActionSubmit prior authorization with documentation confirming MLD subtype, ARSA gene variants, biochemical ARSA deficiency, and absence of prior gene therapy.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

one lifetimetreatment course limit

3 monthsauthorization duration

minimums definedCD34+ threshold

pediatric neurologistprescriber

Coverage Criteria for Lenmeldy (atidarsagene autotemcel)

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Initial therapy (one-time)

Covered when ALL of the following are met:

Primary coverage criteria: Member has a diagnosis of Pre-symptomatic late infantile (PSLI), Pre-symptomatic early juvenile (PSEJ), or Early symptomatic early juvenile (ESEJ) metachromatic leukodystrophy (MLD) as defined in the policy; diagnosis confirmed by biochemical testing documenting ARSA activity below the laboratory normal range and presence of two disease-causing ARSA alleles (known or novel) on genetic testing; if novel ARSA mutations are identified, elevated 24-hour urine sulfatide levels are required; member has not previously received Lenmeldy or any other gene therapy; member does not have malignant neoplasia (except local skin cancer), a documented hereditary cancer syndrome, myelodysplasia/AML-related cytogenetic alterations, or other serious hematologic disorders; no evidence of residual donor cells if prior allogeneic HSCT; no symptomatic uncontrolled infections (including active/uncontrolled HBV or HCV) or symptomatic herpes zoster not responsive to treatment; member is not enrolled in any interventional trials for MLD; member is not late-juvenile, adult-onset, or other non-eligible forms of MLD.all conditions met

Authorization of 3 months for a one-time administration may be granted.

Continuation of therapy

Continuation/Repeat therapy stance:

Repeat therapy: Repeat treatment of Lenmeldy for any indication is considered investigational; approvals are limited to one treatment course per lifetime.no repeat allowed

Lenmeldy is considered not medically necessary for members who do not meet the specified criteria or for repeat treatments beyond one dose.

Coverage excludes members with late-juvenile, adult-onset, or other forms of metachromatic leukodystrophy (MLD) outside of PSLI, PSEJ, and ESEJ. The policy limits eligibility to the defined pediatric subtypes: pre-symptomatic late infantile (PSLI), pre-symptomatic early juvenile (PSEJ), and early symptomatic early juvenile (ESEJ).

Lenmeldy (atidarsagene autotemcel) is not medically necessary for members who do not meet the specified coverage criteria. Additionally, repeat treatment beyond a single lifetime administration is not covered; approvals are limited to one treatment course per lifetime because safety and efficacy beyond one dose have not been established.

Coding and Minimum Cell Dose Requirements

Covered HCPCS Biologic CodeHCPCSCovered

J3391

Inj, atidarsagene autotemcel (effective 7/1/2025)

Unclassified biologicsHCPCS

J3590

Unclassified biologics (when specified as [Lenmeldy] (atidarsagene autotemcel))

Unclassified drugsHCPCS

J3490

Unclassified drugs (when specified as [Lenmeldy] (atidarsagene autotemcel))

Unclassified drugs/biologics (revenue or other)mixed

C9399

Unclassified drugs or biologics (when specified as [Lenmeldy] (atidarsagene autotemcel))

Minimum CD34+ cells/kg by MLD subtype

PSLI (Pre-symptomatic late infantile)Minimum CD34+ cells/kg = 4.2 x10^6

PSEJ (Pre-symptomatic early juvenile)Minimum CD34+ cells/kg = 9 x10^6

ESEJ (Early symptomatic early juvenile)Minimum CD34+ cells/kg = 6.6 x10^6

Provider Requirements and Prior Authorization

Prior Authorization

Prior Authorization Required

Prior authorization is required. Submit documentation confirming MLD subtype, ARSA gene variant(s), biochemical deficiency, and other required materials listed below to initiate review.

Prior authorization required before Lenmeldy (one-time administration) will be authorized.
Documentation must confirm MLD subtype (PSLI, PSEJ, or ESEJ), ARSA mutation results, and biochemical evidence of ARSA deficiency or elevated sulfatides when applicable.

Step Therapy

Preparative / Step Therapy Requirements

Providers must complete preparative steps prior to infusion. Lenmeldy is autologous, single-dose cell suspension given once. Children must undergo HSC mobilization and apheresis to collect CD34+ cells for manufacturing. A myeloablative conditioning regimen must be administered before infusion. Approvals may be subject to dosing limits per FDA labeling and practice guidelines.

Autologous HSC mobilization and apheresis to obtain CD34+ cells is required.
Myeloablative conditioning must be administered prior to Lenmeldy infusion.
Lenmeldy is recommended as a one-time, single-dose intravenous infusion; dosing based on CD34+ cells/kg and subtype-specific minimums.

Documentation Required

Required Documentation for Prior Authorization

Submission of the following information is necessary to initiate the prior authorization review: chart notes, medical records, or lab results documenting all required items.

Documentation of MLD subtype classification (Pre-symptomatic late infantile [PSLI], Pre-symptomatic early juvenile [PSEJ], or Early symptomatic early juvenile [ESEJ]).
Genetic testing results showing two disease-causing ARSA alleles (identified variants).
Biochemical testing documenting ARSA activity below the laboratory's normal range.
If novel ARSA mutations are identified, a 24-hour urine collection demonstrating elevated sulfatide levels, when applicable.
Prescribing clinician specialty: prescription by or in consultation with a pediatric neurologist with expertise in MLD.
Medical history confirming member has not previously received Lenmeldy or any other gene therapy and lacks exclusionary conditions (e.g., active uncontrolled infections, malignant neoplasia as specified, recent allo-HSCT with residual donor cells, enrollment in interventional MLD trials).

Denial Risk

Repeat Treatment Denial Risk

Repeat treatment beyond a single course is not authorized. Safety and efficacy have not been established for repeated dosing.

Repeat treatment of Lenmeldy for any indication is considered investigational and will be denied.
Approval is limited to one treatment course per member lifetime.

MLD Subtype Definitions and Clinical Criteria

Pre-symptomatic late infantile (PSLI) definition

Expected age of onset< 30 months

Genotype requirementARSA genotype consistent with PSLI

Pre-symptomatic clinical criteriaAbsence of neurological signs or physical exam limited to abnormal reflexes and/or clonus

Pre-symptomatic early juvenile (PSEJ) definition

Expected age of onset> 30 months and < 7 years

Genotype requirementARSA genotype consistent with early juvenile (EJ) MLD

Pre-symptomatic clinical criteriaAbsence of neurological signs or physical exam limited to abnormal reflexes and/or clonus

Early symptomatic early juvenile (ESEJ) definition

Age of onset> 30 months and < 7 years

Functional walking statusWalking independently (GMFC-MLD Level 0 with ataxia or GMFC-MLD Level 1)

Cognitive requirementIQ ≥ 85

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyLenmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy

Policy CodePolicy 05.05.47

Change TypeNo material changes

Effective DateAug 26, 2024

Next Review Date

Key ActionSubmit prior authorization with documentation confirming MLD subtype, ARSA gene variants, biochemical ARSA deficiency, and absence of prior gene therapy.

SourceLink

On This Page

OpenPayer

Lenmeldy (atidarsagene autotemcel) for metachromatic leukodystrophy

Coverage Criteria for Lenmeldy (atidarsagene autotemcel)

Coding and Minimum Cell Dose Requirements

Provider Requirements and Prior Authorization

Background on Lenmeldy and Indication

MLD Subtype Definitions and Clinical Criteria