Pharmacy Prior Authorization Criteria

Luxturna (voretigene neparvovec)

For diagnosis of RPE65 Mutation-Associated Retinal Dystrophy
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).

Prior Authorization

Length of Approval: 1 time for each eye [D]

Diagnosis of confirmed biallelic RPE65 mutation-associated retinal dystrophy (e.g., Leber's congenital amaurosis [LCA], retinitis pigmentosa [RP], early onset severe retinal dystrophy [EOSRD], etc.) [1-6]

AND

AND

Visual acuity worse than 20/60 in both eyes
Visual field less than 20 degrees in any meridian as measured by III4e isopter or equivalent in both eyes

AND

Patient has sufficient viable retinal cells as determined by optical coherence tomography (OCT) demonstrating an area of retina within the posterior pole of greater than 100 micron thickness [1, 6, C]

AND

Prescribed by or in consultation with one of the following physicians associated with an ocular gene therapy treatment Center of Excellence: [B]

AND

Administered by a retinal specialist/surgeon experienced in performing intraocular surgery [2-6, B]

AND

Patient has not previously received RPE65 gene therapy in the intended eye [2-5, D, E]

2024-05-17, 2023-06-12, 2022-06-16, 2021-08-02, 2021-06-15, 2020-05-14

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;390:849-60.
Food and Drug Administration (FDA) Advisory Committee. Cellular, Tissue and Gene Therapies Advisory Committee Meeting Announcement. Website. October 12, 2017. https://www.fda.gov/advisorycommittees/calendar/ucm574394.htm. June 1, 2022.
FDA. Voretigene briefing information. Website. October 12, 2017. https://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/bloodvaccinesandotherbiologics/cellulartissueandgenetherapiesadvisorycommittee/ucm579290.pdf. Accessed June 1, 2022.
FDA. Voretigene briefing information. [errata]. Website. October 12, 2017. https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM579307.pdf. Accessed June 1, 2022.
Spark Therapeutics. Voretigene briefing information. Website. October 12, 2017. https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM579300.pdf. Accessed June 1, 2022.
Luxturna Prescribing Information. Spark Therapeutics, Inc. Philadelphia, PA. April 2024.