IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Izervay (avacincaptad pegol)BENEF IT TYPE Medical ST AT US Prior Authorization Required Izervay, approved by the FDA in 2023, is a complement C5 inhibitor indicated for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). It is the second drug approved f or this indication f ollowing C3 inhibitor pegcetacoplan. There are 2 types of AMD: dry or wet (neovascular). Izervay 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 t h at can impair vision or lead to blindness. Approval of Izervay was based the GAT HER studies . Although it slows the growth rate of GA lesions, Izervay does not appear to preserve visual f unction. It may also accelerate the development of new-onset wet AMD .Izervay (avacincaptad pegol) will be considered for coverage when the following criteria are met:Geographic Atrophy (GA)For initial authorization: 1. Member is at le as t 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 conf irmed by f undus autof luorescence (FAF) imaging showing all of the f ollowing: a) Total GA area must be 2.5 and 17.5 mm2 (1 and 7 disk areas [DA] respectively) and b) If GA is multif ocal, at least one f ocal lesion must be 1.25 mm2 (0.5 DA) c) The GA lesion must be, in part, within 1.5 mm f r o m, but NOT involving the f oveal center ; AND 5. Documentation of best corrected visual acuity (BCVA) between 20/25 and 20/320 in the af f ected eye(s) ; AND 6. Member does NOT have any of the f ollowing: a) GA secondary to any condition other than AMD b) History or current evidence of wet AMD 7. Dosage allowed/Quantity limit: Intravitreal injection to each af f ected eye once monthly . QL: 2 vials per 28 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 Izervay (avacincaptad pegol) 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/DESCRIPTION09/18/2023 New policy f or Izervay created. 03/06/2025 Updated ref erences. Added renewal criteria; label no longer limits to 12 months total duration. Ref erenc es : 1. Izerv ay [p res c rib ing inf o rmatio n] . IV ERIC b i o , Inc . ; 202 5. 2. Jaf fe GJ , Westby K, Cs ak y KG, et al. C5 Inhib ito r Av ac inc ap tad Peg o l f o r Geo g rap hic Atro p hy Due to Ag e-Related Mac ular Degeneratio n: A Rand o mized Piv o tal Phas e 2/3 Trial. Ophthalmology . 2021;128(4):576-586. d o i:10.1016/j.o p htha.2020.08.027 3. Patel SS, Lally DR, Hs u J , et al. Avacincaptad p egol for g eo g rap hic atro p hy s ec o nd ary to ag e-related mac ular d eg eneratio n: 18-mo nth f ind ing s f ro m the GATHER1 trial [p ub lis hed o nline ahead o f p rint, 2023 Mar 24] [p ub lished c orrection ap pears in Ey e (Lo nd ). 2023 May 26;:]. Ey e (Lond). 2023;10.1038/s 41433-023-02497-w. d o i:10.1038/s 41433-023-02497-w 4. Tzo umas N, Rid ing G, Williams MA, Steel DH. Co mp lement inhib ito rs f o r ag e-related mac ular d eg eneratio n. Coc hrane Databas e Sy s t Rev . 2023;6(6):CD 009300. Pub lis hed 2023 Jun 14. d o i:10.1002/14651858.CD 009300.p ub 3 5. Cruz-Pimentel M, Wu L. Co mp lement Inhib ito rs f o r Ad v anc ed Dry Ag e-Related Mac ular Deg eneratio n (Geo g rap hic Atrophy): Some Light at the End of the Tunnel?. JClin Med. 2023;12(15):5131. Published 2023 Aug 4. d o i:10.3390/jc m12155131 6. Vemulak o nda GA, Bailey ST, Kim SJ , et al. Ag e-Related Mac ular Deg eneratio n Pref erred Prac tic e Pattern . Ophthalmology . Pub lis hed o nline Feb ruary 7, 2025. d o i:10.1016/j.o p htha.2024.12.018 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 03/06/2025
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Imaavy (nipocalimab-aahu)BENEF IT TYPE Medical ST AT US Prior Authorization Required I maav y is a neonatal Fc receptor blocker indicated for the treatment of generalized my as t h e n ia g r av is ( gMG) in adult and pediatric patients 12 years of age and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specif ic tyrosine kinase (MuSK) antibody positive. Myasthenia gravis is an autoimmune disorder af f ecting the neuromuscular junction, characterized by muscle weakness and f atigue. The cause is an antibody-mediated immunologic attack directed at proteins in the postsynaptic membrane of the neuromuscular junc tion, most commonly the acetylcholine receptor (90%). Autoantibodies attack the AChR, blocking or destroying the receptors and damaging the neuromuscular junction, which impairs neuromuscular transmission and prevents muscles from contracting, as acetylcholine is unable to activate its receptor. Ocular motility, swallowing, speech, mobility, and respiratory f unction can all be af f ected. Pyridostigmine, an acetylcholinesterase inhibitor, is the initial drug of choice prescribed f or MG. It eases symptoms by slowing the breakdown of acetylcholine. If control is inadequate, immunosuppressive treatment is added, such as prednisone and/or azathioprine. Other drugs are used in cases of severe or refractory MG or in myasthenic crisis, which is an emergency .Imaavy (nipocalimab-aahu) will be considered for coverage when the following criteria are met:Myasthenia Gravis For initial authorization: 1. Member is at le as t 12 years of age; AND 2. Medication must be prescribed by or in consultation with a neurologist; AND 3. Member has a documented diagnosis of MGFA class II-IV myasthenia gravis (see appendix) ; AND 4. Lab result in chart notes shows the member is seropositive f or AChR or MuSK antibodies ; AND 5. Member has tried and f ailed at least 1 conventional therapy: a) pyridostigmine b) corticosteroid f or at least 4 weeks c) non-steroid immunosuppressant (e.g., azathioprine) f or at least 6 months 6. Dosage allowed/Quantity limit: Initial: 30 mg/kg once via IV inf usion Maintenance: two weeks af ter initial dose, administer 15 mg/kg IV inf usion every two weeks thereafter If all the above requirements are met , the medication will be approved for 6 months . IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023For reauthorization :1. Chart notes must document clinically meaningf ul improvement in symptom severity and daily f unctioning compared to pre-treatment baseline (e.g., improved MG-ADL or QMG scores ). If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Imaavy (nipocalimab-aahu) 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/DESCRIPTION05/06/2025 New policy f or I maav y created. Ref erenc es : 1. Imaav y [p res c rib ing inf o rmatio n]. Jans s en Bio tec h, Inc .; 2025. 2. Anto zzi C, Vu T, Ramc handren S, et al. Safety and efficacy of nipocalimab in ad ults with generalised my as thenia gravis (Vivacity-MG3): a p has e 3, rand o mis ed , d o ub le-blind, placebo-controlled study. Lanc et Neurol . 2025;24(2):105-116. d o i:10.1016/S1474-4422(24)00498-8 3. Sand ers DB, Wolfe GI, Benatar M, et al. International c onsensus g uidance for management of myasthenia g ravis: Ex ec utiv e s ummary . Neurology . 2016;87(4):419-425. d o i:10.1212/WNL.0000000000002790 4. Naray anas wami P, Sand ers DB, Wo lf e G, et al. Internatio nal Co ns ens us Guid anc e f o r Manag ement o f My as thenia Grav is : 2020 Up d ate. Neurology . 2021;96(3):114-122. d o i:10.1212/WNL.0000000000011124 5. Alhaid ar MK, Ab umurad S, So liv en B, Rezania K. Current Treatment o f My as thenia Grav is . JClin Med. 2022;11(6):1597. Pub lis hed 2022 Mar 14. d o i:10.3390/jc m11061597 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 05/06/2025
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Iluvien (fluocinolone acetonide )BENEF IT TYPE Medical ST AT US Prior Authorization Required Iluvien , approved by the FDA in 2014, is an intravitreal implant containing 0.19 mg (190 mcg) f luocinolone acetonide in a 36-month sustained-release drug delivery system . It is indicated f or the treatment of diabetic macular edema (DME) in patients who have been previously treated with a course of corticosteroids and did not have a clinically signif icant rise in intraocular pressure. DME is a common complication of diabetic retinopathy. Iluvien is also indicated f or the treatment of chronic non-inf ectious uveitis af f ecting the posterior segment of the eye. Uveitis is an inf lammation of the uvea (middle layer of the eye). It can be inf ectious or non-in f ec t ious. Noninf ectious uveitis (NIU) is of ten associated with inf lammatory conditions such as rheumatoid arthritis. If the anterior segment of the uvea is af f ected, it can be treated with topical glucocorticoids. If resistant or af f ecting the intermediate or posterior segments, more invasive or systemic treatment is needed.Iluvien (fluocinolone acetonide) will be considered for coverage when the following criteria are met:D iabetic Macular Edema (DME)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 conf irmed diagnosis of diabetic macular edema; AND 4. Member has been previously treated with a course of corticosteroids and did not have a clinically signif icant increase in intraocular pressure; AND 5. Member has tried and f ailed Ozurdex or an an t i-VEGF drug ( bevacizumab pref erred); AND 6. Member does not have active or suspected ocular or periocular inf ection; AND 7. Member does not have glaucoma with a cup to disc ratio greater than 0.8. 8. Dosage allowed/Quantity limit: One implant (0.19 mg) per eye Limit: 2 implants (1 per eye) per 36 months. If all the above requirements are met , the medication will be approved for 3 months . For reauthorization : 1. Chart notes must show improved or stabilized visual acuity f ollowing treatment; AND 2. At least 36 months have elapsed since the prior treatment (of the same eye). If all the above requirements are met , the medication will be approved for an additional 3 months.IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023UveitisFor 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 chronic (1 year or more) non-inf ectious uveitis af f ecting the posterior segment of the eye; AND 4. Member has tried and f ailed at least one of the f ollowing f or at least 3 months: a) Systemic corticosteroid (e.g., prednisone) b) Non-biologic immunosuppressive (e.g., mycophenolate mof etil, methotrexate, cyclosporine, tacrolimus); AND 5. Member does not have active or suspected ocular or periocular inf ection; AND 6. Dosage allowed/Quantity limit: One implant (0.19 mg) per eye Limit: 2 implants (1 per eye) per 36 months. If all the above requirements are met , the medication will be approved for 3 months . For reauthorization : 1. Chart notes must show improved or stabilized visual acuity f ollowing treatment and/or an improved vitreous haze score; AND 2. At least 36 months have elapsed since the prior treatment (of the same eye) ; AND 3. Member has recurrent symptoms. If all the above requirements are met , the medication will be approved for an additional 3 months . CareSource considers Iluvien (fluocinolone acetonide) 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/DESCRIPTION10/27/2021 New policy created f or Iluvien. 10/16/2023 Ref erences updated . Added an t i-VEGF as trial option. 04/02/2025 Added criteria f or new indication: Uveitis. Ref erenc es : 1. Iluv ien [p res c rib ing inf o rmatio n]. Alimera Sc ienc es , Inc .; 2025. 2. Flax el CJ , Adelman RA, Bailey ST, et al. Diabetic Retinopathy Preferred Practice Pattern [pub lis hed c o rrec tio n ap p ears in Op hthalmo lo g y . 2020 Sep ;127(9):1279]. Ophthalmology . 2020;127(1):P66-P145. d o i:10.1016/j.o p htha.2019.09.025 3. Virg ili G, Parrav ano M, Evans JR, Gordon I, Luc enteforte E. Anti-vas cular endo thelial g ro wth f ac to r f o r d iab etic mac ular o ed ema: a network meta-analy s is . Coc hrane Databas e Sy s t Rev . 2018;10(10):CD 007419. Pub lis hed 2018 Oc t 16. d o i:10.1002/14651858.CD 007419.p ub 6 4. Rittip hairoj T, Mir TA, Li T, Virg ili G. Intrav itreal steroids for macular edema in diabetes . Coc hrane Databas e Sy s t Rev . 2020;11(11):CD 005656. Pub lis hed 2020 No v 17. d o i:10.1002/14651858.CD 005656.p ub 3 5. Zur D , Ig lic k i M, Loewenstein A. The Ro le of Steroids in the Management of Diabetic Macular Edema. Ophthalmic Res . 2019;62(4):231-236. d o i:10.1159/000499540 6. Sc hmid t-Erfurth U, Garcia-Arumi J , Bandello F, et al. Guidelines for the Management of Diabetic Mac ular Ed ema b y the Euro p ean So c iety o f Retina Sp ec ialis ts (EURETINA). Ophthalmologic a. 2017;237(4):185-222. d o i:10.1159/000458539 7. Bailey C, Chak rav arthy U, Lotery A, Menon G, Talk s J ; Medis o f t Aud it Gro up . Ex tend ed real-wo rld ex p erienc e with the ILUVIEN (f luocinolone acetonide) implant in the United King d o m: 3-y ear res ults f ro m the Med is o f t aud it s tudy [published online ahead of print, 2021 May 10]. Eye (Lond) . 2021;1-7. d oi:10.1038/s41433-021-01542-wIN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/20238. Yuen YS, Gilho tra JS, Dalto n M, et al. Diab etic Mac ular Oed ema Guid elines : An Aus tralian Pers p ec tiv e. JOphthalmol . 2023;2023:6329819. Pub lis hed 2023 Feb 14. d o i:10.1155/2023/6329819 9. Jaf fe GJ , Pavesio CE; Study Inv estigators . Ef f ec t o f a Fluo c ino lo ne Ac eto nid e Ins ert o n Rec urrenc e Rates in No ninf ectious Intermediate, Po s terio r, o r Panuv eitis : Three-Year Res ults . Ophthalmology. 2020;127(10):1395-1404. d o i:10.1016/j.o p htha.2020.04.001 10. Red d y A, Liu SH, Brady CJ , Sieving PC, Palestine AG. Corticosteroid implants for c hronic no n-infec tio us uv eitis . Coc hrane Databas e Sy s t Rev . 2023;1(1):CD 010469. Pub lis hed 2023 Jan 16. d o i:10.1002/14651858.CD 010469.p ub 3 11. Tan HY, Ag arwal A, Lee CS, et al. Management of no ninfectious p os terio r uv eitis with intrav itreal d rug therap y . Clin Ophthalmol . 2016;10:1983-2020. Pub lis hed 2016 Oc t 13. d o i:10.2147/OPTH.S89341 12. Wu X, Tao M, Zhu L, Zhang T, Zhang M. Pathogenesis and current therapies for non-infectio us uv eitis. Clin Ex p Med. 2023;23(4):1089-1106. d o i:10.1007/s 10238-022-00954-6 13. Ab d ulla D, Ali Y, Menezo V, Tay lo r SRJ . The Us e o f Sus tained Releas e Intrav itreal Stero id Imp lants in No n-Inf ec tio us Uv eitis Af f ec ting the Po s terio r Seg ment o f the Ey e. Ophthalmol Ther . 2022;11(2):479-487. d o i:10.1007/s 40123-022-00456-4 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 04/02/2025
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Gomekli (m irdametinib)BENEF IT TYPE Ph ar mac y ST AT US Prior Authorization Required Gomekli is a kinase inhibitor initially approved by the FDA in 2025. It is indicated f or the treatment of neurof ibromatosis type 1 (NF1) in patients 2 years or older who have symptomatic plexif orm neurof ibromatosis not amendable to complete resection. NF1 is a genetic condition that causes various changes to skin pigments and tumor growth on nerve tissue. The tumors can grow anywhere in the nervous system but are typically benign. However, if large tumors are present, it could press onto nerves or organ s and cause damage. Gross-total resection is the f irst-line treatment f or NF1 with plexif orm neurofibromas (PN). For PNs that cannot be completely removed by surgery, systemic therapy may be appropriate. Gomekli was approved based on data f rom the phase IIb ReNeu trial in which adults and children reported signif icantly PN volume reductions and clinically meaningf ul improvement in pain.Gomekli (mirdametinib ) will be considered for coverage when the following criteria are met:Neurofibromatosis Type 1 (NF1)For initial authorization: 1. Member is at le as t 2 years of age; AND 2. Medication must be prescribed by or in consultation with an oncologist or neurologist; AND 3. Member has a diagnosis of neurof ibromatosis type 1 (NF1) with at least 1 of the f ollowing: a) Positive genetic test f or NF1 b) 6 or more caf-au-lait macules (CALMs) c) Axillary or inguinal f reckling d) Optic glioma e) 2 or more Lisch nodules f ) A distinctive osseous lesion g) Member is the child of a parent diagnosed with NF1; AND 4. Member has a diagnosis of symptomatic plexif orm neurof ibromatosis ; AND 5. Member has at least one measurable plexif orm neurofibromas (PN) as evidenced by MRI or PET-CT scan; AND 6. Prescriber attests that the members disease is not amendable to complete resection; AND 7. Member has received the f ollowing evaluation and testing prior to starting therapy: a) Comprehensive ophthalmic testing; b) Echocardiogram; AND 8. Gomekli is used as monotherapy; AND 9. Member has NOT previously had disease progression with a MEK inhibitor treatment. 10. Dosage allowed/Quantity limit: 2 mg/m 2 twice daily f or the f irst 21 days of each 28-day cycle. QL : 8 mg/day. If all the above requirements are met , the medication will be approved for 6 months. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023For reauthorization :1. Chart notes must show that the member had at least a partial response (def ined as 20% reduction in PN volume) f rom baseline and no disease progression and/or 2. Clinical improvement such as reduction of tumor pain or increased physical f unctioning. If all the above requirements are met , the medication will be approved for an additional 12 months CareSource considers Gomekli (mirdametinib) 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/DESCRIPTION05/05/2025 New policy f or Gomekli created. Ref erenc es : 1. Go mek li (Mird ametinib ) [p ac k ag e ins ert]. Stamf o rd , CT: Sp ring Wo rk s Therap eutic s , Inc .; 2025. 2. Pellerino A, Verd ijk RM, Nic helli L, And rats c hk e NH, Id b aih A, Go ld b runner R. Diag no s is and Treatment o f Perip heral and Cranial Nerv e Tumo rs with Expert Rec ommendations: An EUro p ean Netwo rk f o r RAre CANc ers (E URA CA N) Init iat iv e. Canc ers (Bas el ). 2023 Mar 23;15(7):1930. d o i: 10.3390/c anc ers 15071930. PMID : 37046591; PMCID : PMC10093509. 3. Neuro f ibromatosis ty pe 1 Symptoms and c auses. Mayo Clinic. Up dated September 10, 2024. Acc essed May 5, 2025. 4. Mo ertel CL, Hirb e AC, Shuhaib er HH, et al . ReNeu: A Piv o tal, Phas e IIb Trial o f Mird ametinib in Ad ults and Child ren With Sy mptomatic Neurofibro matos is Ty pe 1-Assoc iated Plex if o rm Neuro f ib ro ma. JClin Onc o l. 2025 Feb 20;43(6):716-729. d o i: 10.1200/J CO.24.01034. Ep ub 2024 No v 8. Erratum in: JClin Onc ol . 2025 Jan 10;43(2):239. d o i: 10.1200/J CO-24-02561. PMID : 39514826; PMCID : PMC11825507. 5. Leg ius E, Messiaen L, Wolkenstein P, et al. Rev ised diagnostic c riteria for neurofib ro mato s is ty p e 1 and Leg ius s y nd ro me: an international c onsensus recommendation. Genet Med. 2021;23(8):1506-1513. doi:10.1038/s 41436-021-01170-5 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 05/05/2025
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Galafold (migalastat)BENEF IT TYPE Ph ar mac y ST AT US Prior Authorization Required Galaf o ld is an alpha-galactosidase A (alpha-Gal A) pharmacological chaperone indicated f or the treatment of adults with a conf irmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data. It is estimated that the amenable variants are present in 35-50% of the Fabry disease patient population. Galaf old is an alternative to enzyme replacement therapy (ERT) and is taken orally. It increases activity of the def icient enzyme instead of replacing it. Galaf o ld was ap p roved by the FDA in 2018 with accelerated approval status. Fabry disease is an X-linked lysosomal storage disorder caused by mutations in the GLA gene that cause def iciency of the alpha-galactosidase A (alpha-Gal A) lysosomal enzyme. Normally this enzyme breaks down certain lipids in lysosomes, such as globotriaos ylceramide (GL-3). Without it, GL-3 accumulates in blood vessels, the kidneys, heart, nerves, and other organs.Galafold (migalastat) will be considered for coverage when the following criteria are met:Fabry DiseaseFor initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with a medical geneticist, nephrologist, cardiologist, neurologist, or metabolic specialist; AND 3. Member has a conf irmed diagnosis of Fabry disease; AND 4. Member has an amenable galactosidase alpha gene (GLA) variant (ref er to package insert) based on in vitro assay data documented in chart notes; AND 5. Member does NOT have severe renal impairment or end-stage renal disease requiring dialysis; AND 6. Galaf old will NOT be used in combination with ERT . 7. Dosage allowed/Quantity limit: 123 mg (1 capsule) orally every other day. QL: 14 capsules / 28 days If all the above requirements are met , the medication will be approved for 6 months . For reauthorization : 1. Chart notes must show positive clinical response such as stabilized kidney f unction (e.g., GFR, proteinuria), reduced plasma or tissue GL-3 levels, reduced left ventricular mass index, or other Fabry symptom improvement. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Galafold (migalastat) 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/DESCRIPTION05/20/2019 New policy f or Galaf o ld created.06/18/2021 Transf erred to new template. Updated ref erences. Added neurology to specialists.Removed baseline GL-3 level. Removed exclusions except renal impairment and combination therapy. Increased initial approval duration to 6 months and renewal to 12 months. Revised renewal criteria; removed % reductions. 11/22/2022 Annual review. Reorganized summary and added ref erence. 04/30/2025 Updated ref erences. Changed not to be used with Fabrazyme to not to be used with ERT . Split diagnosis and amenable variant into separate criteria. Ref erenc es : 1. Galaf o ld [p res c rib ing inf o rmatio n]. Philad elp hia, PA: Amic us Therap eutic s US, LLC; 2025. 2. Hug hes D , et al. Oral pharmacological c haperone migalastat c ompared with enzy me rep lac ement therap y in Fab ry d is eas e: 18-month res ults from the rand omised p has e III ATTRACTs tud y . Jo urnal o f med ic al g enetic s . 2017 Ap r 1;54(4):288-96. 3. Feld t-Ras mussen U, Hughes D , Sunder-Plas smann G, et al. Lo ng-term efficac y and s afety of migalastat treatment in Fab ry d iseas e: 30-month res ults fro m the open-label ex tens io n o f the rand o mized , p has e 3 ATTRACTs tud y . Mol Genet Metab. 2020;131(1-2):219-228. d o i:10.1016/j.y mg me.2020.07.007 4. Germain D , et al. Treatment of Fabrys d is ease with the p harmacologic c haperone migalastat. New Eng land Jo urnal o f Med ic ine. 2016 Aug 11;375(6):545-55. 5. Natio nal ins titute for health and c are ex cellenc e. Migalas tat f o r treating Fab ry d is eas e. 2017 Feb . Av ailab le f ro m: nic e.o rg .uk /g uid anc e/hs t4/c hap ter/1-Rec o mmend atio ns . 6. Ortiz A, et al. Fab ry disease rev isited: management and treatment rec o mmend atio ns f o r ad ult p atients . Mo lec ular g enetic s and metab o lis m. 2018 Ap r 1;123(4):416-27. 7. Wang R, et al. Ly s osomal s torage diseases: diagnostic confirmation and management of pres ymptomatic indiv iduals. Genetic s in Med ic ine. 2011 May ;13(5):457. 8. Laney DA, Bennett RL, Clarke V, et al. Fabry disease p ractic e guidelines: rec ommendations of the Natio nal So c iety o f Genetic Co uns elo rs . JGenet Couns . 2013;22(5):555-564. d o i:10.1007/s 10897-013-9613-3 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 04/30/2025
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Enzyme Replacement Therapy (ERT ) for Fabry Disease: Fabrazyme (agalsidase beta) and Elfabrio (pegunigalsidase alfa-iwxj ) BENEF IT TYPE Medical ST AT US Prior Authorization Required Fabrazyme, approved by the FDA in 2003, is an enzyme replacement therapy (ERT) indicated f or the treatment of conf irmed Fabry disease, to replace the enzyme alpha-galactosidase A (alpha-G al A) . Fabry disease, a lysosomal storage disorder, is a rare genetic disease caused by certain mutations of the GLA gene resulting in def icient alpha-Gal A. Normally this enzyme breaks down certain lipids in lysosomes, such as globotriaosylceramide (GL-3). Without it, GL-3 accumulates in blood vessels, the kidneys, heart, nerves, and other organs. The continuous build-up of GL-3 results in progressive cell damage and subsequent symptoms and manif estations in the af f ected organ systems. Elf abrio is a biobetter of Fabrazyme and was designed to have an increased half-lif e and reduced immunogenicity.ERT for Fabry Disease will be considered for coverage when the following criteria are met:Fabry DiseaseFor initial authorization: 1. For Fabrazyme: Member is at least 2 years of age OR f or Elf abrio: Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a medical geneticist, nephrologist, cardiologist, neurologist, or metabolic specialist ; AND 3. Member has a diagnosis of Fabry disease conf irmed by genetic testing which identif ies a pathogenic mutation of the GLA gene; AND 4. Member displays symptoms of Fabry disease (i.e., neuropathic pain, renal disease, cardiac disease, abdominal pain, impaired sweating) NOTE: Exception — Males with "classic" gene variants do not need to be symptomatic to qualif y f or treatment. Males with "non-classic" gene variants and asymptomatic f emales may be treated if there is evidence of injury to the heart, kidney, or central nervous system (CNS) ; AND 5. ERT will NOT be used in combination with Galaf old. 6. Dosage allowed/Quantity limit: 1 mg/kg every 2 weeks as an IV inf usion. If all the above requirements are met , the medication will be approved for 6 months . IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023For reauthorization :1. Chart notes must show positive clinical response such as stabilized kidney f unction (e.g., GFR, proteinuria), reduced plasma or tissue GL-3 levels, or other improved Fabry symptoms (such as neuropathic pain) . If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers ERT for Fabry Disease 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/DESCRIPTION06/17/2021 New policy f or Fab r az y me created.11/22/2022 Annual review; added ref erence.06/28/2023 Changed name of policy and added Elf abrio. Clarif ied note in #4. 04/30/2025 Updated ref erences. Ref erenc es : 1. Fab razy me (ag als id as e b eta) [p ac k ag e ins ert]. Camb rid g e, MA; Genzy me Co rp o ratio n; 2024. 2. Banik azemi M, Bultas J , Waldek S, et al. Agalsidas e-b eta therap y f o r ad v anc ed Fab ry d is eas e: a rand o mized trial. Ann Intern Med. 2007;146(2):77-86. d o i:10.7326/0003-4819-146-2-200701160-00148 3. Eng CM, Guf f o n N, Wilc o x WR, et al. Saf ety and ef f ic ac y o f rec o mb inant human alp ha-g alac to s id as e A rep lac ement therapy in Fabry 's d is ease. NEngl JMed. 2001;345(1):9-16. d o i:10.1056/NEJ M200107053450102 4. Wallac e EL, Go ker-Alpan O, Wilcox WR, et al. Head-to-head trial of pegunigalsidase alfa vers us agals id as e b eta in p atients with Fab ry d is eas e and d eterio rating renal f unc tio n: res ults f ro m the 2-y ear rand o mis ed p has e III BALANCE study. JMed Genet . 2024;61(6):520-530. Pub lis hed 2024 May 21. d o i:10.1136/jmg-2023-109445 5. Laney DA, Bennett RL, Clark e V, et al. Fab ry d is eas e p rac tic e g uid elines : rec o mmend atio ns o f the Natio nal So c iety o f Genetic Co uns elo rs . JGenet Couns . 2013;22(5):555-564. d o i:10.1007/s 10897-013-9613-3 6. Ho p k in RJ , Jef f eries JL, Laney DA, et al. The manag ement and treatment o f c hild ren with Fab ry d is eas e: A United States-based p ers p ec tiv e. Mol Genet Metab. 2016;117(2):104-113. d o i:10.1016/j.y mg me.2015.10.007 7. Ortiz A, Germain DP, Desnick RJ , et al. Fabry disease rev isited: Manag ement and treatment rec o mmend atio ns f o r ad ult p atients . Mol Genet Metab. 2018;123(4):416-427. d o i:10.1016/j.y mg me.2018.02.014 8. Germain DP, Fouilhoux A, Decramer S, et al. Co ns ens us rec o mmend atio ns f o r d iag no s is , manag ement and treatment o f Fab ry d is eas e in p aed iatric p atients . Clin Genet . 2019;96(2):107-117. d o i:10.1111/c g e.13546 9. Elf ab rio . [p res c rib ing inf o rmatio n]. Chies i USA, Inc .; 2024 . 10. Sc hiffmann R, Goker-Alpan O, Holida M, et al. Peg unig als id as e alf a, a no v el PEGy lated enzy me rep lac ement therap y f or Fabry d is ease, p rovides s ustained plasma conc entrations and favorable pharmacodynamics : A 1-y ear Phas e 1/2 c linic al trial. JInherit Metab Dis . 2019;42(3):534-544. d o i:10.1002/jimd .12080 11. Germain DP, Altares cu G, Barriales-Villa R, et al. An ex pert consens us on p ractical c linical rec ommendations and g uid anc e f o r p atients with c las s ic Fab ry d is eas e. Mol Genet Metab. 2022;137(1-2):49-61. d o i:10.1016/j.y mg me.2022.07.010 12. Bieg s traaten M, Arng rms s o n R, Barb ey F, et al. Rec o mmend atio ns f o r initiatio n and c es s atio n o f enzy me rep lac ement therapy in p atients with Fabry disease: the European Fabry Work ing Gro up c o ns ens us d o c ument. Orphanet JRare Dis . 2015;10:36. Pub lis hed 2015 Mar 27. d o i:10.1186/s 13023-015-0253-6 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 0 4/30/2025
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Enzyme Replacement Therapy (ERT ) forGaucher Disease : Cerezyme (imiglucerase), Elelyso (taliglucerase alfa), Vpriv ( velaglucerase alfa ) BENEF IT TYPE Medical ST AT US Prior Authorization Required Gaucher disease is a rare, inherited, lysosomal storage disorder. In Gaucher disease, mutations of t he GBA gene cause def iciency of the enzyme glucocerebrosidase ( ac id beta-glucosidase) , resulting in the accumulation of glucocerebroside ( glucosylceramide [GLC ]) in the lysosomes of macrophages to f orm Gaucher cells, especially in the bone marrow, spleen, and liver. Prominent symptoms include hepatosplenomegaly, anemia, thrombocytopenia, and skeletal problems (e.g., bone pain, osteopenia, osteonecrosis, f racture, def ormity) . Type 1 Gaucher disease is the most common f orm and does not af f ect the central nervous system. Type 2 and 3 Gaucher disease are characterized by the presence of primary neurologic disease. Type 2 has an onset bef ore age two years and is rapidly progressive with death by age two to f our years. Individuals with type 3 of ten have a more slowly progressive course. Available treatments are indicated f or Type 1 Gaucher disease and include enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). Individuals with type 2 Gaucher disease are not likely to respond to ERT or SRT. This policy f ocuses on ERT . Cerezyme was the f irst ERT product approved by the FDA f or Gaucher disease, approved in 1994. Notably, Gaucher disease was the f irst lysosomal storage disorder f or which an ef f ective ERT was developed.Enzyme replacement therapy for Gaucher disease will be considered for coverage when the following criteria are met:Gaucher DiseaseFor initial authorization: 1. Member meets the labeled age requirement: a) Cerezyme: At least 2 years of age b) Elelyso: At least 4 years of age c) Vpriv : At least 4 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 or Type 3 confirmed by documentation of at le as t one of the f ollowing: a) Reduced activity of glucocerebrosidase via enzyme assay (0 to 15% of normal) , and/or b) Molecular genetic test documenting 2 mutations (biallelic variants) of the GBA gene; AND 4. Member has at least one of the f ollowing as a result of Gaucher disease: a) Anemia b) Thrombocytopenia c) Bone symptoms d) Enlarged spleen or liver; AND 5. Member does NO Th av e an y of the f ollowing: IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023a) Type 2 Gaucher disease,b) Severe or rapidly progressing neurological complications , c) Concomitant use of miglustat or eliglustat . 6. Dosage allowed/Quantity limit: Type 1 Gaucher disease: Up to 60 units/kg every other week IV inf usion Type 3 Gaucher disease: Based on clinical literature and physician expertise. NOTE: Treatment of Type 3 Gaucher disease is of f label. If all the above requirements are met , the medication will be approved for 12 months . For reauthorization :1. Chart notes must show improvement f rom baseline in at least one of the f ollowing signs or symptoms:a) Hemoglobin level b) Platelet count c) Reduced liver and/or spleen volume(s) d) Skeletal manif estations (e.g., less bone pain, f ewer bone crises, etc.) If all the above requirements are met , the medication will be approved for an additional 12 months. CareSource considers Cerezyme (imiglucerase), Elelyso (taliglucerase alfa), Vpriv (velaglucerase alfa) 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/DESCRIPTION08/02/2021 New policy f or ERT f or Gaucher disease created. 05/17/2023 Updated and added ref erences. Clarif ied that the gene mutation should be biallelic. 04/29/2025 Updated ref erences. Ref erenc es : 1. Cerezy me [p ac k ag e ins ert]. Camb rid g e, MA: Genzy me Co rp o ratio n; 2024. 2. Elely s o [p ac k ag e ins ert]. NY, NY: Pf izer Inc .; 2025. 3. Vp riv [p ac k ag e ins ert]. Lex ing to n, MA: Shire Human Genetic Therap ies , Inc .; 2024. 4. Shemes h E, Dero ma L, Bemb i B, et al. Enzy me rep lac ement and s ub s trate red uc tio n therap y f o r Gauc her d is eas e. Coc hrane Databas e Sy s t Rev . 2015;(3):CD 010324. Pub lis hed 2015 Mar 27. d o i:10.1002/14651858.CD 010324.p ub 2 5. Wang RY, Bo damer OA, Watson MS, Wilcox WR; ACMG Work Gro up on Diagnostic Confirmation o f Ly s o s o mal Sto rag e Diseases . Ly sosomal s torage diseases: diagnostic c o nf irmatio n and manag ement o f p res y mp to matic ind ivid uals. Genet Med. 2011;13(5):457-484. d o i:10.1097/GIM.0b 013e318211a7e1 6. El-Bes hlawy A, Ty lki-Szymanska A, Vellodi A, et al. Lo ng-term hematological, vis c eral, and g ro wth o utc o mes in c hild ren with Gauc her disease ty pe 3 treated with imigluceras e in the International Collab o rativ e Gauc her Gro up Gauc her Reg is try . Mol Genet Metab. 2017;120(1-2):47-56. d o i:10.1016/j.y mg me.2016.12.001 7. Mistry PK, Cappellini MD, Lukina E, et al. A reappraisal of Gaucher d isease-diagnosis and d is eas e manag ement alg o rithms . Am JHematol . 2011;86(1):110-115. d o i:10.1002/ajh.21888 8. Stirnemann J , Belmato ug N, Camo u F, et al. A Rev iew o f Gauc her Dis eas e Patho p hy s io lo g y , Clinic al Pres entation and Treatments. Int JMol Sci . 2017;18(2):441. Published 2017 Feb 17. d o i:10.3390/ijms 18020441 9. Dard is A, Michelakakis H, Ro zenfeld P, et al. Patient c entered guidelines for the laboratory d iagnos is o f Gauc her d is eas e type 1. Orphanet JRare Dis . 2022;17(1):442. Published 2022 Dec 21. d o i:10.1186/s 13023-022-02573-6 10. Bieg s traaten M, Co x TM, Belmato ug N, et al. Manag ement g o als f o r ty p e 1 Gauc her d is eas e: An ex p ert c o ns ens us document from the European wo rking group o n Gauc her diseas e. Blood Cells Mol Dis . 2018;68:203-208. d o i:10.1016/j.b c md .2016.10.008IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/202311. Hug hes DA, Pas to res GM. Gauc her Dis eas e. 2000 Jul 27 [Up d ated 2023 Dec 7]. In: Ad am MP, Feld man J , Mirzaa GM, et al., ed itors . GeneReviews [Internet]. Seattle (WA): Univers ity of Washington, Seattle; 1993-2025. Available f rom: https://www.ncbi.nlm.nih.gov /b o o k s /NBK1269/ Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 0 4/29/2025
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Eculizumab (Soliris, Epysqli, Bkemv)BENEF IT TYPE Medical ST AT US Prior Authorization Required Soliris is a C5 Complement inhibitor initially approved by the FDA in 2007. It is approved f or the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis , atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy , generalized myasthenia gravis (gMG) in adult and pediatric patients six years of age and older who are anti-acetylcholine receptor (AchR) antibody positive , and neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. PNH is a rare hematopoietic stem cell disorder in which activation of the complement system destroys red blood cells . aHUS is a type of t hrombotic microangiopathy (TMA) , a group of syndromes def ined by the presence of hemolytic anemia, low platelets and organ damage due to microscopic blood clots in the capillaries . Unlike typical HUS, aHUS is usually genetic . The three main signs of aHUS are hemolytic anemia, thrombocytopenia, and acute kidney f ailure. Of note, the other type of TMA is called thrombotic thrombocytopenic purpura (TTP); Soliris is not used to treat TTP. Epysqli and Bkemv are biosimilars of Soliris.Eculizumab (Soliris, Epysqli, Bkemv) will be considered for coverage when the following criteria are met:Paroxysmal N octurnal H emoglobinuria (PNH)For initial authorization: 1. Member is at le as t 18 years of age; AND 2. Medication is prescribed by or in consultation with a hematologist; AND 3. Member has a diagnosis of PNH as conf irmed by f low cytometry; AND 4. Member has a lactate dehydrogenase (LDH) level >1.5x upper limit of normal (ULN); AND 5. Member has at least one PNH-related sign/symptom e.g., f atigue, hemoglobin 10% (to rule out TTP); AND 5. Member has tried and f ailed or is unable to try Ultomiris or Epysqli, and Bkemv ; AND 6. Member has received meningococcal vaccine. 7. Dosage allowed/Quantity limit: Pediatrics: See weight-based dosing in package insert. Adults: 900mg IV weekly x 4 weeks, then 1200mg 1 week later, then 1200mg every 2 weeks thereaf ter. If all the above requirements are met , the medication will be approved for 6 months . For reauthorization :1. Chart notes must demonstrate hematologic normalization as evidenced by increased platelet count or LDH maintained below upper limit of normal; AND 2. Improved or preserved kidney f unction. If all the above requirements are met , the medication will be approved for an additional 12 months . Generalized Myasthenia Gravis (gMG)For initial authorization: 1. Member is at le as t 6 years of age; AND 2. Medication is prescribed by or in consultation with a neurologist; AND 3. Member has a documented diagnosis of MGFA class II-IV myasthenia gravis (see Appendix); AND 4. Lab result in chart notes shows the member is seropositive f or AChR antibodies ; AND 5. Member has tried and f ailed at le as t 1 conventional therapy: a) Pyridostigmine b) Corticosteroid f or at least 4 weeks c) Non-steroid immunosuppressant (e.g., azathioprine) f or at least 6 months; AND 6. If an adult, m ember has tried and f ailed or is unable to try Ultomiris (requires trial of IV Vyvgart) or Epysqli, and Bkemv ; AND 7. Member has received meningococcal vaccine. 8. Dosage allowed/Quantity limit: Pediatrics: See weight-based dosing in package insert . Adults: 900 mg IV weekly f or the f irst 4 weeks, f ollowed by 1200 mg f or the f if th dose 1 week later, then 1200 mg every 2 weeks thereaf ter. If all the above requirements are met , the medication will be approved for 6 months . IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023For reauthorization :1. Chart notes must demonstrate improvement in activities of daily living, muscle strength, and/or health related quality of lif e; f ewer exacerbations or hospitalizations, or reduced use of rescue medication. If all the above requirements are met , the medication will be approved for an additional 12 months . N euromyelitis O ptica Spectrum D isorder (NMOSD)For 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 documented diagnosis of NMOSD and is seropositive f or aquaporin-4 (AQP4) IgG antibodies ; AND 4. Member had had 1 or more relapses within the past year; AND 5. Member has tried and f ailed r ituximab f or at least 6 months (requires prior aut h); AND 6. Member has tried and f ailed or is unable to try Ultomiris ; AND 7. Member has received meningococcal vaccine. 8. Dosage allowed/Quantity limit: 900 mg IV weekly f or the f irst 4 weeks, f ollowed by 1200 mg f or the f if th dose 1 week later, then 1200 mg every 2 weeks thereaf ter. If all the above requirements are met , the medication will be approved for 6 months . For reauthorization : 1. Chart notes must document disease stabilization, symptom improvement, and/or reduced f requency of relapses . If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Eculizumab (Soliris, Epysqli, Bkemv) 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/DESCRIPTION11/14/2017 New policy f or Soliris created. 10/26/2019 New diagnosis of Neuromyelitis optica spectrum disorder (NMOSD) added . 10/15/2020 Revised criteria f or NMOSD to align with other products. Only require at least 1 relapse in past year. Added trial of a standard therapy. Added trial of Enspryng. Reworded the criteria f or meningitis vaccine. Removed the part about stable immunosuppressive therapy (just assessed for study purpose). Removed restrictions on prior Rituxan, mitoxantrone, IVIG (only applicable to the study design). Changed initial auth duration to 6 months. Edited the renewal criteria to be more appropriate. Also corrected the d ose inf ormation error. Changed to nonpref erred drug status. 02/08/2021 gMG: Updated ref erences. Added specialist requirement. Removed MG-ADL score. Amended prerequisite drugs to more closely match guidelines and literature. Removed clinical trial exclusion criteria. Reduced initial auth duration to 6 months. Revised renewal c riteria 06/02/2021 aHUS: Updated ref erences. Added specialist requirement. Revised diagnostic parameters. Removed list of restrictions f rom clinical trials. Stated Ultomiris as pref erred. Amended dosing inf ormation. Revised renewal criteria. IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PNH: Updated ref erences. Added age limit. Removed nephrology as specialist. Removed transf usion and organ damage requirements. Pref erence f or Ultomiris. Amended dosing inf ormation. Reduced initial auth duration f rom 12 months to 6 months. Revised renewal c riteria.07/25/2023 Transf erred to new template. PNH: Updated ref erences. Added that they must be symptomatic . aHUS: Updated and added ref erences. Corrected ADAMTS13 level cutof f . Changed evidence of hemolysis to evidence of MAHA . NMOSD: Added ref erences. Removed requirement f or trial of Enspryng. MG: Added ref erence. Removed severe, ref ractory and added MGFA class II-IV. Added MGFA appendix. Added trial of Ultomiris. Shortened and simplif ied list of conventional therapy trials. 04/10/2024 NMOSD: Added trial of Ultomiris. Removed azathioprine, mycophenolate trial options. 04/07/2025 MG: Age limit changed f rom at least 18 to at least 6 years per label update; update d dosing inf o, added reference, added that Ultomiris/Vyvgart trial only applies to adults. Changed steroid trial duration f rom 3 months to 4 weeks (Sanders, Alhaidar). 06/25/2025 Edited policy title to include biosimilars. Added biosimilar step edits. APPENDIX: Ref erenc es : 1. 2021 Geo rg ia Co de Title 33 Ins uranc e Chap ter 20A-Managed Health Care Plans A rt ic le 2-Patient’ s Rig ht to Ind ep end ent Rev iew 33-20A-31 Def initio ns . Jus tia US Law. Ac c es s ed Ap ril 25, 2023. https://law.justia.com/codes/georgia/2021/title-33/c hap ter-20a/artic le-2/s ec tio n-33-20a-31/.2. So liris [p res c rib ing inf o rmatio n]. Bo s to n, MA: Alex io n Pharmac eutic als Inc ; 2025. 3. Epysqli [prescribing information]. Sams ung Bio ep is Co ., Ltd . ; 2025. 4. Bk emv [p res c rib ing inf o rmatio n]. Amg en Inc .; 2025. 5. Hillmen P, Yo ung NS, Sc hub ert J , et. al. The c o mp lement inhib ito r ec ulizumab in p aro x y s mal no c turnal hemo g lo b inuria. NEng JMed. 2006;355:1233-1243. Do i: 10.1056/NEJ MMo a061648. 6. Bro d s ky RA, Young NS, Antonioli E, et. al. Multicenter phase 3 s tudy of the complement inhib ito r ec ulizumab f o r the treatment o f p atients with p aro x y s mal no c turnal hemo g lo b ulinemia. Blood. 2008;111:1840-1847. Do i: 10.1182/b lo o d-2007-06-094136. 7. Sahin F, Ak ay OM, Ayer M, et al. Pesg PNH d iag no s is , f o llo w-up and treatment g uid elines . Am JBlood Res . 2016;6(2):19-27. Pub lis hed 2016 Aug 5. 8. Park er CJ . Up date on the diagnosis and management of parox ysmal no cturnal hemoglo b inuria. Hematology Am Soc Hematol Educ Program . 2016;2016(1):208-216. d o i:10.1182/as hed uc atio n-2016.1.208 9. Dev o s T, Meers S, Boeck x N, et al. Diagnosis and management of PNH: Rev iew and rec o mmend atio ns f ro m a Belg ian ex p ert p anel. Eur JHaematol . 2018;101(6):737-749. d o i:10.1111/ejh.13166 10. Patriq uin CJ , Kiss T, Cap lan S, et al. Ho w we treat paroxy smal noc turnal hemoglobinuria: A c onsensus s tatement o f the Canad ian PNH Netwo rk and rev iew o f the natio nal reg is try . Eur JHaematol . 2019;102(1):36-52. d o i:10.1111/ejh.13176 11. Bo d I, Amine I, Bo b an A, et al. Co mp lement Inhib itio n in Paro x y s mal No c turnal Hemo g lo b inuria (PNH): A Sy s tematic Rev iew and Ex p ert Op inio n f ro m Central Euro p e o n Sp ec ial Patient Po p ulatio ns . Adv Ther . 2023;40(6):2752-2772. d o i:10.1007/s 12325-023-02510-4 12. Leg end re CM, Lic ht C, Muus P, et. al. Terminal c o mp lement inhib ito r ec ulizumab in aty p ic al hemo ly tic-uremic s y nd ro me. NEng JMed. 2013;368:2169-2181. Do i: 10.1056/NEJ MMo a1208981. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/202313. Kato H, Nang aku M, Hatay a H, et al. Clinical guides for atypical hemo ly tic uremic s y nd ro me in Jap an. Clin Ex p Nephrol . 2016;20(4):536-543. d o i:10.1007/s 10157-016-1276-6 14. Lo irat C, Fak ho uri F, Aric eta G, et al. An internatio nal c o ns ens us ap p ro ac h to the manag ement o f aty p ic al hemo ly tic uremic s y nd ro me in c hild ren. Pediatr Nephrol . 2016;31(1):15-39. d o i:10.1007/s 00467-015-3076-8 15. Pug h D , O' Sulliv an ED , Duthie FA, Mas s o n P, Kav anag h D . Interv entio ns f o r aty p ic al haemo ly tic uraemic s y nd ro me. Coc hrane Databas e Sy s t Rev . 2021;3(3):CD 012862. Pub lis hed 2021 Mar 23. d o i:10.1002/14651858.CD 012862.p ub 2 16. Ts eng MH, Lin SH, Ts ai JD, et al. Atypical hemolytic uremic s yndrome: Consens us of diagnosis and treatment in Taiwan. JFormos Med As s oc . 2023;122(5):366-375. d o i:10.1016/j.jf ma.2022.10.006 17. Lee H, Kang E, Kang HG, et al. Consens us regarding diagnosis and manag ement o f aty p ic al hemo ly tic uremic s y nd ro me. Korean JIntern Med. 2020;35(1):25-40. d o i:10.3904/k jim.2019.388 18. Sc ully M, Go o d s hip T. Ho w I treat thro mb o tic thro mb o c y to p enic p urp ura and aty p ic al haemo ly tic uraemic s y nd ro me. Br JHaematol . 2014;164(6):759-766. d o i:10.1111/b jh.12718 19. Ho ward Jr, James F., et al. "Safety and efficac y of ec ulizumab in anti-ac ety lc ho line rec ep to r antib o d y-p o sitive ref rac tory generalised myasthenia g ravis (REGAIN): a p hase 3, rand omis ed , d o ub le-blind, placebo-controlled, multic entre s tud y ." The Lanc et Neurology 16.12 (2017): 976-986. 20. Dhillo n, So hita. "Ec ulizumab : A Rev iew in Generalized My as thenia Grav is ." Drugs 78.3 (2018): 367-376. 21. Sand ers DB, Wolfe GI, Benatar M, et al. International c onsensus g uidance for management of myasthenia g ravis: Ex ec utiv e s ummary . Neurology . 2016;87(4):419-425. d o i:10.1212/WNL.0000000000002790 22. Naray anas wami P, Sand ers DB, Wo lf e G, et al. Internatio nal Co ns ens us Guid anc e f o r Manag ement o f My as thenia Grav is : 2020 Up d ate. Neurology . 2021;96(3):114-122. d o i:10.1212/WNL.0000000000011124 23. Alhaid ar MK, Ab umurad S, So liv en B, Rezania K. Current Treatment o f My as thenia Grav is . JClin Med. 2022;11(6):1597. Pub lis hed 2022 Mar 14. d o i:10.3390/jc m11061597 24. O' Co nnell K, Ramd as S, Palac e J . Manag ement o f Juv enile My as thenia Grav is . Front Neurol . 2020;11:743. Pub lis hed 2020 Jul 24. d o i:10.3389/f neur.2020.00743 25. Weins henk er B. Neuro myelitis Op tic a Sp ec trum Dis o rd er. NORD (Natio nal Org anizatio n f o r Rare Dis o rd ers ). http s ://raredis eases.org/rare-diseas es/neuromyelitis-optica/. Pub lis hed Aug us t 25, 2020. Ac c es s ed Oc to b er 2, 2020. 26. Kes s ler RA, Mealy MA, Levy M. Treatment of Neuro myelitis Optica Sp ec trum Dis o rd er: Ac ute, Prev entiv e, and Symptomatic. Curr Treat Options Neurol . 2016;18(1):2. d o i:10.1007/s 11940-015-0387-9 27. Mealy MA, Wingerchuk DM, Palac e J, Greenberg BM, Levy M. Comparison of relapse and treatment failure rates amo ng p atients with neuro myelitis o ptica: multic enter s tudy of treatment efficac y . JAMA Neurol. 2014;71(3):324-330. d o i:10.1001/jamaneuro l.2013.5699 28. Pitto ck SJ , Berthele A, Fujihara K, et al. Ec ulizumab in Aq uap o rin-4-Po s itiv e Neuro my elitis Op tic a Sp ec trum Dis o rd er. NEngl JMed. 2019;381(7):614-625. d o i:10.1056/NEJ Mo a1900866 29. Pard o S, Giovannoni G, Hawk es C, Lec hner-Scott J , Waub ant E, Lev y M. Editorial on: Eculizumab in aq uaporin4-p o s itiv e neuro my elitis o p tic a s p ec trum d is o rd er. Mult Sc ler Relat Dis ord. 2019;33:A1-A2. d o i:10.1016/j.ms ard .2019.07.001 30. Framp to n JE. Eculizumab: A Rev iew in Neuro myelitis Optica Spectrum Disorder [published c orrection ap p ears in Drug s . 2020 Ap r 21;:] [p ub lis hed c o rrec tio n ap p ears in Drug s . 2020 Ap r 22;:]. Drugs . 2020;80(7):719-727. d o i:10.1007/s 40265-020-01297-w 31. Pitto ck SJ , Fujihara K, Palac e J, et al. Eculizumab monotherapy for NMOSD: Data from PREVENT and its o p en-lab el ex tens io n. Mult Sc ler . 2022;28(3):480-486. d o i:10.1177/13524585211038291 32. Aung s umart S, Youngkong S, Dejthev ap o rn C, et al. Ef f ic ac y and s af ety o f mo no c lo nal antib o d y therap y in p atients with neuro my elitis o p tic a s p ec trum d is o rd er: As y s tematic rev iew and netwo rk meta-analy s is . Front Neurol . 2023;14:1166490. Pub lis hed 2023 Ap r 4. d o i:10.3389/f neur.2023.1166490 33. Wing erc huk DM, Zhang I, Kielhorn A, et al. Network Meta-analy sis of Fo o d and Drug Ad minis tratio n-approved Treatment Op tio ns f o r Ad ults with Aq uap o rin-4 Immuno g lo b ulin G-p o s itiv e Neuro my elitis Op tic a Sp ec trum Dis o rd er. Neurol Ther . 2022;11(1):123-135. d o i:10.1007/s 40120-021-00295-8 Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 04/ 07/2025
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Ctexli (chenodiol)BENEF IT TYPE Ph ar mac y ST AT US Prior Authorization Required Ctexli, approved by the FDA in 2025, is a bile acid indicated f or treatment of adults with cerebrotendinous xanthomatosis (CTX) , a neurodegenerative lipid storage disorder and bile ac id synthesis disorder . CTX is caused by pathogenic variants in the CYP27A1 gene which lead to def iciency of the enzyme s t e r ol 27-hydroxylase , preventing cholesterol f rom converting to bile acids, particularly depleting chenodeoxycholic ac id ( chenodiol, CDCA). This causes abnormal deposition of cholesterol and cholestanol in various tissues, and increased excretion of bile alcohols in urine. Symptoms are variable and age-dependent and include diarrhea, cataracts, xanthomas, and neurologic dysf unction. Ctexli replaces def icient levels of the endogenous CDCA bile acid. This results in reductions of plasma cholestanol and urine 23S-pentol concentrations to slow CTX progression. Exogenous CDCA is the f irst-line treatment and is given lif elong. Chenodiol was originally approved as brand name Chenodal in 2009 f or the treatment of g all bladder stones and has been used of f-label f or CTX.Ctexli (chenodiol) will be considered for coverage when the following criteria are met:C erebrotendinous Xanthomatosis (CT X)For initial authorization: 1. Member is at le as t 18 years of age; AND 2. Medication must be prescribed by or in consultation with a neurologist or metabolic specialist ; AND 3. Member has a documented diagnosis of CTX confirmed by g enetic test results that show mutation of the CYP27A1 gene; AND 4. Biochemical test results show elevated plasma cholestanol at baseline; AND 5. Baseline liver transaminase and total bilirubin levels have been or will be obtained bef ore starting. 6. Dosage allowed/Quantity limit: 250 mg orally three times daily . QL: 90 tablets per 30 days If all the above requirements are met , the medication will be approved for 3 months . For reauthorization : 1. Chart notes must show positive clinical response to therapy such as reduced plasma cholestanol or urine 23S-pentol levels, or symptom improvement f rom baseline. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Ctexli (chenodiol) 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/DESCRIPTION03/17/2025 New policy f or Ctexli created. Ref erenc es : 1. Ctex li [p res c rib ing inf o rmatio n]. Mirum Pharmac eutic als , Inc .; 2025. 2. Stelten BML, Do tti MT, Verrip s A, et al. Ex p ert o p inio n o n d iag no s ing , treating and manag ing p atients with c ereb ro tendinous xanthomatos is (CTX): a modified Delphi s tudy. Orphanet JRare Dis . 2021;16(1):353. Published 2021 Aug 6. d o i:10.1186/s 13023-021-01980-5 3. Fed eric o A, Gallus GN. Cerebrotendinous Xantho mato s is . 2003 Jul 16 [Up d ated 2024 No v 14]. In: Ad am MP, Feld man J , Mirzaa GM, et al., editors. GeneRev iews [Internet]. Seattle (WA): Univers ity of Was hington, Seattle; 1993-2025. Av ailab le f ro m: https://www.ncbi.nlm.nih.gov/books/NBK1409/4. Patni N, Wils o n DP. Cereb ro tend ino us Xantho mato s is . [Up d ated 2023 Mar 8]. In: Feing o ld KR, Ahmed SF, Anawalt B, et al., editors . Endotex t [Internet]. South Dartmouth (MA): MD Tex t.c o m, Inc .; 2000- . Av ailab le f ro m: http s ://www.nc b i.nlm.nih.g o v /b o o k s /NBK395578/ 5. Cars o n BE, De Jesus O. Cereb rotend ino us Xantho mato s is . [Up d ated 2023 Aug 23]. In: StatPearls [Internet]. Treas ure Is land (FL): StatPearls Pub lis hing ; 2025 Jan- . Av ailab le f ro m: http s ://www.nc b i.nlm.nih.g o v /b o o k s /NBK564330/Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 03/17/2025
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Cerdelga (eliglustat)BENEF IT TYPE Ph ar mac y ST AT US Prior Authorization Required Cerdelga is a substrate reduction therapy, FDA approved in 2014 f or 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 def iciency of the enzyme glucocerebrosidase (acid beta-glucosidase), resulting in the accumulation of glucocerebroside ( glucosylceramide [GLC]) in the lysosomes of macrophages to f orm 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 m ost common f o r m and does not af f ect 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 conf irmed by documentation of at least one of the f ollowing: a) Def iciency of glucocerebrosidase activity by enzyme assa y (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 f ollowing as a result of Gaucher disease: a) Anemia , b) Thrombocytopenia , c) Spleen and/or liver enlargement; AND 6. Member does NOT have an y of the f ollowing: 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 inf ormation f rom the manuf acturer. 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 f ollowing 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 f or Cerdelga created.05/11/2023 Added ref erences. Clarif ied that 2 mutations of the GBA gene should be present. Removed bone outcomes f rom reauth section; was not measured in clinical trials and unlikely to be evident at 12 mo.04/29/2025 Updated ref erences. Ref erenc es : 1. Cerd elg a [p ac k ag e ins ert]. Waterf o rd , Ireland : Genzy me Ireland , Ltd .; 2024 . 2. Mis try PK, Luk ina E, Ben Turk ia H, et al. Ef f ec t o f o ral elig lus tat o n s p leno meg aly in p atients with Gauc her d is eas e type 1: the ENGAGE randomized clinical trial. JA MA . 2015;313(7):695-706. d o i:10.1001/jama.2015.459 3. Luk ina E, Watman N, Arreguin EA, et al. A phase 2 s tudy of elig lus tat tartrate (Genz-112638), an o ral s ub s trate red uc tion therapy for Gauc her dis eas e type 1. Blood. 2010;116(6):893-899. d o i:10.1182/b lo o d-2010-03-273151 4. Luk ina E, Watman N, Dragosky M, et al. Outcomes after 8 y ears of eliglustat therapy for Gauc her diseas e typ e 1: Final res ults f ro m the Phas e 2 trial. Am JHematol . 2019;94(1):29-38. d o i:10.1002/ajh.25300 5. Co x TM, Drelic hman G, Cravo R, et al. Eliglustat maintains lo ng-term c linic al s tab ility in p atients with Gauc her d is eas e ty p e 1 s tab ilized o n enzy me therap y . Blood. 2017;129(17):2375-2383. d o i:10.1182/b lo o d-2016-12-758409 6. Belmato ug N, Di Roc co M, Fraga C, et al. Management and monitoring recommendations for the us e of eliglus tat in ad ults with type 1 Gauc her d is ease in Europe. Eur JIntern Med. 2017;37:25-32. d oi:10.1016/j.ejim.2016.07.011 7. Balwani M, Burro w TA, Charro w J , et al. Recommendations for the us e of eliglus tat in the treatment of adults with Gauc her d is eas e ty p e 1 in the United States . Mol Genet Metab. 2016;117(2):95-103. d o i:10.1016/j.y mg me.2015.09.002 8. Dard is A, Michelakakis H, Ro zenfeld P, et al. Patient c entered guidelines for the laboratory d iagnos is o f Gauc her d is eas e type 1. Orphanet JRare Dis . 2022;17(1):442. Published 2022 Dec 21. d o i:10.1186/s 13023-022-02573-6 9. To rralb a-Cab eza M, Mo rad o-Arias M, Pijierro-Amad o r A, Fernnd ez-Canal MC, Villarrub ia-Espinosa J. Rec o mmendations for o ral treatment for adult patients with ty pe 1 Gauc her d is eas e [p ub lis hed o nline ahead o f p rint, 2022 Jun 5]. Rev Clin Es p (Barc ). 2022;S2254-8874(22)00043-1. d o i:10.1016/j.rc eng .2022.02.008IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/202310. Sto ne WL, Basit H, Muk kamalla SKR, et al. Gaucher Disease. [Upd ated 2023 No v 12]. In: StatPearls [Internet]. Treas ure Is land (FL): StatPearls Pub lis hing ; 2025 Jan- . Av ailab le f ro m: http s ://www.nc b i.nlm.nih.g o v /b o o k s /NBK448080/ Ef f ec tiv e d ate: 10/01/2025 Rev is ed d ate: 04/29/2025
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