CurrentUnitedHealthcarePolicy IEXD0063.07

Luxturna (voretigene neparvovec-rzyl)

Defines UnitedHealthcare medical benefit coverage criteria and coding guidance for Luxturna (voretigene neparvovec-rzyl) gene therapy for biallelic RPE65 mutation-associated inherited retinal dystrophy, including patient eligibility, provider requirements, lifetime limits, and applicable HCPCS/ICD-10 codes.

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna (voretigene neparvovec-rzyl)

Policy CodePolicy IEXD0063.07

Change TypeSupporting information update (Clinical Evidence and References)

Effective DateNovember 1, 2024

Next Review Date

Key ActionObtain prior authorization and document genetic testing and OCT evidence of viable retina; approvals are valid for no more than one treatment per lifetime per eye and for up to 45 days from approval.

SourceLink

POLICY UPDATE CHANGES

Archived previous policy version IEXD0063.06

No material clinical or coverage changes — archived prior version and updated supporting information only.

1Treatment per lifetime per eye

>=100 µmOCT retinal thickness threshold

12 monthsMinimum patient age

Coverage Summary

Trek Health ingests and normalizes Transparency in Coverage data and payer policy updates to give provider organizations a clear view of how commercial reimbursement behaves across markets, payers, and services. Our platform transforms raw payer disclosures into structured intelligence that supports contract evaluation, payer negotiations, and service line strategy. By combining market benchmarks with ongoing policy visibility, Trek helps teams identify variability, risk, and opportunity in commercial reimbursement. The result is faster insight, stronger negotiating positions, and more informed financial decisions.

Defines UnitedHealthcare medical benefit coverage criteria and coding guidance for Luxturna (voretigene neparvovec-rzyl) gene therapy for confirmed biallelic RPE65 mutation-associated inherited retinal dystrophy, including patient eligibility (minimum age 12 months), provider requirements, lifetime limits, and applicable HCPCS/ICD-10 codes. (Policy IEXD0063.07, Effective 2024-11-01.)

Coverage stance: Covered with criteria when all specified medical necessity criteria are met. Authorization is limited to 1 treatment per lifetime per eye and approvals are valid for up to 45 days from approval. (Last review/effective 2024-11-01.)

Medically Necessary / Initial Coverage Criteria

Luxturna is proven and/or medically necessary when ALL of the following are met:

Age: 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: Genetic testing documenting biallelic mutations of the RPE65 gene
Document genetic testing in the medical record
Retinal Viability: 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
OCT demonstrating area of retina within the posterior pole > 100 µm
Provider Qualifications: Prescribed and administered by an ophthalmologist or retinal surgeon with experience providing sub-retinal injections
Document provider credentials and experience
Prior Treatment: Patient has not previously received Luxturna treatment gene therapy in intended eye
Limit = 1 treatment per lifetime per eye
Authorization Validity: Authorization will be issued for no more than one treatment per lifetime per eye and for no longer than 45 days from approvalLimit = 1 treatment per lifetime per eye; authorization validity = up to 45 days

Provider Requirements & Authorization

Prior Authorization

Authorization required and validity period

Obtain prior authorization before administering Luxturna. Approvals will be issued for no more than one treatment per lifetime per eye and are valid for no longer than 45 days from approval. Affected code: J3398.

Affected code(s): J3398
Limit: 1 treatment per lifetime per eye
Authorization validity: up to 45 days from approval

Documentation Required

Genetic testing documentation

Document genetic testing in the medical record showing confirmed biallelic RPE65 mutations to support the coverage determination.

Documentation Required

OCT documentation of viable retina

Provide an OCT report that demonstrates sufficient viable retinal cells, specifically an area of retina within the posterior pole with thickness greater than 100 µm.

Documentation Required

Provider qualifications

Treatment must be prescribed and administered by an ophthalmologist or retinal surgeon experienced in performing sub-retinal injections. Document the provider's credentials and experience in the medical record.

Evidence Summary

72% (21/29)Phase 3 trial MLMT improvement: 72% of treated subjects (21 of 29) achieved maximum possible MLMT improvement at one year

>=2 yearsLong-term durability: Benefits observed at one year continued through at least two years post-administration

Luxturna is an adeno-associated virus (AAV) vector–based gene therapy for patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Because the target retinal cells are post-mitotic, a one-time administration can provide durable benefit for as long as the treated retinal cells remain viable; treatment effectiveness therefore depends on having sufficient viable retinal cells at the time of administration. Optical coherence tomography (OCT) showing retinal thickness within the posterior pole of ≥ 100 µm is used to assess retinal viability.

Background & Definitions

Luxturna (voretigene neparvovec-rzyl) is indicated for confirmed biallelic RPE65 mutation-associated retinal dystrophy. It is an AAV vector–based gene therapy that augments RPE65 in target retinal cells; because the target cells are post-mitotic, a single administration is expected to provide benefit while those cells remain viable. Gene therapy does not create new retinal tissue, so sufficient viable retinal cells must be present prior to treatment, commonly assessed by OCT documenting an area of retina within the posterior pole of ≥ 100 µm thickness.

Viable retinal cellsArea of retina within the posterior pole > 100 µm thickness on OCT indicating sufficient target cells for therapy

Policy Summary

PayerUnitedHealthcare

PolicyLuxturna (voretigene neparvovec-rzyl)

Policy CodePolicy IEXD0063.07

Change TypeSupporting information update (Clinical Evidence and References)

Effective DateNovember 1, 2024

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)

Coverage Summary

Provider Requirements & Authorization

Applicable Codes

Evidence Summary

Background & Definitions

Quick Thresholds & Limits

Policy Revision History