We develop Medical Policies to provide guidance to Members and Providers. This Medical Policy relates only to the services or supplies described in it. The existence of a Medical Policy is not an authorization, certification, explanation of benefits or a contract for the service (or supply) that is referenced in the Medical Policy. For a determination of the benefits that a Member is entitled to receive under his or her health plan, the Member's health plan must be reviewed. If there is a conflict between the Medical Policy and a health plan or government program (e.g., TennCare), the express terms of the health plan or government program will govern.

POLICY

INDICATIONS

The indications below including FDA-approved indications and compendial uses are considered a covered benefit provided that all the approval criteria are met and the member has no exclusions to the prescribed therapy.

FDA-Approved Indications

Luxturna is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Patients must have viable retinal cells as determined by the treating physician(s).

All other indications are considered experimental/investigational and not medically necessary.

DOCUMENTATION

Submission of the following information is necessary to initiate the prior authorization review:

Testing or analysis confirming a genetic diagnosis of pathogenic/likely pathogenic biallelic RPE65 gene mutations.

PRESCRIBER SPECIALTIES

This medication must be prescribed by or in consultation with an ophthalmologist.

COVERAGE CRITERIA

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Authorization of 90 days for a one-time administration per eye may be granted for treatment of biallelic RPE65 mutation-associated retinal dystrophy when all of the following criteria are met:

The member has biallelic pathogenic and/or likely pathogenic RPE65 mutations via genetic testing (single gene test or multi gene panel test if medically necessary).
The RPE65 gene mutations classifications are based on the current American College of Medical Genetics and Genomics (ACMG) standards and guidelines for the interpretation of sequence variants.
Pathogenic and/or likely pathogenic classification of the RPE65 mutations has been affirmed within the last 12 months.
The member is at least 12 months of age but less than 65 years of age.
The member has viable retinal cells in each eye to be treated as determined by optical coherence tomography (OCT) and/or ophthalmoscopy; and must have any of the following:

An area of retina within the posterior pole of greater than 100 μm thickness shown on OCT
Greater than or equal to 3 disc areas of retina without atrophy or pigmentary degeneration within the posterior pole
Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent

The member has not received a previous treatment course of Luxturna.

APPLICABLE TENNESSEE STATE MANDATE REQUIREMENTS

BlueCross BlueShield of Tennessee’s Medical Policy complies with Tennessee Code Annotated Section 56-7-2352 regarding coverage of off-label indications of Food and Drug Administration (FDA) approved drugs when the off-label use is recognized in one of the statutorily

ADDITIONAL INFORMATION

For appropriate chemotherapy regimens, dosage information, contraindications, precautions, warnings, and monitoring information, please refer to one of the standard reference compendia (e.g., the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) published by the National Comprehensive Cancer Network®, Drugdex Evaluations of Micromedex Solutions at Truven Health, or The American Hospital Formulary Service Drug Information).

REFERENCE

Luxturna [package insert]. Philadelphia, PA: Spark Therapeutics, Inc.; May 2022.
Russel S, Bennet J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomized, controlled, open-label phase 3 trial. Lancet 2017; 390:849-860.
Richards S, Aziz N, Bale S, et al; ACMG Laboratory Quality Assurance Committee. Standards and guidelines for the interpretation of sequence variants: A joint consensus recommendation of the American College of Medical Genetics and Genomics and the Association for Molecular Pathology. Genet Med. 2015;17(5):405-24.

ORIGINAL EFFECTIVE DATE: 1/26/2018

MOST RECENT REVIEW DATE: 9/30/2025

ID_CHS

Policies included in the Medical Policy Manual are not intended to certify coverage availability. They are medical determinations about a particular technology, service, drug, etc. While a policy or technology may be medically necessary, it could be excluded in a member's benefit plan. Please check with the appropriate claims department to determine if the service in question is a covered service under a particular benefit plan. Use of the Medical Policy Manual is not intended to replace independent medical judgment for treatment of individuals. The content on this Web site is not intended to be a substitute for professional medical advice in any way. Always seek the advice of your physician or other qualified health care provider if you have questions regarding a medical condition or treatment.

This document has been classified as public information.