ModifiedUnitedHealthcarePolicy CSOH2026D0064.C

Luxturna® (Voretigene Neparvovec-Rzyl) (for Ohio Only)

Ohio-only UnitedHealthcare Community Plan medical benefit drug policy governing coverage of Luxturna (voretigene neparvovec-rzyl) for treatment of inherited retinal dystrophies caused by biallelic RPE65 mutations, including clinical criteria, applicable codes, and authorization limits.

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna® (Voretigene Neparvovec-Rzyl) (for Ohio Only)

Policy CodePolicy CSOH2026D0064.C

Change TypeRevised coverage criteria

Effective DateFeb 1, 2026

Next Review Date

Key ActionObtain prior authorization (one treatment per lifetime per eye; valid up to 45 days) and include genetic testing and OCT documentation showing > 100 µm posterior pole retinal thickness; do not request inpatient admission solely to administer Luxturna.

SourceLink

POLICY UPDATE CHANGES

Revised coverage criteria; added criterion requiring the provider does not request a planned inpatient admission for the sole purpose of administering Luxturna.

1Treatment per lifetime per eye

45 daysAuthorization validity (from approval)

>=100 µmOCT posterior pole thickness threshold

Coverage Summary & Rationale

Coverage Rationale (Medical Necessity Criteria)

Covered when ALL of the following are met:

Patient is greater than 12 months of age
Diagnosis of a confirmed biallelic RPE65 mutation-associated retinal dystrophy (e.g., Leber's congenital amaurosis, retinitis pigmentosa, early onset severe retinal dystrophy)
Genetic testing documenting biallelic mutations of the RPE65 gene
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
Prescribed and administered by an ophthalmologist or retinal surgeon with experience providing sub-retinal injections
Patient has not previously received Luxturna treatment in intended eye
Provider does not request a planned inpatient admission for the sole purpose of administering Luxturna

Authorization, Documentation & Billing Instructions

Prior Authorization

Authorization required and limits

Obtain prior authorization; authorization issued for no more than one treatment per lifetime per eye and is valid for up to 45 days from approval.

Affected code: J3398
Limit: 1 treatment per lifetime per eye
Authorization valid for up to 45 days from approval

Documentation Required

Genetic testing documentation

Provide documentation of genetic testing confirming biallelic RPE65 mutations as part of the authorization request.

Submit genetic testing showing pathogenic or likely pathogenic mutations in both alleles of RPE65 (biallelic RPE65 mutations)

Documentation Required

OCT documentation of viable retina

Provide OCT results documenting an area of retina within the posterior pole > 100 µm thickness to demonstrate sufficient viable retinal cells.

OCT threshold: area of retina within the posterior pole > 100 µm thickness

Documentation Required

Provider qualifications

Ensure treating clinician is an ophthalmologist or retinal surgeon experienced with sub-retinal injections; include provider specialty/credentials in the request.

Include provider specialty and credentials (ophthalmologist or retinal surgeon experienced with sub-retinal injections)

Billing Rule

Administration setting restriction

Do not request a planned inpatient admission for the sole purpose of administering Luxturna; such requests would violate coverage criterion.

Planned inpatient admission solely for administration is not allowed per coverage criterion

Summary of Key Clinical Evidence

Durable ≥3 yrsPhase 1 safety/efficacy: improved light sensitivity, navigational abilities, or visual acuity with durable effect through ≥3 years

Luxturna is an adeno-associated virus (AAV) vector-based gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy (e.g., Leber's congenital amaurosis and some forms of autosomal recessive retinitis pigmentosa). Durable benefit from a one-time administration is expected only while target retinal cells remain viable; sufficient viable retinal cells are operationalized by OCT documenting an area of retina within the posterior pole with > 100 µm thickness.

Background and Definitions

Leber's congenital amaurosis (LCA) and autosomal recessive retinitis pigmentosa (RP) are inherited, early-onset, severe retinal dystrophies that cause substantial sight impairment. Biallelic RPE65 mutations impair the visual cycle by reducing production of 11-cis retinal, causing photoreceptor dysfunction and progressive degeneration. Augmentation of RPE65 via gene therapy can improve retinal and visual function in animal models and clinical trials; because target retinal cells are post-mitotic, one-time administration is expected to provide durable benefit only when sufficient viable retinal cells exist.

Term	Definition
Viable retinal cells
Presence of retinal tissue sufficient for gene therapy benefit, operationalized as OCT documenting an area of retina within the posterior pole with > 100 µm thickness.
Biallelic RPE65 mutation
Pathogenic or likely pathogenic mutations in both alleles of the RPE65 gene confirmed by genetic testing.

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna® (Voretigene Neparvovec-Rzyl) (for Ohio Only)

Policy CodePolicy CSOH2026D0064.C

Change TypeRevised coverage criteria

Effective DateFeb 1, 2026

Next Review Date

SourceLink

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) (for Ohio Only)

Coverage Summary & Rationale

Authorization, Documentation & Billing Instructions

Applicable Codes

Summary of Key Clinical Evidence

Background and Definitions

Clinical Thresholds and Authorization Limits

Policy Revisions