CurrentCignaPolicy IP0160

Ophthalmology - Gene Therapy - Luxturna

Defines Cigna's coverage policy, criteria, dosing, administration, and coding for Luxturna (voretigene neparvovec-rzyl) for treatment of biallelic RPE65 variant-associated retinal dystrophy, including prior authorization, required documentation, administration by a retinal specialist, and dosing interval rules.

Policy Summary

PayerCigna

PolicyOphthalmology - Gene Therapy - Luxturna

Policy CodePolicy IP0160

Change TypeName update; criteria and approval duration revisions

Effective DateMar 26, 2026

Next Review Date

Key ActionPrior authorization is recommended and providers must submit documentation confirming genetic biallelic RPE65 variants, viable retinal cell determination by the treating physician, retinal specialist administration, and no prior Luxturna treatment of the eyes.

SourceLink

POLICY UPDATE CHANGES

Policy Name updated from 'Voretigene Neparvovec-rzyl' to 'Ophthalmology - Gene Therapy - Luxturna'.

Removed requirement for presence of sufficiently viable retinal cells determined by OCT and/or ophthalmoscopy with specific numeric OCT or disc-area thresholds and visual field measurement criteria.

Approval duration changed from 30 days to 90 days to allow scheduling of implant procedure.

Replaced word 'mutation' with 'variant' throughout the RPE65 diagnostic requirement and clarified that one dose per eye will be approved.

Annual revision dated 3/20/2025 indicated 'No criteria changes'.

1FDA-Approved Indication

2Injections per patient allowed

Coverage Summary & Medical Necessity

FDA-Approved Indication - Medical Necessity Criteria

Approve for one-time treatment course (one injection per eye) if the patient meets ALL of the following (A-F):

A-F

A. Genetic confirmation: Patient has a genetically confirmed diagnosis of biallelic RPE65 variant-associated retinal dystrophy
[documentation required]
B. Age: Patient is ≥ 12 months of age and < 65 years of age>= 12 months and < 65 years
C. Administration by specialist: Luxturna is administered by a retinal specialist
[documentation required]
D. Viable retinal cells: Patient must have viable retinal cells as determined by the treating physician
[documentation required]; clinical determination by treating physician
E. No prior Luxturna treatment: Patient is not receiving re-treatment of eye(s) previously treated with Luxturna
[documentation required]
F. Approval outcome: If criteria A through E are met, approve one dose of Luxturna (1.5 x 10^11 vector genomes) per eye (two doses per patient) administered by subretinal injection. Note: doses for first and second eye must be separated by at least 6 days.Dose per eye: 1.5 x 10^11 vg; Inter-eye interval: >= 6 days
Approvals are provided for one injection per eye; 90-day approval duration to allow scheduling

Dosing Criteria

Approve the following dosing regimen (both A and B required):

Dosing

A: One 1.5 x 10^11 vector genomes (vg) injection administered by subretinal injection into each eye1.5 x 10^11 vg per eye
administered once per eye by subretinal injection
B: The doses for the first eye and the second eye are separated by at least 6 days>= 6 days between injections of first and second eye
injections to be on separate days within a close interval, but no fewer than 6 days apart

Not Medically Necessary

Luxturna is not medically necessary for uses other than the FDA-approved indication including:

Exclusions

Re-treatment exclusion: Re-treatment of previously treated eye(s). Luxturna is for one time use in each eye. Repeat dosing in previously treated eye(s) is not approvable.
Claims for non-FDA-approved uses including retreatment will be denied

Applicable Codes

Covered HCPCS for drugHCPCSCovered

J3398

Injection, voretigene neparvovec-rzyl, 1 billion vector genomes

Prior Authorization, Documentation & Billing Rules

Prior Authorization

Prior authorization recommended

Prior Authorization is recommended for prescription benefit coverage of Luxturna; approvals are recommended only for patients who meet the policy's stated clinical criteria and dosing requirements.

J3398

Documentation Required

Required documentation for eligibility

Providers must supply documentation confirming a genetically confirmed diagnosis of biallelic RPE65 variants, that the treating physician has determined the patient has viable retinal cells, that Luxturna will be administered by a retinal specialist, and that the eye(s) to be treated have not previously received Luxturna. Acceptable documentation may include chart notes, laboratory tests, claims records, prescription receipts, and/or other information. All documentation must include patient-specific identifying information.

Background & Definitions

Luxturna is an AAV2-based (adeno-associated virus serotype 2) gene therapy that delivers a functional RPE65 gene to retinal cells for the treatment of confirmed biallelic RPE65 variant-associated retinal dystrophy. It is designed to restore biologically active RPE65 in patients with reduced or absent levels of the enzyme.

The indication requires that patients have viable retinal cells as determined by the treating physician. Treatment is not recommended for patients younger than 12 months because retinal cells are still undergoing proliferation and the therapy could be diluted or lost. The safety and effectiveness have not been established in patients ≥ 65 years as clinical studies did not include patients in that age group.

Viable retinal cells: Clinical determination by the treating physician; prior numeric OCT/ophthalmoscopy thresholds were removed and replaced by physician assessment.

One-time treatment course: A total of two injections, one injection per eye, administered on separate days at least 6 days apart.

Evidence & References

Evidence source: Prescribing information — Luxturna subretinal injection (Spark Therapeutics; May 2022). The policy’s clinical and dosing requirements are aligned with the FDA prescribing information.

Key Thresholds & Limits

Key Operational Thresholds

Age>= 12 months and < 65 years

Inter-eye dosing interval>= 6 days between injections of first and second eye

Dose per eye1.5 x 10^11 vector genomes (vg)

Policy Revision History

07/15/2024name_update

Policy Name updated from 'Voretigene Neparvovec-rzyl' to 'Ophthalmology - Gene Therapy - Luxturna' (material: no).

07/15/2024criteria_removal

Removed numeric OCT/ophthalmoscopy and specific visual field thresholds for determining viable retinal cells; replaced with broader requirement that viable retinal cells be determined by the treating physician (material: yes).

03/26/2026approval_duration_changeLatest