Atidarsagene autotemcel (Lenmeldy) — coverage criteria for metachromatic leukodystrophy
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Medical policy governing coverage criteria, dosing, and administrative requirements for atidarsagene autotemcel (Lenmeldy) intravenous infusion for children with specific subtypes of metachromatic leukodystrophy (MLD). Affects pediatric patients and providers requesting coverage from Blue Cross NC.
Added revenue codes 0891 and 0892 associated with policy HCPCS code(s).
For PSLI and PSEJ MLD subtypes, absence of symptoms wording adjusted to specify neurologic symptoms not associated with functional impairment.
HCPCS code J3391 was added to the dosing reference table; C9399, J3490, and J3590 were deleted (termed).
Coverage Criteria for atidarsagene autotemcel (Lenmeldy)
Initial therapy
Covered when ALL of the following are met:
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
Disease subtype (one required)
- PSLI: Expected disease onset less than or equal to 30 months; ARSA genotype consistent with late infantile MLD (two null [0] mutant ARSA alleles); absence of neurological signs and symptoms of MLD associated with functional impairment
[medical record documentation required]
- PSEJ: Expected disease onset between 30 months and 7 years (has not celebrated 7th birthday); ARSA genotype consistent with early juvenile MLD (one null [0] and one residual [R] allele); absence of neurological signs and symptoms associated with functional impairment or physical findings limited to abnormal reflexes and/or clonus
[medical record documentation required]
- ESEJ: Disease onset between 30 months and 7 years (has not celebrated 7th birthday); ARSA genotype consistent with early juvenile MLD (one null [0] and one residual [R] allele); BOTH walking independently (GMFC-MLD score 0 with ataxia or score 1) AND normal cognitive function (IQ ≥ 85)
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
[medical record documentation required]
Duration of approval: 180 days; one treatment course per lifetime
Coverage excludes patients with late juvenile- or adult-onset metachromatic leukodystrophy (MLD). Patients are also ineligible if they have undergone prior allogeneic hematopoietic stem cell transplantation, have evidence of donor-origin residual cells, or have had any prior gene therapy. These exclusion conditions must be documented in the medical record.
Requests that exceed the FDA‑labeled dosing or quantity limits for the indicated use are not covered. Additionally, requests for patients who do not meet the required documented diagnostic criteria or subtype-specific eligibility criteria (as evidenced in the medical record) are considered not medically necessary.
Dosing and Regimen
| Regimen | Dose / cell dose | Maximum units | HCPCS / Revenue codes |
|---|---|---|---|
| One-time autologous lentiviral hematopoietic stem‑cell gene therapy (atidarsagene autotemcel, Lenmeldy) — IV single‑dose infusion for PSLI, PSEJ, or ESEJ MLD in children | |||
| Minimum recommended cell dose by subtype: PSLI 4.2 x 10^6 CD34+ cells/kg; PSEJ 9 x 10^6 CD34+ cells/kg; ESEJ 6.6 x 10^6 CD34+ cells/kg; Maximum recommended dose 30 x 10^6 CD34+ cells/kg | |||
| 1 (one treatment course per lifetime) | |||
| HCPCS: J3391; Revenue codes (may be applicable): 0891, 0892 |
Billing Codes and Dose Limits
| J3391 | atidarsagene autotemcel (Lenmeldy) HCPCS code |
| 0891 | Revenue code: Special Processed Drugs - FDA Approved Cell Therapy |
| 0892 | Revenue code: Special Processed Drugs - FDA Approved Gene Therapy |
Provider Requirements and Authorization
Prior Authorization and Dosing Verification
Prior authorization is required. The prescriber must document that the requested dose is within FDA‑labeled dosing for the indication and the requested quantity does not exceed the maximum units allowed for the duration of approval. HCPCS J3391 applies.
- Prior authorization required
- Prescriber must document requested dose within FDA‑labeled dosing
- HCPCS code: J3391
Medical Record Documentation Required
Medical record documentation is required to support the diagnosis and eligibility for treatment. Include documentation of: ARSA enzyme activity, ARSA genotyping showing biallelic pathogenic mutations (or explanation and urine sulfatides if novel variants), and any other diagnostic testing referenced below.
- ARSA enzyme activity (peripheral blood mononuclear cells/leukocytes or fibroblasts)
- Biallelic ARSA pathogenic mutations (genotype)
- If novel ARSA variant(s): 24‑hour urine sulfatide collection showing elevated sulfatides
- Documentation of diagnosis of metachromatic leukodystrophy (MLD)
Denial Risk — Missing Documentation
Requests that do not include the required medical record documentation may be denied. Missing or incomplete documentation may include lack of ARSA enzyme activity results, absent ARSA genotyping confirming biallelic pathogenic mutations, no urine sulfatide testing when novel variants are present, or missing evidence of subtype, functional status, transplant candidacy, infection screening, or prior gene/cell therapy history.
- Absent ARSA enzyme activity results
- No documentation of biallelic pathogenic ARSA mutations
- No 24‑hour urine sulfatide results when novel variant(s) present
- Missing evidence of disease subtype or functional status
- No documentation of transplant candidacy, infection screening, or prior gene/cell therapy history
Step Therapy and Treatment Course
No step therapy is specified. Coverage is for a single one‑time treatment course per lifetime; duration of approval is 180 days (one treatment course per lifetime).
- No step therapy required
- Single one‑time treatment course per lifetime
- Duration of approval: 180 days
Diagnostic and Laboratory Requirements
Clinical Definitions Used in Criteria
Background and Indication
Metachromatic leukodystrophy (MLD) is an inherited deficiency of arylsulfatase A (ARSA) that leads to progressive neurologic decline. Lenmeldy (atidarsagene autotemcel) is an autologous lentiviral hematopoietic stem‑cell gene therapy indicated for children with specific MLD subtypes — presymptomatic late infantile (PSLI), presymptomatic early juvenile (PSEJ), or early symptomatic early juvenile (ESEJ). The therapy is administered as a one‑time intravenous infusion following myeloablative conditioning and requires careful pre‑treatment screening and documentation (including ARSA enzyme activity, ARSA genotyping, infection screening, and transplant candidacy).
Policy Changes and Revision History
Added revenue codes 0891 (Special Processed Drugs - FDA Approved Cell Therapy) and 0892 (Special Processed Drugs - FDA Approved Gene Therapy); policy notification given 2026-02-01 for effective date 2026-04-01.
Revised wording for PSLI and PSEJ MLD subtypes to specify 'absence of neurologic symptoms not associated with functional impairment'.
Added HCPCS code J3391 to the dosing reference table effective 2025-07-01; C9399, J3490, and J3590 were deleted as of 2025-06-30.
Original medical policy criteria issued.
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