IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Lamzede (velmanase alfa-tycv)BENEFIT TYPE Medical STATUS Prior Authorization Required Lamzede, approved by the FDA in 2023, is recombinant human lysosomal alpha-mannosidase indicated for the treatment of non-central nervous system manifestations of alpha-mannosidosis in adult and pediatric patients. Alpha-mannosidosis (AM) is a rare, progressive lysosomal storage disorder caused by pathogenic variants in the MAN2B1 gene, resulting in accumulation of mannose-rich oligosaccharides. Lamzede is an enzyme replacement therapy (ERT) intended to provide alpha-mannosidase, the enzyme that is deficient in AM. It is the first approved treatment for AM but does not cross the blood brain barrier and therefore it not expected to benefit the neurological aspects of the disease. In a Phase 3 c linical trial, 3-minute stair climbing test (3MSCT), 6-minute walking test (6MWT) and forced vital capacity (FVC) numerically favored the Lamzede group and results were supported by a reduction in serum oligos accharide concentration.Lamzede (velmanase alfa-tycv) will be considered for coverage when the following criteria are met:A lpha-M annosidosisFor initial authorization: 1. Medication must be prescribed by or in consultation with a metabolic or genetics specialist, or other specialist experienced with lysosomal storage disorders; AND 2. Member has a diagnosis of alpha-mannosidosis confirmed by enzyme assay showing alpha-mannosidase activity less than 10% of normal; AND 3. Members disease is mild to moderate, without significant central nervous system (CNS) manifestations; AND 4. Member has NOT had a bone marrow transplant or HSCT. 5. Dosage allowed/Quantity limit: 1 mg/kg (actual body weight) once every week as IV infusion. If all the above requirements are met , the medication will be approved for 12 months. For reauthorization : 1. Chart notes must show improvement or stabilized signs and symptoms of disease demonstrated by at least one of the following: a) Clinically significant reduction of serum oligosaccharide concentration from baseline b) Stable or improved 3MSCT, 6MWT, or FVC 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 Lamzede (velmanase alfa-tycv) 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/DESCRIPTION04/13/2023 New policy for Lamzede created.04/25/2024 Annual review; no updates.References: 1. Lamzede [prescribing information]; Chiesi USA, Inc.; 2023. 2. Borgwardt L, Guffon N, Amraoui Y, et al. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial. JInherit Metab Dis . 2018;41(6):1215-1223. doi:10.1007/s10545-018-0185-0 3. Lund AM, Borgwardt L, Cattaneo F, et al. Comprehensive long-term efficacy and safety of recombinant human alpha-mannosidase (velmanase alfa) treatment in patients with alpha-mannosidosis. JInherit Metab Dis . 2018;41(6):1225-1233. doi:10.1007/s10545-018-0175-2 4. Guffon N, Tylki-Szymanska A, Borgwardt L, et al. Recognition of alpha-mannosidosis in paediatric and adult patients: Presentation of a diagnostic algorithm from an international working group. Mol Genet Metab. 2019;126(4):470-474. doi:10.1016/j.ymgme.2019.01.024 5. Malm D, Nilssen . Alpha-Mannosidosis. 2001 Oct 11 [Updated 2019 Jul 18]. 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/NBK1396/Effective date: 10/01/2024 Revised date: 04/25/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Kanuma (sebelipase alfa)BENEFIT TYPE Medical STATUS Prior Authorization Required Kanuma, approved by the FDA in 2015, is a recombinant human lysosomal acid lipase ( a lysosomal enzyme) indicated for the treatment of Lysosomal Acid Lipase (LAL) deficiency . LAL deficiency is a lysosomal storage disorder caused by a genetic defect of the LIPA gene that results in decreased or absent activity of the LAL enzyme. Deficient LAL enzyme activity results in progressive complications due to the lysosomal accumulation of cholesteryl esters and triglycerides in multiple organs, including the liver, spleen, intestine, and the walls of blood vessels . Accumulation of lipids in the liver can lead to fibrosis and cirrhosis. Lipid accumulation in the intestinal walls can lead to malabsorption and growth failure. Dyslipidemia and accelerated atherosclerosis also occur. Kanuma works as an enzyme replacement therapy (ERT) and catalyzes the lysosomal hydrolysis of cholesteryl esters and triglycerides to break them down. It is the first drug to be approved for LAL-D and the first treatment to target the underlying pathology . Previous management focused on strategies like lipid lowering medications, diet modification, liver transplant , or stem cell transplant. Wolman disease is the early infantile-onset phenotype of LAL-D which is more severe and rapidly progressive, with survival not expected beyond infancy. Kanuma significantly prolongs the life expectancy of these patients. Cholesterol ester storage disease (CESD) has a more variable phenotype with onset during childhood or adulthood. Kanuma has proven benefits for these patients as well.Kanuma (sebelipase alfa) will be considered for coverage when the following criteria are met:Lysosomal Acid Lipase (LAL) DeficiencyFor initial authorization: 1. Member is at least 1 month of age; AND 2. Medication must be prescribed by or in consultation with a hepatologist, cardiologist, metabolic specialist , or geneticist ; AND 3. Member has a diagnosis of LAL deficiency confirmed by at least one of the following methods : a) Deficient LAL enzyme activity (assay) b) Identification of biallelic pathogenic mutations of the LIPA gene (genetic testing); AND 4. Member demonstrates at least one clinical feature of LAL-Dsuch as dyslipidemia, elevated transaminases (ALT, AST), impaired growth or failure to thrive, malabsorption, hepatomegaly, adrenal calcification (in Wolmans disease), or advanced liver disease. 5. Dosage allowed/Quantity limit: Infants with rapidly progressive LAL-Dwithin first 6 months of life: Starting dose of 1 mg/kg once weekly via IV infusion. If clinical response is suboptimal, may increase to 3 mg/kg once weekly, and further to 5 mg/kg once weekly if needed. Pediatric and adult LAL-D: 1 mg/kg IV infusion every other week. If clinical response is suboptimal, may increase to 3 mg/kg every other week. 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 a positive clinical response to treatment such as improved lipid profile (LDL-c, triglycerides ), liver biomarkers (ALT, AST), liver volume, or growth /weight z-scores (if pediatric) . If all the above requirements are met , the medication will be approved for an additional 12 months. CareSource considers Kanuma (sebelipase 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/DESCRIPTION04/11/2018 New policy for Kanuma created. 01/10/2022 Transferred to new template. Updated references. Corrected max dose from 3 mg/kg to 5 mg/kg; added ped/adult dosing section which was missing. Changed prescribed by to by or in consultation with; also removed general specialist, added geneticist . Amended age minimum from 8 months to 1 month. Added tests for diagnostic confirmation. Removed all criteria that listed manifestations from a specific clinical trial and was divided by age; replaced with general list of manifestations. Specified renewal criteria. 04/18/2024 Annual review; no updates. References: 1. Kanuma [package inset]. New Haven, CT: Alexion Pharmaceuticals Inc.; 2021. 2. Hoffman EP, et al. Lysosomal acid lipase deficiency. In: ed. Adam MP, et al. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2018. 2015 Jul 30 [Updated 2016 Sep 1]. 3. Wilson DP, Patni N. Lysosomal Acid Lipase Deficiency. [Updated 2020 Jan 18]. In: Feingold KR, Anawalt B, Boyce A, et al., editors. Endotext [Internet]. South Dartmouth (MA): MDText.com, Inc.; 2000- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK395569/4. Vijay S, Brassier A, Ghosh A, et al. Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies [published correction appears in Orphanet JRare Dis. 2021 Mar 1;16(1):113]. Orphanet JRare Dis . 2021;16(1):13. Published 2021 Jan 6. doi:10.1186/s13023-020-01577-4 5. Malinov V, Balwani M, Sharma R, et al. Sebelipase alfa for lysosomal acid lipase deficiency: 5-year treatment experience from a phase 2 open-label extension study. Liver Int. 2020;40(9):2203-2214. doi:10.1111/liv.14603 6. Burton BK, Feillet F, Furuya KN, Marulkar S, Balwani M. SEBELIPASE ALFA IN CHILDREN AND ADULTS WITH LYSOSOMAL ACID LIPASE DEFICIENCY: FINAL RESULTS OF THE ARISE STUDY [published online ahead of print, 2021 Nov 10]. JHepatol . 2021;S0168-8278(21)02171-1. doi:10.1016/j.jhep.2021.10.026 7. Demaret T, Lacaille F, Wicker C, et al. Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up. Orphanet JRare Dis . 2021;16(1):507. Published 2021 Dec 14. doi:10.1186/s13023-021-02134-3 Effective date: 10/01/2024 Revised date: 04/18/2024
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Bylvay (odevixibat)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Bylvay, approved by the FDA in July 2021, is indicated for the treatment of pruritus in patients 3 months of age and older with progressive familial intrahepatic cholestasis (PFIC). It is the first drug approved for PFIC and is available as oral pellets or capsules, taken once daily. PFIC is an ultra-rare group of genetic disorders that disrupt bile formation in the liver . It usually presents during infancy and is characterized by cholestasis, jaundice, and intense itching. Most patients will eventually require biliary diversion surgery or liver transplant. PFIC types 1, 2, and 3 are the most common and types 1 and 2 are t he most severe. PFIC1 involves extrahepatic manifestations while PFIC2 does not. However, PFIC2 can be complicated by hepatocellular carcinoma. As of June 2023, Bylvay is also approved for the treatment of cholestatic pruritus in patients 12 months of age and older with Alagille Syndrome ( ALGS), a rare genetic disorder that can affect multiple organ systems, most commonly the liver, with a paucity of interlobular ducts . In cholestatic liver disease, biliary substances arent eliminated from the liver , thus they re-enter circulation. Cholestatic itch is thought to be related to the accumulation of bile acids in the skin. Bylvay is a reversible inhibitor of the ileal bile acid transporter (IBAT). Inhibiting IBAT decreases reuptake of bile salts .Bylvay (odevixibat) will be considered for coverage when the following criteria are met:Progressive familial intrahepatic cholestasis (PFIC)For initial authorization: 1. Member is at least 3 months of age; AND 2. Medication must be prescribed by or in consultation with a gastroenterologist or hepatologist; AND 3. Member has a diagnosis of PFIC type 1 or 2 confirmed by genetic testing identifying pathogenic mutations of the ATP8B1 or ABCB11 genes (results must be provided for review); AND 4. Member has significant pruritis not attributed to another cause; AND 5. Documentation of serum bile acid level 100 mol/L; AND 6. Documentation of baseline liver function tests (e.g., ALT, AST, bilirubin, INR ); AND 7. Trial and failure of ursodiol (may also continue concurrently ); AND 8. Member does NOT have any of the following: a) Decompensated c irrhosis b) Varia nts of the ABCB11 gene (PFIC type 2) that code for non-functional or complete absence of the bile salt export pump (BSEP-3 ) protein (per submitted genetic test result) c) Biliary diversion surgery in the past 6 months d) Liver transplant 9. Dosage allowed/Quantity limit: 40 mcg/kg orally once daily. If no improvement after 3 months, may increase in 40 mcg/kg increments up to 120 mcg/kg. Max dose per day 6 mg. 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 improvement of pruritis compared to baseline; AND 2. Chart notes must show reduced serum bile acid from baseline; AND 3. Member has NOT experienced portal hypertension or a hepatic decompensation event (e.g., variceal hemorrhage, ascites, hepatic encephalopathy) while being treated with Bylvay . If all the above requirements are met , the medication will be approved for an additional 12 months . Alagille Syndrome (ALGS)For initial authorization: 1. Member is at least 12 months of age; AND 2. Medication must be prescribed by or in consultation with a gastroenterologist or hepatologist; AND 3. Member has a diagnosis of Alagille syndrome (ALGS) confirmed by the involvement of at least 3 of the following major clinical features : a) Hepatic (e.g., hyperbilirubinemia, cholestasis, xanthomas ) b) Cardiac (e.g., heart murmur, peripheral pulmonic stenosis ) c) Facial (e.g., inverted triangular face) d) Ocular (e.g., embryotoxon, optic disk drusen) e) Skeletal (e.g., butterfly vertebrae) f) Renal (e.g., renal dysplasia, renal tubular acidosis) g) Vascular (e.g., neurovascular accident, moyamoya disease) NOTE: Member also meets criterion if has one or more clinical features and an affected first-degree relative; AND 4. Member must have liver biopsy demonstrating reduced number of the interlobular bile ducts OR confirmed finding of JAG1 or NOTCH2 gene mutation; AND 5. Member has moderate to severe pruritus; AND 6. Members serum bile acid (sBA) level is elevated; AND 7. Member does NOT have any of the following: a) Previous liver transplant b) Decompensated cirrhosis ; AND 8. Member must have a trial and failure of at least 2 of the following: a) Cholestyramine b) Ursodiol c) Rifampin d) Naltrexone; AND 9. Documented rationale why Livmarli cannot be used instead of Bylvay; AND 10. Documentation of baseline liver function tests (e.g., ALT, AST, bilirubin, INR). 11. Dosage allowed/Quantity limit: 120 mcg/kg taken orally once daily . If all the above requirements are met , the medication will be approved for 6 months . For reauthorization : 1. Pruritis has improved in response to therapy with Bylvay; AND 2. Member has NOT experienced portal hypertension or a hepatic decompensation event (e.g., variceal hemorrhage, ascites, hepatic encephalopathy) while being treated with Bylvay 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 Bylvay (odevixibat) 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/DESCRIPTION11/17/2021 New policy for created for Bylvay . 06/27/2023 Added indication for Alagille Syndrome. 08/02/2024 Updated reference. References: 1. Bylvay [prescribing information]. Albireo Pharma, Inc.; 2024. 2. Baumann U, Sturm E, Lacaille F, et al. Effects of odevixibat on pruritus and bile acids in children with cholestatic liver disease: Phase 2 study. Clin Res Hepatol Gastroenterol. 2021;45(5):101751. doi:10.1016/j.clinre.2021.101751 3. Deeks ED. Odevixibat: First Approval [published correction appears in Drugs. 2021 Sep 23;:]. Drugs . 2021;81(15):1781-1786. doi:10.1007/s40265-021-01594-y 4. Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int . 2020;40(8):1812-1822. doi:10.1111/liv.14553 5. Gunaydin M, Bozkurter Cil AT. Progressive familial intrahepatic cholestasis: diagnosis, management, and treatment. Hepat Med. 2018;10:95-104. Published 2018 Sep 10. doi:10.2147/HMER.S137209 6. Siddiqi I, Tadi P. Progressive Familial Intrahepatic Cholestasis. [Updated 2021 Sep 29]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2021 Jan- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK559317/7. Baker A, Kerkar N, Todorova L, Kamath BM, Houwen RHJ. Systematic review of progressive familial intrahepatic cholestasis [published correction appears in Clin Res Hepatol Gastroenterol. 2020 Feb;44(1):115]. Clin Res Hepatol Gastroenterol . 2019;43(1):20-36. doi:10.1016/j.clinre.2018.07.010 8. Srivastava A. Progressive familial intrahepatic cholestasis. JClin Exp Hepatol . 2014;4(1):25-36. doi:10.1016/j.jceh.2013.10.005 9. Ovchinsky N, Aumar M, Baker A, et al. Efficacy and safety of odevixibat in patients with Alagille syndrome (ASSERT): a phase 3, double-blind, randomised, placebo-controlled trial. Lancet Gastroenterol Hepatol . 2024;9(7):632-645. doi:10.1016/S2468-1253(24)00074-8 10. Ayoub MD and Kamath BM. Alagille Syndrome: Diagnostic Challenges and Advances in Management. Diagnostics . 2020; 10(11):907. https://doi.org/10.3390/diagnostics10110907 11. Lin, Henry. Alagille Syndrome. National Organization for Rare Disorders; Updated May 25, 2023 . Accessed June 27, 2023 . https://rarediseases.org/rare-diseases/alagille-syndrome/12. Diaz-Frias J, Kondamudi NP. Alagille Syndrome. [Updated 2022 Aug 14]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2023 Jan- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK507827/13. Fawaz R, Baumann U, Ekong U, et al. Guideline for the Evaluation of Cholestatic Jaundice in Infants: Joint Recommendations of the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition and the European Society for Pediatric Gastroenterology, Hepatology, and Nutrition. JPediatr Gastroenterol Nutr. 2017;64(1):154-168. doi:10.1097/MPG.0000000000001334 14. Spinner NB, Gilbert MA, Loomes KM, et al. Alagille Syndrome. 2000 May 19 [Updated 2019 Dec 12]. 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/NBK1273/15. Muntaha HST, Munir M, Sajid SH, et al. Ileal Bile Acid Transporter Blockers for Cholestatic Liver Disease in Pediatric Patients with Alagille Syndrome: A Systematic Review and Meta-Analysis. JClin Med . 2022;11(24):7526. Published 2022 Dec 19. doi:10.3390/jcm11247526 Effective date: 01/01/2025 Revised date: 08/02/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Brineura (cerliponase alfa)BENEFIT TYPE Medical STATUS Prior Authorization Required Brineura is an enzyme replacement therapy (ERT); specifically, it is a recombinant form of human tripeptidyl peptidase 1 (TPP1), which is deficient in patients with neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Deficiency of TPP1 enzyme activity results in accumulation of waste that it usually metabolizes in cell lysosomes , particularly in the brain. This build up causes cell death and leads to progressive motor function decline, as well as loss of cognitive and visual functions . CLN2 is a rare, inherited , neurodegenerative lysosomal storage disease, and one of the more common forms of Batten disease. It is caused by mutation of the CLN2 gene (which produces the TPP1 lysosomal enzyme and is also known as the TPP1 gene) . Seizures are commonly the initial symptom , typically with late infantile onset . There is rapid progression to dementia, blindness, and loss of ability to walk or talk , with a greatly shortened life expectancy of about 8-12 years. Brineura was approved in 2017 and is indicated to slow the loss of ambulation in CLN2 patients. It works by helping to replace the deficient TPP1 enzyme. Brineura must be administered into the cerebrospinal fluid (CSF) by infusion via a surgically implanted intraventricular access device. In clinical trials, patients treated with Brineura had less decline in motor and language function when compared with historical cohorts. There is no cure for the disease itself.Brineura (cerliponase alfa) will be considered for coverage when the following criteria are met:Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiencyFor initial authorization: 1. Member is at least 3 years of age; AND 2. Brineura is prescribed by or in consultation with a pediatric neurologist or a geneticist; AND 3. Me mber has a diagnosis of late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase 1 (TPP1) deficiency , confirmed by BOTH of the following: a) Demonstration of deficient TPP1 enzyme activity (in leukocytes, fibroblasts, or dried blood spots) b) Identification of causative mutations in each allele of the TPP1/CLN2 gene; AND 4. Member has mild to moderate disease documented by a two-domain score of 3-6 on motor and language domains of the Hamburg Scale, with a score of at least 1 in each of these two domains . 5. Dosage allowed/Quantity limit: 300 mg administered once every other week as an intraventricular infusion followed by infusion of i ntraventricular electrolytes over approximately 4.5 hours. (2 packages per 28 days; each package contains 2 vials of 150 mg/5 mL) If all the above requirements are met , the medication will be approved for 12 months. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023For reauthorization :1. Documentation of slowed loss of ambulation; AND 2. Member does not have an unreversed (sustained) score of 0 on the motor domain of the CLN2 clinical rating scale. If all the above requirements are met , the medication will be approved for an additional 12 months. CareSource considers Brineura (cerliponase 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/DESCRIPTION05/17/2017 New policy for Brineura created. 01/05/2022 Transferred to new template. Updated the Jcode (effective 1/2019). Updated references. Added specialist requirement. Added diagnostic confirmation criteria. Removed redundancy of not having score of 0. Removed exclusions for other neuro illness, ventilation support, status epilepticus. In renewal section, r emoved meeting initial criteria (would not all apply) , reworded slowed loss of ambulation requirement, added that they cant have score of 0 (patient can no longer walk). 04/17/2024 Removed upper age limit. References: 1. Brineura [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; 2020. 2. Mole SE, Schulz A, Badoe E, et al. Guidelines on the diagnosis, clinical assessments, treatment and management for CLN2 disease patients. Orphanet JRare Dis . 2021;16(1):185. Published 2021 Apr 21. doi:10.1186/s13023-021-01813-5 3. Fietz M, AlSayed M, Burke D, et al. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis. Mol Genet Metab. 2016;119(1-2):160-167. doi:10.1016/j.ymgme.2016.07.011 4. Schulz A, Ajayi T, Specchio N, et al. Study of Intraventricular Cerliponase Alfa for CLN2 Disease. NEngl JMed. 2018;378(20):1898-1907. doi:10.1056/NEJMoa1712649 Effective date: 10/01/2024 Revised date: 04/17/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Enspryng (satralizumab-mwge)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Enspryng is an interleukin-6 (IL-6) receptor antagonist indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive. Neuromyelitis optica spectrum disorder (NMOSD) is a rare, autoimmune disease of the central nervous system that primarily attacks the optic nerves and spinal cord leading to blindness and paralysis.Enspryng (satralizumab-mwge) will be considered for coverage when the following criteria are met:Neuromyelitis Optica Spectrum Disorder (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 for aquaporin-4 (AQP4) IgG antibodies ; AND 4. Member has had 1 or more relapses within the past year; AND 5. Member has tried and failed at least one of the following for 6 months or longer: azathioprine, mycophenolate, rituximab (requires prior auth); AND 6. Member has tested negative for hepatitis Band tuberculosis within the past year or there is attestation they will be tested before starting treatment. 7. Dosage allowed/Quantity limit: 120mg subQ at weeks 0, 2, and 4, then 120mg every 4 weeks thereafter . QL: 1 syringe per 28 days (maintenance) 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 frequency of relapses compared to baseline. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Enspryng (satralizumab-mwge) 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/DESCRIPTION10/16/2020 New policy for Enspryng created. 07/14/2023 Transferred to new template. References: 1. 2021 Georgia Code Title 33 Insurance Chapter 20A-Managed Health Care Plans Article 2-Patient's Right to Independent Review 33-20A-31 Definitions . Justia US Law. Accessed April 25, 2023. https://law.justia.com/codes/georgia/2021/title-33/chapter-20a/article-2/section-33-20a-31/.2. Enspryng (satralizumab-mwge) [package insert]. South San Francisco, CA: Genentech, Inc.; 2022 . 3. Kessler RA, Mealy MA, Levy M. Treatment of Neuromyelitis Optica Spectrum Disorder: Acute, Preventive, and Symptomatic . Curr Treat Options Neurol . 2016;18(1):2. doi:10.1007/s11940-015-0387-9 4. Weinshenker B. Neuromyelitis Optica Spectrum Disorder. NORD (National Organization for Rare Disorders). https://rarediseases.org/rare-diseases/neuromyelitis-optica/. Published August 25, 2020. Accessed October 2, 2020. 5. Mealy MA, Wingerchuk DM, Palace J, Greenberg BM, Levy M. Comparison of relapse and treatment failure rates among patients with neuromyelitis optica: multicenter study of treatment efficacy. JAMA Neurol. 2014;71(3):324-330. doi:10.1001/jamaneurol.2013.5699 6. IPD Analytics. Accessed October 2, 2020. 7. Yamamura T, Kleiter I, Fujihara K, et al. Trial of Satralizumab in Neuromyelitis Optica Spectrum Disorder. NEngl JMed. 2019;381(22):2114-2124. doi:10.1056/NEJMoa1901747 8. Traboulsee A, Greenberg BM, Bennett JL, et al. Safety and efficacy of satralizumab monotherapy in neuromyelitis optica spectrum disorder: a randomised, double-blind, multicentre, placebo-controlled phase 3 trial. Lancet Neurol . 2020;19(5):402-412. doi:10.1016/S1474-4422(20)30078-8 Effective date: 01/01/2024 Revised date: 07/14/2023
IN-MED-P-366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Benlysta (belimumab)BENEFIT TYPE Medical (IV) or Pharmacy (subQ) STATUS Prior Authorization Required Benlysta is a B-lymphocyte stimulator (BLyS) -specific inhibitor indicated for the treatment of patients aged 5 years and older with active systemic lupus erythematosus (SLE) who are receiving standard therapy and for patients aged 5 years and older with active lupus nephritis who are receiving standard therapy . Benlysta is not recommended in patients with severe active central nervous system lupus. SLE is the most common type of lupus. It is a chronic autoimmune disease with periods of flares and remissions that causes inflammation and damage throughout the body. LN is a complication of SLE and can progress to end stage renal disease (ESRD). Proteinuria is often the first sign of LN.Benlysta (belimumab) will be considered for coverage when the following criteria are met:Systemic Lupus Erythematosus (SLE)For initial authorization: 1. Member is at least 5 years of age ; AND 2. Medication must be prescribed by or in consultation with a rheumatologist; AND 3. Member has a documented diagnosis of active, autoantibody-positive SLE as confirmed by documentation of at least one of the following: a) A nti-nuclear antibody (ANA) titer 1:80 b) Elevated (above normal) anti-double-stranded DNA (anti-dsDNA) c) Elevated (above normal) anti-Smith (anti-Sm) antibody ; AND 4. Member has tried and failed hydroxychloroquine OR is unable to reduce steroid to an acceptable dose for chronic use ( 5 mg prednisone per day or less) ; AND 5. Standard therapy (e.g., hydroxychloroquine) will be continued unless contraindicated; AND 6. Member does NOT have severe active central nervous system (CNS) lupus . 7. Benlysta will NOT be used with other biologic therapies . 8. Dosage allowed/Quantity limit: IV (Adult or Pediatric): 10mg/kg every 2 weeks for 3 doses and every 4 weeks thereafter SubQ (Adult): 200 mg once weekly SubQ (Pediatric; autoinjector only): 200 mg once weekly if 40 kg or greater; 200 mg every 2 weeks if 15 to
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Adzynma (ADAMTS13, recombinant-krhn)BENEFIT TYPE Medical STATUS Prior Authorization Required Adzynma, approved by the FDA in 2023, is a human recombinant A disintegrin and metalloproteinase with thrombospondin motifs 13 (rADAMTS13) indicated for prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP ). Adzynma is the first treatment approved for cTTP. Hereditary TTP is much less common than ac quired/immune TTP. Endogenous ADAMTS13 regulates the activity of von Willebrand factor (VWF) by cleaving it into smaller units to reduce its platelet binding properties and microthrombi formation. TTP results from a deficiency of ADAMTS13 which leads to increased circulation of VWF causing clotting and red blood cell destruction. Signs and symptoms can include thrombocytopenia, hemolytic anemia, neurological symptoms, and a skin rash. Plasma infusions have been the main treatment for cTTP , to provide sufficient ADAMTS13.Adzynma (ADAMTS13, recombinant-krhn) will be considered for coverage when the following criteria are met:C ongenital Thrombotic Thrombocytopenic Purpura (cTTP)For initial authorization: 1. Medication must be prescribed by or in consultation with a hematologist; AND 2. Member has a diagnosis of cTTP confirmed by BOTH of the following: a) Genetic test results demonstrating ADAMTS13 gene mutation and b) Plasma ADAMTS13 activity level
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Aldurazyme (laronidase)BENEFIT TYPE Medical STATUS Prior Authorization Required Aldurazyme is an enzyme replacement therapy (ERT) that was approved by the FDA in 2003 for the treatment of Mucopolysaccharidosis type I ( MPS I ), including patients with Hurler syndrome, Hurler Scheie syndrome , and Scheie syndrome with moderate to severe symptoms . The risks and benefits of treating mildly affected patients with Scheie syndrome have not been established. It has not been evaluated for effects on central nervous system manifestations. MPS I is a rare genetic lysosomal storage dis ease, with Hurler syndrome being the most severe and most common subtype and Scheie syndrome as the rarest and mildest of the attenuated forms . Pathogenic mutations of the IDUA gene cause the enzyme alpha Liduronidase (IDUA) to be deficient or absent. Normally this lysosomal enzyme breaks down glycosaminoglycans (GAGs) (previously known as mucopolysaccharides) but when it is reduced in MPS I, the GAG substrates heparan sulfate (HS) and dermatan sulfate (DS) accumulate throughout the body leading to widespread cellular, tissue, and organ dysfunction. Aldurazyme provides an exogenous form of the deficient enzyme.Aldurazyme (laronidase) will be considered for coverage when the following criteria are met:Mucopolysaccharidosis I (MPS I)For initial authorization: 1. Medication must be prescribed by or in consultation with a geneticist, metabolic specialist, or pediatrician experienced with managing m ucopolysaccharidoses; AND 2. Member has a documented diagnosis of ONE of the following forms of MPS I: a) Hurler syndrome (severe) , b) Hurler-Scheie syndrome (attenuated), or c) Scheie syndrome with moderate to severe symptoms (attenuated) ; AND 3. Member s clinical diagnosis of MPS I has been confirmed by at least one of the following: a) Low IDUA enzyme activity (less than 10%) , and/or b) Molecular genetic testing identifies pathogenic IDUA gene mutations ; AND 4. Documentation of elevated baseline urinary GAG (uGAG) level . 5. Dosage allowed/Quantity limit: 0.58 mg/kg IV infusion once weekly 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 improved functional capacity (e.g. 6-minute walk test, forced vital capacity (FVC)) compared to baseline, reduced hepatomegaly , and/or reduced uGAG levels. 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 Aldurazyme (laronidase) 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/DESCRIPTION07/26/2021 New policy for Aldurazyme created. 01/02/2024 Removed age limit. Added the terms severe and attenuated. References: 1. Aldurazyme [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; 2023 . 2. Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). JPediatr . 2004;144(5):581-588. doi:10.1016/j.jpeds.2004.01.046 3. Wang RY, Bodamer OA, Watson MS, Wilcox WR; ACMG Work Group on Diagnostic Confirmation of Lysosomal Storage Diseases. Lysosomal storage diseases: diagnostic confirmation and management of presymptomatic individuals. Genet Med. 2011;13(5):457-484. doi:10.1097/GIM.0b013e318211a7e1 4. Jameson E, Jones S, Remmington T. Enzyme replacement therapy with laronidase (Aldurazyme) for treating mucopolysaccharidosis type I. Cochrane Database Syst Rev . 2019;6(6):CD009354. Published 2019 Jun 18. doi:10.1002/14651858.CD009354.pub5 5. Clarke LA. Mucopolysaccharidosis Type I. 2002 Oct 31 [Updated 2021 Feb 25]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2021. Available from: https://www.ncbi.nlm.nih.gov/books/NBK1162/6. Martins AM, Dualibi AP, Norato D, et al. Guidelines for the management of mucopolysaccharidosis type I. JPediatr . 2009;155(4 Suppl):S32-S46. doi:10.1016/j.jpeds.2009.07.005 7. de Ru MH, Boelens JJ, Das AM, et al. Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. Orphanet JRare Dis . 2011;6:55. Published 2011 Aug 10. doi:10.1186/1750-1172-6-55 8. Kubaski F, de Oliveira Poswar F, Michelin-Tirelli K, et al. Mucopolysaccharidosis Type I. Diagnostics (Basel) . 2020;10(3):161. Published 2020 Mar 16. doi:10.3390/diagnostics10030161 Effective date: 07/01/2024 Revised date: 01/02/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Trastuzumab (Herceptin, Herzuma,Kanjinti, Ogivri, Ontruzant, Trazimera) BENEFIT TYPE Medical STATUS Prior Authorization Required Trastuzumab was initially approved by the FDA in 1998 as Hereptin . Since then, the FDA approved Ogivri (2017), Herzuma, (2018), Ontruzant (2019), Kanjinti (2019 ), and Trazimera (2019) as biosimilars to Herceptin. Bevacizumab is approved for use in breast cancer and for metastatic gastric cancer . All oncology treatments, including trastuzumab , must be submitted to Eviti Connect for review via the NantHealth Eviti Connect portal . For additional information and details, please refer to the CareSource policystatement Oncology Treatment Regimen Review. The approval of Herceptin, Herzuma, Ogivri and Kanjinti requires a trial of Ontruzant and Trazimera.DATE ACTION/DESCRIPTION03/28/2024 New policy for trastuzumab products , including biosimilars, created . References: 1. Herceptin. Package insert . Genentech Inc; 2021. 2. Herzuma. Package insert. Celltrion Inc; 2019. 3. Kanjinti. Package insert. Amgen Inc; 2022 . 4. Ogivri. Package insert. Mylan; 2023 . 5. Ontruzant. Package insert. Samsung Bioepis Co Ltd; 2021 . 6. Trazimera. Package insert. Pfizer Inc; 20 20. Effective date: 07 /01/2024 Revised date: 03/28/2024 IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Lupkynis (voclosporin )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Lupkynis is an oral calcineurin inhibitor ( CNI) immunosuppressant that is structurally similar to cyclosporine A . It was approved by the FDA in 2021 for the treatment of adults with active lupus nephritis (LN), in combination with background immunosuppressive therapy. LN is a complication of systemic lupus erythematosus (SLE) and can progress to end stage renal disease (ESRD). Proteinuria is often the first sign of LN , which is evident by an elevated urine protein creatinine ratio (UPCR) . Diagnosis is confirmed by a kidney biopsy, which reveals the classification of disease and is used to guide treatment. Dosing is based on estimated glomerular filtration rate (eGFR), with most patients likely to be on the higher end of the dose range. Hypertension is a common side effec t and blood pressure monitoring is recommended.Lupkynis (voclosporin) will be considered for coverage when the following criteria are met:Lupus Nephritis (LN)For initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with a nephrologist or rheumatologist; AND 3. Member has a documented diagnosis of active lupus nephritis class III, IV, and/or Vas confirmed by kidney biopsy; AND 4. Medication must be prescribed in combination with an immunosuppressant regimen i.e., mycophenolate mofetil (MMF) and corticosteroid; AND 5. Chart notes must document baseline eGFR and UPCR; AND 6. eGFR is at least 45 mL/min/1.73m 2 OR it has been determined that the benefit exceeds the risk; AND 7. Member is not on dialysis and has not had a kidney transplant; AND 8. Lupkynis will not be used in combination with cyclophosphamide; AND 9. Member has tried and failed Benlysta. 10. Dosage allowed/Quantity limit: Starting dose is 23.7 mg (3 capsules) twice daily (total 6 capsules per day; 3 wallets per 30 days). Modify based on eGFR as directed in prescribing information. If all the above requirements are met , the medication will be approved for 6 months. For reauthorization :1. Member has a r educed UPCR (goal is 0.5 mg/mg or less) ; AND 2. eGFR is at least 60mL/min/1.73m 2 OR has 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 Lupkynis (voclosporin) 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/DESCRIPTION03/24/ 20 21 New policy for Lupkynis created. 08/23/2022 Annual review; no changes. 01/10/2024 Updated references. Added Benlysta step. References: 1. Lupkynis. [prescribing information]. Rockville, MD: Aurinia Pharma U.S., Inc; 2021. 2. Rovin BH, Teng YKO, Ginzler EM, et al. Efficacy and safety of voclosporin versus placebo for lupus nephritis (AURORA 1): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial [published correction appears in Lancet. 2021 May 29;397(10289):2048]. Lancet . 2021;397(10289):2070-2080. doi:10.1016/S0140-6736(21)00578-X3. Hahn BH, McMahon MA, Wilkinson A, et al. American College of Rheumatology guidelines for screening, treatment, and management of lupus nephritis. Arthritis Care Res (Hoboken) . 2012;64(6):797-808. doi:10.1002/acr.21664 4. Fanouriakis A, Kostopoulou M, Cheema K, et al. 2019 Update of the Joint European League Against Rheumatism and European Renal Association-European Dialysis and Transplant Association (EULAR/ERA-EDTA) recommendations for the management of lupus nephritis. Ann Rheum Dis . 2020;79(6):713-723. doi:10.1136/annrheumdis-2020-216924 5. Fanouriakis A, Kostopoulou M, Andersen J, et al. EULAR recommendations for the management of systemic lupus erythematosus: 2023 update. Ann Rheum Dis. 2024;83(1):15-29. Published 2024 Jan 2. doi:10.1136/ard-2023-224762 6. Rovin BH, Adler SG, Barratt J, et al. Executive summary of the KDIGO 2021 Guideline for the Management of Glomerular Diseases. Kidney Int . 2021;100(4):753-779. doi:10.1016/j.kint.2021.05.015 Effective date: 08 /01/2024 Revised date: 01/10/2024
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