71394-0415-XX LUXTURNA VIAL (SPARK THERAPEUTICS)
71394-0065-XX LUXTURNA VIAL (SPARK THERAPEUTICS)
Voretigene neparvovec-rzyl is a live, non-replicating adeno-associated virus serotype 2 which has been genetically modified to express the human RPE65 gene using recombinant DNA techniques. Prepared in a suspension, it is a vector-based gene therapy for subretinal injection containing 5 x 1012 vector genomes (vg) per mL.
This normal copy of the RPE65 gene encodes the human retinal pigment epithelial 65 kDa protein (RPE65) and delivers it directly to the cells of the retina in those individuals with mutations in the RPE65 gene. REP65 protein is normally produced in the retinal pigment epithelial (RPE) cells and is necessary to the visual or retinoid cycle, which in turn is critical to phototransduction, the biological conversion of a photon of light into an electrical signal in the retina. Mutations in the RPE65 gene lead to reduced or absent levels of RPE65 which blocks the visual cycle resulting in visual impairment.
Voretigene neparvovec-rzyl for the treatment of biallelic RPE65 mutation-associated retinal dystrophy is considered medically necessary if the medical appropriateness criteria are met. (See Medical Appropriateness below.)
Voretigene neparvovec-rzyl for the treatment of other conditions/diseases is considered investigational.
Voretigene neparvovec-rzyl is considered medically appropriate if ALL of the following criteria are met:
Individual is 4 years of age or older
Definitive diagnosis of functional mutation-associated retinal dystrophy confirmed by genetic testing for biallelic mutation of the REP65 gene (test may specifically reference AMAUROSIS CONGENITA OF LEBER II; Amaurosis congenita of Leber, type 2; Leber congenital amaurosis 2 [LCA2] or retinitis pigmentosa 20)
Individual has viable retinal cells as determined by non-invasive means, such as optical coherence tomography (OCT) and/or ophthalmoscopy indicating ANY ONE 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 a III4e isopter or equivalent
Individual has not had intraocular surgery within six months
Individual must have an adequate washout period from retinoid therapies prior to receipt of voretigene
Voretigene neparvovec-rzyl is considered NOT medically appropriate for renewal
DOSAGE & ADMINISTRATION
Biallelic RPE65 mutation-associated retinal dystrophy
For subretinal injection only.
Preparing for Administration:
|Notes: Store LUXTURNA and Diluent frozen at ≤ -65 °C. Thaw prior to infusion. LUXTURNA is an adeno-associated virus vector-based gene therapy. Follow universal biohazard precautions for handling.|
LENGTH OF AUTHORIZATION
Coverage will be provided for each eye 1.5 x 1011 vector genomes (vg), administered by subretinal injection in a total volume of 0.3 mL and may not be renewed.
Refer to DOSAGE LIMITS below
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 recognized standard reference compendia or in the published peer-reviewed medical literature.
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, the express terms of the health plan will govern.
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).
MICROMEDEX Healthcare Series. Drugdex Evaluations. (2018, January). Laronidase. Retrieved March 8, 2018 from MICROMEDEX Healthcare Series.
Russell, S., Bennett, J., Wellman, J.A., 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.
U. S. Food and Drug Administration. (2017, December). Center for Drug Evaluation and Research. Luxturna® (voretigene neparvovec-rzyl) intraocular suspension for subretinal injection. Retrieved February 25, 2019 from https://www.fda.gov/downloads/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/UCM589541.pdf.
U. S. National Library of Medicine. (2017, October). National Center for Biotechnology Information. Genetic Testing Registry. RPE65: RPE65, retinoid isomerohydrolase. Retrieved February 25, 2019 from https://www.ncbi.nlm.nih.gov/gtr/genes/6121/.
ORIGINAL EFFECTIVE DATE: 1/26/2018
MOST RECENT REVIEW DATE: 4/9/2019
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.
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Maximum billable units per dose and over time by indication as a Medical Benefit; 1 billable unit = 1 injection