CurrentUnitedHealthcarePolicy 202400063J

Luxturna (voretigene neparvovec-rzyl)

Medical benefit drug policy for Luxturna (voretigene neparvovec-rzyl) describing coverage rationale, documentation requirements, applicable codes, clinical evidence, and administration/benefit considerations for members under UnitedHealthcare standard plans.

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna (voretigene neparvovec-rzyl)

Policy CodePolicy 202400063J

Change TypeTemplate update; Clinical evidence/reference revision

Effective DateNov 1, 2024

Next Review Date

Key ActionObtain prior authorization (one lifetime treatment per eye, valid up to 45 days) and submit required clinical documentation including OCT (>100 µm), genetic test confirming biallelic RPE65 mutations, age, and provider credentialing.

POLICY UPDATE CHANGES

Template Update - Updated Benefit Considerations section.

Supporting Information updated in Clinical Evidence and References sections.

1Lifetime treatments allowed per eye

>12 monthsMinimum patient age

>100 µmOCT thickness threshold

J3398Primary HCPCS code

Coverage Rationale / Medical Necessity Criteria

Covered when ALL of the following are met:

ALL of the following

Age: Patient is greater than 12 months of age> 12 months
Diagnosis of confirmed biallelic RPE65 mutation-associated retinal dystrophy (e.g., Leber's congenital amaurosis, retinitis pigmentosa, early onset severe retinal dystrophy)
Viable retinal cells: Sufficient viable retinal cells as determined by optical coherence tomography (OCT) confirming an area of retina within the posterior pole of > 100 µm thickness> 100 µm thickness
Viable retinal cells may alternatively be assessed by ophthalmoscopy per treating physician assessment
Genetic testing documenting biallelic mutations of the RPE65 gene
Prescriber: Prescribed and administered by an ophthalmologist or retinal surgeon with experience providing sub-retinal injections
Prior treatment: Patient has not previously received Luxturna treatment in intended eye
Authorization limits: Authorization will be issued for no more than one treatment per lifetime per eye and for no longer than 45 days from approvalone treatment per lifetime per eye; authorization valid up to 45 days
Limit: one treatment per lifetime per eye; authorization validity up to 45 days from approval

This policy is a UnitedHealthcare medical benefit drug policy for Luxturna (voretigene neparvovec-rzyl) that describes coverage rationale, documentation requirements, applicable codes, clinical evidence, and administration/benefit considerations for members under UnitedHealthcare standard plans. The coverage stance is covered_with_criteria and applies under the medical benefit.

Policy effective date is November 1, 2024 with the last review/updated entry on July 1, 2025 (template update).

Prior Authorization, Documentation & Billing

Prior Authorization

Prior Authorization Required

Authorization must be obtained prior to treatment. Authorization is limited to no more than one treatment per lifetime per eye and is valid for up to 45 days from approval.

J3398

Documentation Required

Required Clinical Documentation for Authorization

Submit medical documentation to support authorization including: medication name and regimen; documentation that the member has not previously been treated with RPE65 gene therapy in the intended eye; OCT demonstrating sufficient viable retinal cells (>100 µm thickness in an area of the posterior pole); genetic testing results confirming biallelic RPE65 mutations; current age of the patient; and documentation that the treatment will be administered by an ophthalmologist or retinal surgeon experienced in sub-retinal injections.

Billing Rule

HCPCS Reporting

Report product and administration using the applicable HCPCS code for voretigene neparvovec-rzyl.

J3398 — report product/administration using this code

Documentation Required

Benefit Verification

Verify the member-specific benefit plan and any applicable federal or state mandates prior to authorization. If there is a conflict between this policy and the member’s plan document or mandates, the member-specific benefit plan governs.

Supporting Clinical Evidence

Phase 1Safe and stable improvement in retinal and visual function in 12 participants after unilateral subretinal injection; durable ≥3 years

Phase 329 subjects randomized (20 intervention, 9 control); 72% of treated subjects achieved maximum MLMT improvement at 1 year; benefits persisted ≥2 years

Phase 1 trials reported safe and stable improvements in retinal and visual function for the 12 participants who received unilateral subretinal AAV2-hRPE65v2 injections, with durability of benefit observed for at least 3 years.

The Phase 3 randomized trial enrolled 29 subjects (20 intervention, 9 control); overall, 72% (21/29) of treated subjects achieved the maximum possible MLMT improvement at one year, demonstrating significant improvement in functional vision at lower light levels. The benefits observed in the original intervention group persisted through at least 2 years of follow-up.

References and clinical evidence in this policy were updated on 11/01/2024 per the policy revision history.

Background & Definitions

Luxturna is an adeno-associated virus (AAV)-based gene therapy (voretigene neparvovec-rzyl) indicated for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells.

The therapy is a one-time administration intended to augment RPE65 function in post-mitotic retinal cells; benefit is expected to persist only while retinal cells remain viable.

Indication and administration notes specify that patients must have viable retinal cells as determined by the treating physician (e.g., OCT) and that treatment is reported using HCPCS code J3398.

Term	Definition
Viable retinal cells	Determined by OCT documenting an area of retina within the posterior pole with > 100 µm thickness, or by ophthalmoscopy per treating physician assessment
Biallelic RPE65 mutation-associated retinal dystrophy	Inherited retinal dystrophy caused by pathogenic mutations in both alleles of the RPE65 gene (e.g., Leber's congenital amaurosis, retinitis pigmentosa, early onset severe retinal dystrophy)

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna (voretigene neparvovec-rzyl)

Policy CodePolicy 202400063J

Change TypeTemplate update; Clinical evidence/reference revision

Effective DateNov 1, 2024

Next Review Date

On This Page

H35.50	Unspecified hereditary retinal dystrophy
H35.52	Pigmentary retinal dystrophy
H35.54	Dystrophies primarily involving the retinal pigment epithelium

OpenPayer

Luxturna (voretigene neparvovec-rzyl)

Coverage Summary & Medical Necessity

Prior Authorization, Documentation & Billing

Applicable Codes

Supporting Clinical Evidence

Background & Definitions

Clinical & Authorization Thresholds

Policy Revision History