WHA

Luxturna (voretigene neparvovec)

Indications for Prior Authorization

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).

Criteria

Luxturna

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
  • Patient is 12 months of age or older [6, A]
    • AND
  • Used for the treatment of vision loss defined by one of the following: [1]
    • 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]
    • Ophthalmologist
    • Retinal specialist/surgeon
    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]

  • Guideline ID
    GL-146763

  • Formulary
    premium

  • Effective date
    Aug 1, 2024

  • P&T approval date
    Nov 16, 2017

P & T Revisions

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

  1. 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.
  2. 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.
  3. FDA. Voretigene briefing information. Website. October 12, 2017. https://www.fda.gov/downloads/advisorycommittees/committeesmeetingmaterials/bloodvaccinesandotherbiologics/cellulartissueandgenetherapiesadvisorycommittee/ucm579290.pdf. Accessed June 1, 2022.
  4. FDA. Voretigene briefing information. [errata]. Website. October 12, 2017. https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM579307.pdf. Accessed June 1, 2022.
  5. Spark Therapeutics. Voretigene briefing information. Website. October 12, 2017. https://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/BloodVaccinesandOtherBiologics/CellularTissueandGeneTherapiesAdvisoryCommittee/UCM579300.pdf. Accessed June 1, 2022.
  6. Luxturna Prescribing Information. Spark Therapeutics, Inc. Philadelphia, PA. April 2024.
  1. Per Luxturna Prescribing Information (PI), treatment with Luxturna is not recommended for patients younger than 12 months of age, because the retinal cells are still undergoing cell proliferation, and Luxturna would potentially be diluted or lost during cell proliferation. [6] This is consistent with the OptumRx age policy, as there is a specific efficacy concern when using the medication in patients of a certain age.
  2. Voretigene neparvovec will be administered solely through a small number of Centers of Excellence associated with an active ophthalmology practice that treats patients with inherited retinal diseases including RPE65 mutation-associated retinal dystrophy. Voretigene neparvovec will only be prepared and administered by surgeons who have completed the in-person training programs. [5, 6]
  3. According to the FDA Advisory Committee discussions and PI, voretigene neparvovec should only be administered to patients with sufficient viable retinal cells. Treatment failure may occur if patients do not have enough viable retinal cells for exposure to the vector. The injection is also only targeted at 1/5 of the retina, and if not delivered to the appropriate location, may not be able to exert action or may be degraded by other precipitants within the eye (i.e., enzymes). [2-6]
  4. The recommended voretigene neparvovec administration regimen consists of sequential, bilateral subretinal injections of 1.5E11 (or 150 billion) vg delivered in a total subretinal volume of 0.3 mL per eye per lifetime (total of 2 injections per lifetime). The individual administration procedures to each eye are to be performed on separate days no more than 6 to 18 days apart. This interval between administrations was used in the pivotal trial to afford an opportunity for identification of early-emergent potential surgical complications prior to a patient undergoing the second procedure, and to reduce the risk of a deleterious immune response by carrying out the two administration procedures in a near-simultaneous fashion, rather than a more widely spaced interval that could facilitate a prime boost response. [5, 6]
  5. Since there are other RPE65 gene therapies in the pipeline that will also be administered once per lifetime, voretigene neparvovec was not specified in this criterion to concede the possibility that patients may have already received RPE65 gene therapy through participation in clinical trials.
  • 2024-05-17: 2024 Annual Review. Background changes.
  • 2023-06-12: Annual review - updated references.
  • 2022-06-16: Annual review - no changes.
  • 2021-08-02: Annual review - no changes.
  • 2021-06-15: Annual review - no changes.
  • 2020-05-14: Updated References