Last Review Date: 01/06/2025

Date of Origin: 01/03/2018

Dates Reviewed: 01/2018, 03/2018, 07/2018, 01/2019, 01/2020, 01/2021, 01/2022, 01/2023, 01/2024, 01/2025

1. Length of Authorization

Coverage will be provided for one dose per eye and may not be renewed.

2. Dosing Limits

    Max Units (per dose and over time) [HCPCS Unit]:

• 150 billable units for one dose per eye

3. Initial Approval Criteria ^1,2

Coverage is provided in the following conditions:

• Patient is at least 12 months of age; AND
• Patient must have an adequate washout period, defined as a minimum of 3 months, from retinoid therapies prior to receipt of voretigene neparvovec-rzyl; AND 
• Patient has not had intraocular surgery within six months; AND
• Patient has not previously received subretinal administration of a gene therapy vector, or Luxturna, into the intended eye; AND

Retinal Dystrophy † Ф ^1,2

• Patient has a definitive diagnosis confirming biallelic RPE65 mutation-associated retinal dystrophy; AND
• Patient must have viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy indicating one or more of the following:
  • An area of retina within the posterior pole of >100 µm thickness shown on OCT
  •  ≥ 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 an III4e isopter or equivalent

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ¹

• Duration of authorization has not been exceeded (refer to Section I).

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS code:

• J3398 - Injection, voretigene neparvovec-rzyl, 1 billion vector genomes; 1 billable unit = 10⁹ vector genomes

NDC:

• Luxturna carton (one single-dose vial of Luxturna and two vials of diluent): 71394-0415-xx

7. References

1. Luxturna [package insert]. Philadelphia, PA; Spark Therapeutics, Inc., May 2022. Accessed December 2024.
2. Russell S, Bennett J, Wellman JA, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Erratum in: Lancet. 2017 Aug 26;390(10097):848.
3. Palmetto GBA. Local Coverage Article: Billing and Coding: Voretigene Neparvovec-rzyl (Luxturna®) (A56419). Centers for Medicare & Medicaid Services, Inc. Updated on 04/10/2023 with effective date of 04/20/2023. Accessed December 2024.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.