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PM173 Voretigene Neparvovec (Luxterna) Original Department: Pharmacy Management 10/12/2020 Approval: Policy #: PM173 Last Approval: 10/12/2020 Title: Voretigene neparvovec (Luxterna) Approved By: UM Pharmacy Subcommittee Line(s) of Business WAH-IMC (HCA) BHSO Medicare Advantage (CMS) Medicare SNP (CMS) Cascade Select Documentation required to determine medical necessity for Voretigene neparvovec (Luxterna): History and/or physical examination notes and relevant specialty consultation notes that address the problem and need for the service: Diagnosis-Age-Medication list (current and past)- Labs/Diagnostics. BACKGROUND Retinitis pigmentosa (RP) are a complex group of inherited retinal dystrophies characterized by progressive dysfunction and degeneration of the retina affecting photoreceptor and pigment epithelial function. One of the causes of these retinal dystrophies is due to a mutation in the gene encoding retinal pigment epithelium-specific protein 65 kDa (RPE65). Symptoms of RP often begin in childhood and include decreased vision at night or in low light and loss of side vision. Current investigational therapies include gene therapy, cell therapy and retinal prostheses. Although there is no cure for these retinal dystrophies, the use of gene therapy is intended to negate the effects of mutations in the RPE65 gene. Voretigene neparvovec (Luxturna) is an adeno-associated virus vector-based gene therapy used for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Voretigene naparvovec delivers a normal copy of the RPE65 protein to retinal cells augmenting reduced or absent levels of biologically active RPE65 leading to normal production of the protein that converts light to an electrical signal in the retina to restore patients’ vision loss. DEFINITIONS none INDICATIONS/CRITERIA CHPW AH-IMC Excluded benefit. Authorization and billing required directly through WA members Apple Health Fee For Service only. Call 800-562-3022. CHNW Cascade Select Continue to criteria for approval below. members Medicare Members Step-utilization of Part D drugs not required. Continue to criteria for approval below. Coverage of Voretigene neparvovec (Luxterna) is recommended in those who meet the following criteria: FDA-Approved Indications PM173_CCC Voretigene neparvovec (Luxterna) 1 of 3 DATA CONTAINED IN THIS DOCUMENT IS CONSIDERED CONFIDENTIAL AND PROPRIETARY INFORMATION AND ITS DUPLICATION USE OR DISCLOSURE IS PROHIBITED WITHOUT PRIOR APPROVAL OF COMMUNITY HEALTH PLAN OF WASHINGTON. Retinal dystrophy, In patients with viable retinal cells and confirmed biallelic RPE65 mutation 1. Patient is 1 to 64 years of age with a confirmed diagnosis of biallelic RPE65 mutation associated-retinal dystrophy via genetic testing; AND 2. Patient has visual acuity worse than 20/60 in both eyes or visual field less than 20 degrees in any meridian; AND 3. Patient has viable retinal cells and verification must be documented by at least ONE of the following: a. An area of the retina within the posterior pole of >100 um thickness shown on optical coherence tomography; OR b. Three or more disc areas of retina without atrophy or pigmentary degeneration within the posterior pole; OR c. Remaining visual field within 30 degrees of fixation as measured by a III4e isopter or equivalent; AND 4. Patient has not previously received RPE65 gene therapy in the intended eye(s); AND 5. Patient has not had intraocular surgery within six months in the intended eye(s); AND 6. Prescribed and administered by an ophthalmologist or retinal surgeon who specializes in performing intraocular surgery If ALL criteria are met, the request will be approved for one dose per eye, per lifetime. If both eyes are to be treated, injections must be administered at least six days apart. CONDITIONS NOT RECOMMENDED FOR APPROVAL Voretigene neparvovec (Luxterna) has not been shown to be effective, or there are limited or preliminary data or potential safety concerns that are not supportive of general approval for the following conditions. Coverage is not recommended for circumstances not listed in the Recommended Authorization Criteria. Criteria will be updated as new published data are available. SPECIAL CONSIDERATIONS none LIMITATIONS/EXCLUSIONS Please refer to a product line’s certificate of coverage for benefit limitations and exclusions for these services: PRODUCT LINE LINK TO CERTIFICATE OF COVERAGE CHPW Medicare Advantage https://medicare.chpw.org/chpw-washington- state-medicare-advantage-plans/all-medicare- plans-2020/ PM173_CCC Voretigene neparvovec (Luxterna) 2 of 3 DATA CONTAINED IN THIS DOCUMENT IS CONSIDERED CONFIDENTIAL AND PROPRIETARY INFORMATION AND ITS DUPLICATION USE OR DISCLOSURE IS PROHIBITED WITHOUT PRIOR APPROVAL OF COMMUNITY HEALTH PLAN OF WASHINGTON. Washington Apple Health (Medicaid) Integrated https://www.chpw.org/for-members/benefits- Managed Care and-coverage-imc/ CHNW Cascade Select Citations & References CFR 42 CFR § 438.210 WAC WAC 284-43-2050 RCW Contract Citation WAH - IMC AH section 17.3.2.1 General Description of Contracted Services - Pharmacy Benefit and Services - Apple Health Preferred Drug List and Plan Formularies BHSO MA Cascade Select WAC 284-43-5642 - Essential health benefit categories Other Requirements NCQA Elements References 1. Garg S. Retinitis pigmentosa: Treatment. In: Post T, ed. UpToDate. Waltham, MA.: UpToDate; 2018.www.uptodate.com. Accessed February 5, 2020. 2. Luxturna [package insert]. Philadelphia, PA; Spark Therapeutics, Inc., December 2017.Accessed February 2020. 3. 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 4. Voretigene neparvovec-rzyl. Micromedex Solutions. Truven Health Analytics, Inc. Ann Arbor, MI.Available at: http://www.micromedexsolutions.com. Accessed February 4, 2020 5. Washington Healthcare Authority Ophthalmic Agents: Gene Therapy-Voretigene neparvovec- rzyl (Luxterna) Medical Policy no. 86.37.00-1 Effective date July 1, 2020. Revision History Revision Date Revision Description Revision Made By 09/14/2020 New policy Jennifer Farley, PharmD 10/12/2020 Approval UM Committee PM173_CCC Voretigene neparvovec (Luxterna) 3 of 3 DATA CONTAINED IN THIS DOCUMENT IS CONSIDERED CONFIDENTIAL AND PROPRIETARY INFORMATION AND ITS DUPLICATION USE OR DISCLOSURE IS PROHIBITED WITHOUT PRIOR APPROVAL OF COMMUNITY HEALTH PLAN OF WASHINGTON. .
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