IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Apretude (cabotegravir extended-release )BENEFIT TYPE Medical STATUS Prior Authorization Required Apretude is an HIV-1 integrase strand transfer inhibitor (INSTI) initially approved by the FDA in 2021. It is indicated for the pre-exposure prophylaxis (PrEP) of HIV infection in at-risk adults and adolescents weighing at least 35 kg. Apretude is the first injectable medication for the prevention of HIV, taken every two months. The u se of oral cabotegravir (Vocabria) as a lead-in prior to initiating therapy with Apretude is optional.Individuals must have a negative HIV test prior to initiating Apretude to prevent drug resistance. Apretude (cabotegravir extended-release ) will be considered for coverage when the following criteria are met:Pre-exposure Prophylaxis of HIV infectionFor initial authorization: 1. Member must be at least 12 years of age and weigh at least 35 kg; AND 2. Member is not a candidate for oral PrEP (ex.difficulty with adherence, signficiant renal disease, trouble swallowing pills etc.); AND 3. Member has had or will have completed an HIV RNA test before initial and subsequent injections; AND 4. Member is NOT taking any of the following concomitantly with Apretude: a) Rifampin; b) Carbamazepine, oxcarbazepine, phenobarbital or phenytoin; c) Other antiretroviral therapy. 5. Dosage allowed/Quantity limit: Initiate Apretude with a single 600-mg (3-mL) injection given 1 month apart for 2 consecutive months on the last day of an oral lead-in if used or within 3 days and continue with the injections every 2 months thereafter. Quantity Limit: 3 mL per 56 days. If all the above requirements are met , the medication will be approved for 2 months. For reauthorization : 1. Member has had or will have completed an HIV RNA test before injections. If all the above requirements are met , the medication will be approved for an additional 6 months . CareSource considers Apretude (cabotegravir extended-release ) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023DATE ACTION/DESCRIPTION01/12/2022 New policy for Apretude created. 05/11/2023 Simplified HIV RNA testing requirement and removed 7-day window; added quantity limit. 08/16/2023 Rephrased trial of oral PrEP to patient is not a candidate for oral PrEP and added examples. References: 1. Apretude [package insert]. Research Triangle Park, NC; GlaxoSmithKline. February 2023.2. Pre-exposure Prophylaxis for the Prevention of HIV Infection in the United States 2021 Update. Center forDisease Control and Prevention. : Available at : https://www.cdc.gov/hiv/pdf/risk/prep/cdc-hiv-prep-guidelines-2021 . Accessed on January 13, 2022. 3. Landovitz RJ, Li S, Eron Jr JJ, et al. Tail-phase safety, tolerability, and pharmacokinetics of long acting injectable cabotegravir in HIV-uninfected adults: a secondary analysis of the HPTN 077 trial. The Lancet HIV. 2020 4. Landovitz RJ, Donnell D, Clement ME, et al. Cabotegravir for HIV prevention in cisgender men and transgender women. NEngl JMed. 2021;385(7):595-608. 5. Marzinke MA, Grinsztejn B, Fogel JM, et al. Characterization of human immunodeficiency virus (HIV) infection in cisgender men and transgender women who have sex with men receiving injectable cabotegravir for HIV prevention: HPTN 083. Infect Ds. 2021. Effective date: 01/01/2024 Revised date: 08/16/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Turalio (pexidartinib)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Turalio (pexidartinib) is a kinase inhibitor indicated for the treatment of adult patients with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitations and not amenable to improvement with surgery. Initially approved in 2019, Turalio is the first FDA-approved systemic treatment for TGCT. Turalio targets colony stimulating factor 1 receptor (CSF1R) and other tyrosine kinases to inhibit cell proliferation and accumulation. TGCT, also known as pigmented villonodular synovitis or giant cell tumor of the tendon, is a rare non-malignant tumor that affects the synovium and tendon sheath. The tumor causes overgrowth and thickening of the tissues which leads to swelling, pain and reduced range of motion. First-line treatment consists of surgery for patients who are considered amendable to improvement.Turalio (pexidartinib) will be considered for coverage when the following criteria are met:Tenosynovial Giant Cell TumorFor initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with an oncologist or orthopedic surgeon; AND 3. Member has a diagnosis of symptomatic benign TGCT confirmed by MRI or histology; AND 4. Disease is associated with severe morbidity or functional limitations; AND 5. Prescriber attests that the members disease is not amenable to improvement with surgery; AND 6. Chart notes must document that baseline liver tests have been or will be completed prior to starting therapy; AND 7. Member does not have ANY of the following: a) Pregnancy or plan to become pregnant during treatment; b) Active cancer 8. Dosage allowed/Quantity limit: 250 mg orally twice daily with a low-fat meal. Quantity Limit: 120 capsules per 30 days. If all the above requirements are met, the medication will be approved for 6 months. For reauthorization : 1. Chart notes must show improvement or stabilized signs and symptoms of disease (such as decreased pain and stiffness or increased range of motion). If all the above requirements are met, the medication will be approved for an additional 12 months. CareSource considers Turalio (pexidartinib) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023DATE ACTION/DESCRIPTION03/20/2023 New policy for Turalio created. References: 1. Turalio (pexidartinib) [package insert]. Basking Ridge, NJ: Daiichi Sankyo, Inc.; 2022. 2. National Comprehensive Cancer Network. Soft Tissue Sarcoma. (Version 1.2023). https://www.nccn.org/professionals/physician_gls/pdf/sarcoma.pdf. Accessed March 21, 2023. 3. Stacchiotti S, Drr HR, Schaefer IM, et al. Best clinical management of tenosynovial giant cell tumour (TGCT): A consensus paper from the community of experts. Cancer Treat Rev. 2023;112:102491. doi:10.1016/j.ctrv.2022.102491 4. Healey JH, Bernthal NM, van de Sande M. Management of Tenosynovial Giant Cell Tumor: A Neoplastic and Inflammatory Disease. JAm Acad Orthop Surg Glob Res Rev. 2020;4(11):e20.00028. doi:10.5435/JAAOSGlobal-D -20-00028 5. Tap WD, Gelderblom H, Palmerini E, et al. Pexidartinib versus placebo for advanced tenosynovial giant cell tumour (ENLIVEN): a randomised phase 3 trial. Lancet. 2019;394(10197):478-487. doi:10.1016/S0140-6736(19)30764-0 6. Baldini, E. Treatment for tenosynovial giant cell tumor and other benign neoplasms affecting soft tissue and bone. In: Post TW, ed. UpToDate. Waltham, MA: UpToDate; Updated November 17, 2022. Accessed March 21, 2023. Effective date: 10/01/2023 Revised date: 03/20/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Syfovre (pegcetacoplan )BENEFIT TYPE Medical STATUS Prior Authorization Required Syfovre , approved by the FDA in 2023, is a complement (C3) inhibitor indicated for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). It is the first drug approved for this indication. There are 2 types of AMD : dry or wet (neovascular). Syfovre is approved for dry AMD which is more common but progresses more slowly to vision loss than wet AMD . GA can occur in the intermediate and advanced stages of dry AMD and is caused by the breakdown of cells in the macula, resulting in irreversible lesions that can impair vision or lead to blindness . Approval of Syfovre was based on combined analysis of the Phase 3 DERBY and OAKS trials. It was also studied in the Phase 2 FILLY trial. Although it slows the growth rate of GA lesions, Syfovre does not preserve visual function. It may also accelerate the development of new-onset wet AMD.Syfovre (pegcetacoplan) will be considered for coverage when the following criteria are met:Geographic Atrophy (GA)For initial authorization: 1. Member is at least 50 years of age ; AND 2. Medication must be prescribed by or in consultation with an ophthalmologist; AND 3. Member has a documented diagnosis of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) ; AND 4. Diagnosis has been confirmed by fundus autofluorescence (FAF) imaging with both of the following: a) Total GA area must be 2.5 and 17.5 mm2 (1 and 7 disk areas [DA] respectively) and b) If GA is multifocal, at least one focal lesion must be 1.25 mm2 (0.5 DA); AND 5. Documentation of best corrected visual acuity (BCVA) of 24 letters or better using Early Treatment Diabetic Retinopathy Study (ETDRS) charts (approximately 20/320 Snellen equivalent); AND 6. Member does NOT have any of the following: a) GA secondary to any condition other than AMD b) History or current evidence of wet AMD. 7. Dosage allowed/Quantity limit: 15 mg (0.1 mL of 150 mg/mL solution) administered by intravitreal injection to each affected eye once every 25 to 60 days. (QL: 4 vials per 60 days) If all the above requirements are met , the medication will be approved for 12 months. For reauthorization : 1. GA lesion growth rate has slowed or stabilized. If all the above requirements are met , the medication will be approved for an additional 12 months . IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023 CareSource considers Syfovre (pegcetacoplan) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION04/06/2023 New policy for Syfovre created. References: 1. Syfovre [prescribing information]. Apellis Pharmaceuticals, Inc.; 2023. 2. Flaxel CJ, Adelman RA, Bailey ST, et al. Age-Related Macular Degeneration Preferred Practice Pattern [published correction appears in Ophthalmology. 2020 Sep;127(9):1279]. Ophthalmology. 2020;127(1):P1-P65. doi:10.1016/j.ophtha.2019.09.024 3. Liao DS, Grossi FV, El Mehdi D, et al. Complement C3 Inhibitor Pegcetacoplan for Geographic Atrophy Secondary to Age-Related Macular Degeneration: A Randomized Phase 2 Trial. Ophthalmology. 2020;127(2):186-195. doi:10.1016/j.ophtha.2019.07.011 4. Heier J, et al. Efficacy of intravitreal pegcetacoplan in geographic atrophy: results from the DERBY and OAKS trials. Presented at: Retina Society Annual Meeting; September 29 October 2, 2021; Chicago, IL. Accessed April 5 , 2023. https://apellismedicalhub.com/geographic-atrophy-ga/retina-society-2021/efficacy-of-intravitreal-pegcetacoplan-in-geographic-atrophy-results-from-the-derby-and-oaks-trials/5. Goldberg R, et al. Efficacy of intravitreal pegcetacoplan in patients with geographic atrophy (GA): 18-month results from the Phase 3 OAKS and DERBY studies. Presented at: 2022 ARVO Annual Meeting; May 3 7, 2022; Denver, CO. Accessed April 5 , 2023. https://apellismedicalhub.com/science/geographic-atrophy/the-association- for-research-in-vision-and-opthalmology-arvo-2022/efficacy-of-intravitreal-pegcetacoplan-in-patients-with-geographic-atrophy-ga-18-month-results-from-the-phase-3 -oaks-and-derby-studie s/ 6. Heier J, et al. Efficacy of intravitreal pegcetacoplan in geographic atrophy: 24-month results from the phase 3 OAKS and DERBY trials. Presented at: Retina Society 55th Annual Meeting; November 2 5, 2022; Pasadena, CA. Accessed February 7, 2023. https://investors.apellis.com/static-files/78d1b209-7324-4c4c-8b20- bf7778493bae 7. Singh R, et al. Safety of intravitreal pegcetacoplan in geographic atrophy: 24-month results from the OAKS and DERBY phase 3 trials. Presented at: Retina Society 55th Annual Meeting; November 2 5, 2022; Pasadena, CA. Accessed February 7, 2023. https://investors.apellis.com/static-files/faa3ab29-face-436a-b19d-4ec393f6436e 8. Garg S, et al. Impact of baseline imbalances on the efficacy of pegcetacoplan for the treatment of geographic atrophy (GA): a post-hoc analysis of OAKS, DERBY, and FILLY. Presented at: 2022 ARVO Annual Meeting; May 3 7, 2022; Denver, CO. Accessed February 7, 2023. https://apellismedicalhub.com/science/geographic- atrophy/the-association-for-research-in-vision-and-opthalmology-arvo-2022/impact-of-baseline-imbalances-on-the-efficacy-of-pegcetacoplan-for-the-treatment-of-geographic-atrophy-ga-a -post-hoc-analysis-of-oaks-derby-and-filly/ Effective date: 10/01/2023 Revised date: 04/06/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Susvimo (ranibizumab)BENEFIT TYPE Medical STATUS Prior Authorization Required Susvimo , an intravitreal ocular implant, was approved by the FDA in 2021. It is indicated for the treatment of patients with Neovascular (wet) Age-related Macular Degeneration (AMD) who have previously responded to at least two intravitreal injections of a Vascular Endothelial Growth Factor (VEGF) inhibitor medication. VEGF inhibitors suppress endothelial cell proliferation, neovascularization, and vascular permeability . Susvimo was previously referred to as Lucentis Port Delivery System (PDS) since it is essentially a longer lasting version of Lucentis, releasing ranibizumab over a 6-month period rather than needing to be administered monthly . After 6 months, the port can be re-filled. Lucentis is approved for other indications aside from just wet AMD. Susvimo has a black box warning for endophthalmitis , an infection inside the eye which is a medical emergency. Approval was based on the phase 3 A rchway trial which demonstrated equivalent visual acuity results between Susvimo and Lucentis.Susvimo (ranibizumab) will be considered for coverage when the following criteria are met:Neovascular (wet) Age-related Macular Degeneration (AMD)For initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with an ophthalmologist; AND 3. Member has a diagnosis of wet AMD; AND 4. Member has previously responded to at least 2 intravitreal injections of a VEGF inhibitor; bevacizumab is the preferred product (others require prior authorization); AND 5. Documentation of best-corrected visual acuity (BCVA); AND 6. Member does NOT have any ocular or periocular infections or active intraocular inflammation. 7. Dosage allowed/Quantity limit: 2 mg via surgical administration every 6 months. (1 single dose vial per eye per 6 months) If all the above requirements are met , the medication will be approved for 6 months. For reauthorization : 1. Chart notes must include documentation of improved or stabilized visual acuity. If all the above requirements are met , the medication will be approved for an additional 12 months . IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023 CareSource considers Susvimo (ranibizumab) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION11/09/2021 New policy for Susvimo created.05/19/2023 Added baseline BCVA documentation.References: 1. SUSVIMO [package insert]. South San Francisco, CA: Genentech, Inc; 2022. 2. Flaxel CJ, Adelman RA, Bailey ST, et al. Age-Related Macular Degeneration Preferred Practice Pattern [published correction appears in Ophthalmology. 2020 Sep;127(9):1279]. Ophthalmology . 2020;127(1):P1-P65. doi:10.1016/j.ophtha.2019.09.024 3. Solomon SD, Lindsley K, Vedula SS, Krzystolik MG, Hawkins BS. Anti-vascular endothelial growth factor for neovascular age-related macular degeneration. Cochrane Database Syst Rev . 2019;3(3):CD005139. Published 2019 Mar 4. doi:10.1002/14651858.CD005139.pub4 4. A Phase III Study to Evaluate the Port Delivery System With Ranibizumab Compared With Monthly Ranibizumab Injections in Participants With Wet Age-Related Macular Degeneration (Archway) . ClinicalTrials.gov Identifier: NCT03677934. Updated October 28, 2021. Accessed November 10, 2021. https://clinicaltrials.gov/ct2/show/NCT03677934Effective date: 10/01/2023 Revised date: 05/19/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Ranibizumab (Lucentis, Byooviz, Cimerli)BENEFIT TYPE Medical STATUS Prior Authorization Required Lucentis was approved by the FDA in 2006. It is indicated for the treatment of several ophthalmic conditions. Lucentis is a vascular endothelial growth factor (VEGF) inhibitor for intravitreal use. VEGF inhibitors suppress endothelial cell proliferation, neovascularization, and vascular permeability . There are 2 forms of age-related macular degeneration (AMD), dry and wet (neovascular). Lucentis is approved for the treatment of Wet AMD which is less common but progresses more quickly. Neovascular in the context of AMD means growth of new blood vessels under the macula which can lead to loss of central vision. Diabetic eye disease includes diabetic retinopathy (DR) and diabetic macular edema (DME). DR affects blood vessels in the retina at the back of the eye. DME is a consequence of DR that occurs in about half of DR patients. It causes fluid build-up in the macula part of the retina. Retinal Vein Occlusion (RVO ) occurs when there is a partial or complete obstruction of a retinal vein. Macular edema is a complication of RVO and can lead to vision loss. It is treated first-line with anti-VEGF drugs. Myopia (nearsightedness) occurs when the eyeball becomes elongated. In pathological myopia, progressive elongation can cause a weakened sclera to bulge at the posterior of the eye which can lead to thinning of the retina and growth of new blood vessels. This can result in vision loss if not treated. Two biosimilar products have also been approved, Byooviz and Cimerli. Cimerli is interchangeable with Lucentis but Byooviz is not.Ranibizumab will be considered for coverage when the following criteria are met:Retinal DiseaseFor initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with an ophthalmologist; AND 3. Member has a confirmed diagnosis of one of the following conditions: a) Neovascular (Wet) Age-Related Macular Degeneration (AMD) b) Macular Edema Following Retinal Vein Occlusion (RVO ) c) Diabetic Macular Edema (DME) [Lucentis or Cimerli only] d) Diabetic Retinopathy (DR) [Lucentis or Cimerli only] e) Myopic Choroidal Neovascularization (mCNV) ; AND 4. Member has tried and failed bevacizumab intravitreal injection; AND 5. Documentation of best-corrected visual acuity (B CVA); AND 6. Member does NOT have active infection or inflammation in or around the eye(s) to be treated. 7. Dosage allowed/Quantity limit: AMD: 0.5 mg every 28 days (may extend to every 3 months after 4 monthly doses). RVO: 0.5 mg every 28 days DME or DR: 0.3 mg every 28 days CNV: 0.5 mg every 28 days for up to 3 months. Re-treat if needed. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023If all the above requirements are met , the medication will be approved for 6 months (3 months for mCNV). For reauthorization : 1. Chart notes must include documentation of improved or stabilized visual acuity. If all the above requirements are met , the medication will be approved for an additional 12 months (3 months for mCNV) . CareSource considers Ranibizumab not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION10/26/2021 New policy for Lucentis created. 04/07/2023 Changed policy name to Ranibizumab and added biosimilars. References: 1. Lucentis [prescribing information]. Genentech, Inc.; 2018. 2. Byooviz [prescribing information]. Biogen, Inc.; 2022. 3. Cimerli [prescribing information]. Coherus BioSciences, Inc.; 2022. 4. Flaxel CJ, Adelman RA, Bailey ST, et al. Age-Related Macular Degeneration Preferred Practice Pattern [published correction appears in Ophthalmology. 2020 Sep;127(9):1279]. Ophthalmology . 2020;127(1):P1-P65. doi:10.1016/j.ophtha.2019.09.024 5. Solomon SD, Lindsley K, Vedula SS, Krzystolik MG, Hawkins BS. Anti-vascular endothelial growth factor for neovascular age-related macular degeneration. Cochrane Database Syst Rev . 2019;3(3):CD005139. Published 2019 Mar 4. doi:10.1002/14651858.CD005139.pub4 6. Holekamp, Nanvy M. Review of Neovascular Age-Related Macular Degeneration Treatment Options. Am JManag Care. July 2019; 25: -S0 7. Flaxel CJ, Adelman RA, Bailey ST, et al. Retinal Vein Occlusions Preferred Practice Pattern [published correction appears in Ophthalmology. 2020 Sep;127(9):1279]. Ophthalmology . 2020;127(2):P288-P320. doi:10.1016/j.ophtha.2019.09.029 8. Shalchi Z, Mahroo O, Bunce C, Mitry D. Anti-vascular endothelial growth factor for macular oedema secondary to branch retinal vein occlusion. Cochrane Database Syst Rev . 2020;7(7):CD009510. Published 2020 Jul 7. doi:10.1002/14651858.CD009510.pub3 9. Flaxel CJ, Adelman RA, Bailey ST, et al. Diabetic Retinopathy Preferred Practice Pattern [published correction appears in Ophthalmology. 2020 Sep;127(9):1279]. Ophthalmology . 2020;127(1):P66-P145. doi:10.1016/j.ophtha.2019.09.025 10. Virgili G, Parravano M, Evans JR, Gordon I, Lucenteforte E. Anti-vascular endothelial growth factor for diabetic macular oedema: a network meta-analysis. Cochrane Database Syst Rev . 2018;10(10):CD007419. Published 2018 Oct 16. doi:10.1002/14651858.CD007419.pub6 11. Zhu Y, Zhang T, Xu G, Peng L. Anti-vascular endothelial growth factor for choroidal neovascularisation in people with pathological myopia. Cochrane Database Syst Rev . 2016;12(12):CD011160. Published 2016 Dec 15. doi:10.1002/14651858.CD011160.pub2 12. Pham B, Thomas SM, Lillie E, et al. Anti-vascular endothelial growth factor treatment for retinal conditions: a systematic review and meta-analysis. BMJ Open. 2019;9(5):e022031. Published 2019 May 28. doi:10.1136/bmjopen-2018-022031 Effective date: 10/01/2023 Revised date: 04/07/2023
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Cerdelga (eliglustat)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Cerdelga is a substrate reduction therapy, FDA approved in 2014 for the treatment of Gaucher disease type 1. Gaucher disease is a rare, inherited, lysosomal storage disorder. In Gaucher disease, mutations of the GBA gene cause deficiency of the enzyme glucocerebro sidase (acid beta-glucosidase), resulting in the accumulation of glucocerebroside (glucosylceramide [GLC ]) in the lysosomes of macrophages to form Gaucher cells, especially in the bone marrow, spleen, and liver. Prominent symptoms include hepatosplenomegaly, anemia, thrombocytopenia, and skeletal problems. Type 1 Gaucher disease is the most common form and does not affect the central nervous system. In contrast to standard of care enzyme replacement therapy (ERT), Cerdelga reduces synthesis of the accumulating substrate to compensate for its impaired degradation.Cerdelga (eliglustat) will be considered for coverage when the following criteria are met:Gaucher disease type 1 (GD1)For initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a geneticist, hematologist, or metabolic specialist ; AND 3. Member has a diagnosis of Gaucher disease type 1 confirmed by document ation of at least one of the following: a) Deficiency of glucocerebrosidase activity by enzyme assay (0 to 15% of normal), and/or b) Molecular genetic test documenting 2 mutations (biallelic variants) of the GBA gene ; AND 4. CYP2D6 genotype analysis shows the member is an extensive, intermediate, or poor metabolizer; AND 5. Member exhibits at least one of the following as a result of Gaucher disease: a) Anemia, b) Thrombocytopenia, c) Spleen and/or liver enlargement; AND 6. Member does NOT have any of the following: a) Neurologic symptoms suggestive of type II or III Gaucher disease (i.e., supranuclear gaze palsy, cognitive decline, epilepsy, myoclonus and/or ataxia), b) CYP2D6 ultra-rapid or indeterminate metabolizer status , c) Pre-existing cardiac disease, long QT syndrome, or in combination with Class IA (e.g., quinidine, procainamide) or Class III (e.g., amiodarone, sotalol) antiarrhythmic medications , d) Concomitant use with Zavesca (miglustat) or ERT. 7. Dosage allowed/Quantity limit: Recommended dosing is based on CYP2D6 metabolizer status: IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023NOTE: Dose adjustments may be needed based on other medications the member is taking . Consult the complete prescribing information from the manufacturer . QL: 56 capsules per 28 days. If all the above requirements are met , the medication will be approved for 12 months .For reauthorization :1. Chart notes must document improvement in one or more of the following parameters compared to baseline: a) Hemoglobin level b) Platelet count c) Spleen and/or liver volumes If all the above requirements are met , the medication will be approved for an additional 12 months.CareSource considers Cerdelga (eliglustat) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION08/04/2021 New policy for Cerdelga created.05/11/2023 Added references. Clarified that 2 mutations of the GBA gene should be present. Removed bone outcomes from reauth section; was not measured in clinical trials and unlikely to be evident at 12 mo.References: 1. Cerdelga [package insert]. Waterford, Ireland: Genzyme Ireland, Ltd.; 2022. 2. Mistry PK, Lukina E, Ben Turkia H, et al. Effect of oral eliglustat on splenomegaly in patients with Gaucher disease type 1: the ENGAGE randomized clinical trial. JAMA. 2015;313(7):695-706. doi:10.1001/jama.2015.459 3. Lukina E, Watman N, Arreguin EA, et al. A phase 2 study of eliglustat tartrate (Genz-112638), an oral substrate reduction therapy for Gaucher disease type 1. Blood . 2010;116(6):893-899. doi:10.1182/blood-2010-03-273151 4. Lukina E, Watman N, Dragosky M, et al. Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial. Am JHematol . 2019;94(1):29-38. doi:10.1002/ajh.25300 5. Cox TM, Drelichman G, Cravo R, et al. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood . 2017;129(17):2375-2383. doi:10.1182/blood-2016-12-758409 6. Belmatoug N, Di Rocco M, Fraga C, et al. Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe. Eur JIntern Med. 2017;37:25-32. doi:10.1016/j.ejim.2016.07.011 7. Balwani M, Burrow TA, Charrow J, et al. Recommendations for the use of eliglustat in the treatment of adults with Gaucher disease type 1 in the United States. Mol Genet Metab. 2016;117(2):95-103. doi:10.1016/j.ymgme.2015.09.002 8. Stone WL, Basit H, Master SR. Gaucher Disease. [Updated 2022 Jun 7]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK448080/ 9. Pastores GM, Hughes DA. Gaucher Disease. 2000 Jul 27 [Updated 2023 Mar 9]. In: Adam MP, Mirzaa GM, Pagon RA, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2023. Available from: https://www.ncbi.nlm.nih.gov/ books/NBK1269/IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/202310. Dardis A, Michelakakis H, Rozenfeld P, et al. Patient centered guidelines for the laboratory diagnosis of Gaucher disease type 1. Orphanet JRare Dis . 2022;17(1):442. Published 2022 Dec 21. doi:10.1186/s13023-022-02573-6 11. Torralba-Cabeza M, Morado-Arias M, Pijierro-Amador A, Fernndez-Canal MC, Villarrubia-Espinosa J. Recommendations for oral treatment for adult patients with type 1 Gaucher disease [published online ahead of print, 2022 Jun 5]. Rev Clin Esp (Barc ). 2022;S2254-8874(22)00043-1. doi:10.1016/j.rceng.2022.02.008 Effective date: 10/01/2023 Revised date: 05/11/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Amvuttra (vutrisiran)BENEFIT TYPE Medical STATUS Prior Authorization Required Amvuttra is a transthyretin-directed small interfering RNA indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. It is a n RNA interference (RNAi) drug that causes degradation of mutant and wild-type TTR mRNA, which results in a reduction of serum TTR protein and TTR protein deposits in tissues by delivering t he vutisiran small interfering RNA ( siRNA) to hepatocytes where TTR protein is synthesized. In the HELIOS-A study, Amvuttra met the primary effica cy endpoint of change from baseline to Month 9 in modified Neuropathy Impairment Score +7 (mNIS+7) , an objective assessment of neuropathy . hATTR is a rare and progressive inherited disorder where misfolded TTR accumulates as amyloid fibrils in the body. In polyneuropathy of hATTR (hATTR-PN), these fibrils deposit in the peripheral nerves which leads to pain, muscle weakness, and autonomic dysfunction. Onpattro, which has the same mechanism as Amvuttra, and Tegsedi are two othe r drugs approved for the treatment of hATTR-PN. Amvuttra has the advantage of less frequent dosing than Onpattro.Amvuttra (vutrisiran) will be considered for coverage when the following criteria are met:Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) : PolyneuropathyFor initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a neurologist; AND 3. Member has a diagnosis of hATTR amyloidosis with documentation of a transthyretin (TTR) mutation confirmed by genetic testing; AND 4. Member has signs/symptoms of polyneuropathy; AND 5. Member has a polyneuropathy disability (PND) score of IIIb or less (i.e., member is not wheelchair-bound or bedridden) ; AND 6. Member has NOT had a liver transplant; AND 7. Amvuttra is NOT being used in combination with another hATTR drug (e.g., Onpattro, Tegsedi, Vyn daqel, Vyndamax) . 8. Dosage allowed/Quantity limit: 25 mg subQ every 3 months, administered by a healthcare professional . (1 syringe per 84 days) If all the above requirements are met , the medication will be approved for 9 months. For reauthorization : 1. Chart notes must include documentation of positive clinical response to therapy such as improvement or stabilization of neuropathy impairment, gait speed, nutritional status, disability, or quality of life compared to baseline. If all the above requirements are met , the medication will be approved for an additional 12 months. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023 CareSource considers Amvuttra (vutrisiran) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION08/01/2022 New policy for Amvuttra created.04/19/2023 Added or stabilization to the renewal section.References: 1. Amvuttra. Prescribing information. Alnylam Pharmaceuticals, Inc.; 2022. 2. Adams D, Tournev IL, Taylor MS, et al. Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial [published online ahead of print, 2022 Jul 23]. Amyloid. 2022;1-9. doi:10.1080/13506129.2022.2091985 3. Ando Y, Adams D, Benson MD, et al. Guidelines and new directions in the therapy and monitoring of ATTRv amyloidosis [published online ahead of print, 2022 Jun 2]. Amyloid . 2022;1-13. doi:10.1080/13506129.2022.2052838 4. Sekijima Y. Hereditary Transthyretin Amyloidosis. 2001 Nov 5 [Updated 2021 Jun 17]. In: Adam MP, Mirzaa GM, Pagon RA, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2022. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1194/ 5. Dyck PJB, Gonzlez-Duarte A, Obici L, et al. Development of measures of polyneuropathy impairment in hATTR amyloidosis: From NIS to mNIS+7. JNeurol Sci . 2019;405:116424. doi:10.1016/j.jns.2019.116424 6. Adams D, Ando Y, Beiro JM, et al. Expert consensus recommendations to improve diagnosis of ATTR amyloidosis with polyneuropathy. JNeurol . 2021;268(6):2109-2122. doi:10.1007/s00415-019-09688-0 Effective date: 10/01/2023 Revised date: 04/19/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME R evcovi (elapegademase-lvlr )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Revcovi is a recombinant adenosine deaminase (rADA) indicated for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients . It acts as an enzyme replacement therapy (ERT). SCID associated with a deficiency of ADA enzyme is a very rare, inherited, and often fatal purine salvage pathway defect . ADA deficiency is a primary immunodeficiency caused by a genetic mutation that affects white blood cell (WBC) production. ADA-SCID is the most complete form of ADA deficiency and typically leads to a severe combined immunodeficiency with dysfunction of T, B, and natural killer (NK) cells (T-B-NK-SCID), presenting in the first few months of life. Non-immune symptoms also occur . Revcovi provides an exogenous source of ADA enzyme that is associated with a decrease in toxic adenosine and deoxyadenosine nucleotides levels as well as an increase in lymphocyte number. It is a second-generation ADA replacement therapy, taking the place of the discontinued first-generation ADA replacement product, Adagen. All newly diagnosed patients should initially receive ERT. Ideally, it is used as a bridge prior to allogeneic hematopoietic stem cell transplantation (HSCT) when that is an option. Treatment with Revcovi requires extensive monitoring. Immune function, including the ability to produce antibodies, generally improves after 2-6 months of therapy, and matures over a longer period. Improvement in the general clinical status of the patient may be gradual but should be apparent by the end of the first year . I mmune deficient patients must maintain precautions from infections until immune function has been improved.Revcovi (elapegademase-lvlr) will be considered for coverage when the following criteria are met:A denosine D eaminase Severe C ombined Immune D eficiency (ADA-SCID) For initial authorization: 1. Medication must be prescribed by or in consultation with an immunologist , geneticist , or hematologist/oncologist ; AND 2. Member has a diagnosis of ADA-SCID confirmed by at least one of the following: a) Absent or very low ADA activity (
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Takhzyro (lanadelumab-flyo)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Takhzyro is a plasma kallikrein inhibitor (monoclonal antibody) indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 2 years and older . HAE is a rare autosomal dominant disease characterized by episodic unpredictable swelling, which can occur in a variety of anatomic locations. The swelling results from excess production of the vasodilator bradykinin. Attacks may be painful and cause funct ional impairment but are not associated with pruritis. The most common types of HAE are caused by deficiency (type 1) or dysfunction (type 2) of C1 inhibitor (C1-INH). Type 1 is the most prevalent.Takhzyro (lanadelumab-flyo) will be considered for coverage when the following criteria are met:Hereditary Angioedema (HAE)For initial authorization: 1. Member must be 2 years of age or older; AND 2. Medication must be prescribed by or in consultation with an allergist or immunologist; AND 3. Member has a diagnosis of HAE type I or type II confirmed by both of the following: a) Low C4 level; b) Low (6 years of age. References: 1. Takhzyro [package insert]. Lexington, MA: Dyax Corp.; 202 3. 2. Lumry W. Management and Prevention of Hereditary Angioedema Attacks. Am JManag Care. 2013;19:S111-S118. 3. Busse PJ, Christiansen SC, Riedl MA, et al. US HAEA Medical Advisory Board 2020 Guidelines for the Management of Hereditary Angioedema [published online ahead of print, 2020 Sep 6]. JAllergy Clin Immunol Pract . 2020;S2213-2198(20)30878-3. doi:10.1016/j.jaip.2020.08.046. 4. Banerji A, Riedl MA, Bernstein JA, et al. Effect of Lanadelumab Compared With Placebo on Prevention of Hereditary Angioedema Attacks: A Randomized Clinical Trial [published correction appears in JAMA. 2019 Apr 23;321(16):1636]. JAMA . 2018;320(20):2108-2121. doi:10.1001/jama.2018.16773 5. Banerji A, Bernstein JA, Johnston DT, et al. Long-term prevention of hereditary angioedema attacks with lanadelumab: The HELP OLE Study. Allergy . 2022;77(3):979-990. doi:10.1111/all.15011 6. Betschel S, Badiou J, Binkley K, et al. The International/Canadian Hereditary Angioedema Guideline [published correction appears in Allergy Asthma Clin Immunol. 2020 May 6;16:33]. Allergy Asthma Clin Immunol. 2019;15:72. Published 2019 Nov 25. doi:10.1186/s13223-019-0376-8 7. Maurer M, Magerl M, Betschel S, et al. The international WAO/EAACI guideline for the management of hereditary angioedema-The 2021 revision and update. World Allergy Organ J . 2022;15(3):100627. Published 2022 Apr 7. doi:10.1016/j.waojou.2022.100627 8. Mendivil J, Malmens M, Haeussler K, Hunger M, Jain G, Devercelli G. Indirect Comparison of Lanadelumab and Intravenous C1-INH Using Data from the HELP and CHANGE Studies: Bayesian and Frequentist Analyses. Drugs RD . 2021;21(1):113-121. doi:10.1007/s40268-021-00337-4 Effective date: 08/01/2023 Revised date: 02/20/2023
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Tavalisse (fostamatinib disodium hexahydrate)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Tavalisse, approved by the FDA in 2018, is a kinase inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment . It has demonstrated activity against spleen tyrosine kinase (SYK), and the active metabolite (R406) reduces antibody-mediated destruction of platelets. Approval was based on the FIT clinical trial program. Immune thrombocytopenia (ITP) is a rare autoimmune disorder characterized by low levels of platelets due to platelet destruction and insufficient platelet production. ITP duration of less than 3 months is referred to as newly diagnosed, 3-12 months as persistent, and greater than 12 months is considered chronic.Tavalisse (fostamatinib disodium hexahydrate) will be considered for coverage when the following criteria are met:C hronic Immune Thrombocytopenia (ITP)For initial authorization: 1. Member is at least 1 8 years of age; AND 2. Medication must be prescribed by or in consultation with a hematologist; AND 3. Member has a documented diagnosis of chronic ITP of at least 6 months duration; AND 4. Member had an inadequate response, intolerance, or contraindication to documented prior therapy with at least one of the following treatments: a) Corticosteroids ( i.e., prednisone, prednisolone, methylprednisolone, dexamethasone) b) Immunoglobulins c) Splenectomy; AND 5. Member has tried and failed treatment with a thrombopoietin receptor agonist (TPO-RA) (i.e., Promacta, Nplate, or Doptelet ); AND 6. Member meets one of the following: a) Current platelet count is 60 years), other clearly identified comorbidity) ; AND 7. Member does NOT have any of the following: a) Secondary immune thrombocytopenia (i.e., non-idiopathic, due to another condition) b) Uncontrolled or poorly controlled hypertension c) History of coagulopathy including prothrombotic conditions . 8. Dosage allowed/Quantity limit: Initiate at 100 mg orally twice daily . After 4 weeks, increase to 150 mg twice daily, if needed, to achieve platelet counts of at least 50 x 10 9 /L as necessary to reduce the risk of bleeding . IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023QL: 60 tablets/30 daysNote : Discontinue Tavalisse after 12 weeks of treatment if the platelet count does not increase to a level sufficient to avoid clinically important bleeding. If all the above requirements are met , the medication will be approved for 6 months . For reauthorization :1. Members platelet count of at least 50 x 10 9 /L was achieved and documented in chart notes . If all the above requirements are met, the medication will be approved for an additional 12 months .CareSource considers Tavalisse (fostamatinib disodium hexahydrate) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy.DATE ACTION/DESCRIPTION08/31/2018 New policy for Tavalisse created. 02/02/2023 Transferred policy to new template. Updated and added references. Changed disease duration of at least 3 months to at least 6 months to match definition of chronic disease more closely. Added Doptelet as a TPO-RA option with Promacta, Nplate. Changed platelet count of
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