IN-MED-P-366647 OMPP Approved Template on: 01/22/2021PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Givlaari (givosiran)BENEFIT TYPE Medical STATUS Prior Authorization Required Givlaari, approved by the FDA in 2019, is an aminolevulinate synthase 1-directed small interfering RNA indicated for the treatment of adults with acute hepatic porphyria (AHP). It works by caus ing degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes to lower induced ALAS1, thereby leading to reduced accumulation of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG ) which are associated with AHP attacks and chronic manifestations . In the pivotal phase 3 trial ENVISION, patients treated with Givlaari experienced an average of 70% fewer porphyria attacks than the placebo group. Porphyrias are rare genetic disorders characterized by abnormally high levels of porphyrins in the body due to certain enzyme defects in the hem e bio synthesis pathway. There are four types of acute hepatic porphyria (AHP), which are characterized by potentially life-threatening neurologic attacks. The most common and severe type is acute intermittent porphyria ( AIP). Women of childbearing age are the most affected population, and the most common attack symptom is severe abdominal pain. Up to 8% of AHP patients have recurrent attacks, defined as more than 4 attacks per year. Givlaari is a treatment option for such patients. Commonly, IV hem in (Panhematin) has been used off label for attack prevention and is the mainstay for treating acute attacks.Givlaari (givosiran) will be considered for coverage when the following criteria are met:A cute H epatic Porphyria (AHP )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, hematologist, gastroenterologist, or hepatologist; AND 3. Member has a documented diagnosis of Acute Hepatic Porphyria with one of the following types: a cute intermittent porphyria (AIP), hereditary coproporphyria (HCP) , variegate porphyria (VP), or aminolevulinic acid dehydratase deficiency porphyria (ADP); AND 4. Members diagnosis must be confirmed by labs showing an elevated level of urinary ALA or PBG (4 times the upper limit of the normal) within the past year; AND 5. Chart notes must include documentation of active recurrent porphyria attacks with one of the following: a) A t least 4 attacks in the past year requiring hospitalization, urgent care visit, or intravenous hemin administration at home OR b) Member is currently receiving off-label hemin treatment for attack prophylaxis; AND 6. Member has been assessed for possible attack triggers such as certain drugs, smoking, alcohol, or low carb diet s, and counseled to avoid these potential caus ative factors; AND 7. Member does NOT have any of the following: a) Prior or anticipated liver transplant b) Active HIV , hepatitis B, or hepatitis Cinfection ; AND 8. Member will not be receiving concomitant prophylactic treatment with IV hemin ( Panhematin ). Note: Hemin for treatment of acute attacks is permitted. 9. Dosage allowed/Quantity limit: 2.5 mg/kg subcutaneous injection once monthly . IN-MED-P-366647 OMPP Approved Template on: 01/22/2021If all the above requirements are met , the medication will be approved for 6 months .For reauthorization :1. Chart notes must document at least one of the following: a) Reduced number of porphyria attacks requiring hospitalization, urgent care, or Panhematin use b) Reduced severity of attack symptoms such as pain and decreased opioid use; AND 2. Member is not using Panhematin for attack prophylaxis (allowed for acute use only); AND 3. Member has not had and is not anticipating a liver transplant. If all the above requirements are met , the medication will be approved for an additional 12 months .CareSource considers Givlaari (givosiran) 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/DESCRIPTION04/23/2020 New policy for Givlaari created.12/20/2022 Transferred to new template. Updated Jcode. Updated and added references. Added diagnostic confirmation via biochemical testing. Added criterion for trigger avoidance. Added neurology and hematology to accepted prescriber specialties. Rephrased re-auth criteria and made attack reduction more specific; added symptom/pain reduction, added liver transplant exclusion. 01/26/2024 Added new reference (AGA 2023). Changed recurrent attack definition from clinical trial definition to guideline definition. Added that if they have already been receiving prophylactic hemin then they dont have to meet recurrent attack definition. References: 1. Givlaari [package insert]. Summit, NJ: Celgene Corporation, November 20 23. 2. Balwani M, Wang B, Anderson KE, et al. Acute hepatic porphyrias: Recommendations for evaluation and long-term management. Hepatology. 2017;66(4):1314 1322. doi:10.1002/hep.29313. 3. Balwani M, Sardh E, Ventura P, et al. Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. NEngl JMed. 2020;382(24):2289-2301. doi:10.1056/NEJMoa1913147 4. Ventura P, Bonkovsky HL, Gouya L, et al. Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study. Liver Int . 2022;42(1):161-172. doi:10.1111/liv.15090 5. Stlzel U, Doss MO, Schuppan D. Clinical Guide and Update on Porphyrias. Gastroenterology . 2019;157(2):365-381.e4. doi:10.1053/j.gastro.2019.04.050 6. Stein PE, Badminton MN, Rees DC. Update review of the acute porphyrias. Br JHaematol. 2017;176(4):527-538. doi:10.1111/bjh.14459 7. Majeed CN, Ma CD, Xiao T, Rudnick S, Bonkovsky HL. Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy. Drug Des Devel Ther . 2022;16:1827-1845. Published 2022 Jun 16. doi:10.2147/DDDT.S281631 8. Anderson KE. Acute hepatic porphyrias: Current diagnosis & management. Mol Genet Metab. 2019;128(3):219-227. doi:10.1016/j.ymgme.2019.07.002 9. Kothadia JP, LaFreniere K, Shah JM. Acute Hepatic Porphyria. [Updated 2022 May 8]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK537178/ 10. Wang B, Ventura P, Takase KI, et al. Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. Orphanet JRare Dis . 2022;17(1):327. Published 2022 Aug 26. doi:10.1186/s13023-022-02463-xIN-MED-P -366647 OMPP Approved Template on: 01/22/202111. Wang B, Bonkovsky HL, Lim JK, Balwani M. AGA Clinical Practice Update on Diagnosis and Management of Acute Hepatic Porphyrias: Expert Review. Gastroenterology . 2023;164(3):484-491. doi:10.1053/j.gastro.2022.11.034 Effective date: 08/01/2024 Revised date: 01/26/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Filsuvez (birch triterpenes )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Filsuvez topical gel was approved by the FDA in 2023. It is a botanical drug product indicated for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa in adult and pediatric patients 6 months of age and older . Epidermolysis Bullosa (EB) is a rare genetic disease with skin fragility and mechanically induced blistering that can lead to infections, scarring, and disfigurement. Dystrophic EB (DEB ) is one of four EB subtypes. It is caused by mutations in COL7A1, the gene that codes collagen type VII (C7), the major component of anchoring fibrils in part of the skin. DEB can be autosomal dominant (DDEB) or recessive (RDEB). RDEB is more severe. Junctional EB (JEB) is usually caused by mutations in the laminin-332 genes. In the EASE phase III study , the primary endpoint was met for first complete closure of target wound within 45 days .Filsuvez (birch triterpenes) will be considered for coverage when the following criteria are met:Epidermolysis B ullosa (EB)For initial authorization: 1. Member is at least 6 months of age ; AND 2. Medication must be prescribed by or in consultation with a dermatologist; AND 3. Member has a diagnosis of dystrophic or junctional EB confirmed by genetic testing results; AND 4. Member has at least 1 partial-thickness wound to be treated, that has been present for at least 21 days and does not appear infected; AND 5. Member will continue standard wound care . 6. Dosage allowed/Quantity limit: Apply a 1 mm layer of gel to the affected wound surface and cover with wound dressing or apply directly to dressing so that the topical gel is in direct contact with the wound. Apply at wound dressing changes until the wound is healed. QL: 30 tubes/30 days If all the above requirements are met , the medication will be approved for 3 months. For reauthorization : 1. Documentation of complete closure of EB wound treated with Filsuvez ; AND 2. Member has other remaining EB wound(s) to be treated that meet initial criteria. 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 Filsuvez (birch triterpenes) 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/DESCRIPTION01/30/2024 New policy for Filsuvez created. References: 1. Filsuvez [prescribing information]. Lichtenheldt GmbH; 2023. 2. Kern JS, Sprecher E, Fernandez MF, et al. Efficacy and safety of Oleogel-S10 (birch triterpenes) for epidermolysis bullosa: results from the phase III randomized double-blind phase of the EASE study. Br JDermatol. 2023;188(1):12-21. doi:10.1093/bjd/ljac001 3. Torres Pradilla M, lvarez E, Novoa M, Lozano I, Trujillo M. Oleogel-S10 in Dystrophic Epidermolysis Bullosa: A Case Series Evaluating the Impact on Wound Burden Over Two Years. Adv Ther. 2024;41(2):867-877. doi:10.1007/s12325-023-02749-x 4. Has C, Liu L, Bolling MC, et al. Clinical practice guidelines for laboratory diagnosis of epidermolysis bullosa. Br JDermatol . 2020;182(3):574-592. doi:10.1111/bjd.18128 4 5. Has C, Bauer JW, Bodemer C, et al. Consensus reclassification of inherited epidermoly sis bullosa and other disorders with skin fragility. Br JDermatol. 2020;183(4):614-627. doi:10.1111/bjd.18921 Effective date: 07/01/2024 Revised date: 01/30/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Fabhalta (iptacopan )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Fabhalta, approved by the FDA in 2023, is a first-in-class, oral complement factor Binhibitor, indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) . Like Empaveli, Fabhalta controls both intravascular and extravascular hemolysis , unlike Soliris and Ultomiris, which only impact intravascular hemolysis . The APPLY-PNH study demonstrated superiority of Fabhalta versus continuation of Soliris or Ultomiris for outcomes including hemoglobin levels and transfusion avoidance. PNH is a hematopoietic stem cell disorder in which activation of the complement system destroys red blood cells because of an acquired mutation in the PIGA gene. Common manifestations can include hemolytic anemia and fatigue. Thrombosis and bone marrow suppression may also occur .Fabhalta (iptacopan) will be considered for coverage when the following criteria are met:Paroxysmal N octurnal H emoglobinuria (PNH )For initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a hematologist; AND 3. Member has a documented diagnosis of PNH as confirmed by high-sensitivity flow cytometry with clone size 10% ; 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., fatigue, hemoglobin
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Elaprase (idursulfase)BENEFIT TYPE Medical STATUS Prior Authorization Required Elaprase is an enzyme replacement therapy that was approved by the FDA in 2006 for the treatment of Mucopolysaccharidosis type I I, also known as MPS II or Hunter syndrome. MPS I I is a rare, X-linked lysosomal storage dis ease mostly affecting males, which distinguishes it from the other MPS types which are autosomal recessive . Hunter syndrome can be classified as either severe or attenuated. Pathogenic mutations of the iduronate 2-sulfatase (IDS or I2S ) gene cause the enzyme iduronate 2-sulfatase to be deficient or absent. Normally this lysosomal enzyme breaks down glycosaminoglycans (GAGs) (previously known as mucopolysaccharides) but when reduced in MPS I I, the GAG substrate s hepar an sulfate (HS) and dermatan sulfate (DS) accumulate throughout the body causing chronic progressive damage. Elaprase has been shown to improve somatic manifestations but does not impact neurologic symptoms because it does not penetrate the blood-brain barrier. MPS I and II are the MPS types that display both somatic and neurologic symptoms. MPS I progresses faster than MPS II.Elaprase (idursulfase) will be considered for coverage when the following criteria are met:Mucopolysaccharidosis II (MPS II ; Hunter Syndrome )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 diagnosis of MPS II confirmed by at least one of the following: a) Low iduronate 2-sulfatase enzyme activity AND normal activity of a second sulfatase (to exclude Multiple Sulfatase Deficiency) , and/or b) Molecular genetic testing identifies pathogenic IDS gene mutation; AND 3. Documentation of baseline urinary GAG (uGAG) level; AND 4. Member does NOT have severe neurologic impairment (such as being in a vegetative state or fed by gastrostomy due to inability to swallow). 5. Dosage allowed/Quantity limit: 0.5 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 liver and spleen volumes, 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 Elaprase (idursulfase ) 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/DESCRIPTION07/22/2021 New policy for Elaprase created. 12/29/2023 Updated references. Removed age limit. References: 1. Elaprase [package insert]. Lexington, MA: Shire Human Genetic Therapies, Inc.; 2021 . 2. da Silva EM, Strufaldi MW, Andriolo RB, Silva LA. Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome). Cochrane Database Syst Rev . 2016;2(2):CD008185. Published 2016 Feb 5. doi:10.1002/14651858.CD008185.pub4 3. Muenzer J, Wraith JE, Beck M, et al. A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome) [published correction appears in Genet Med. 2006 Sep;8(9):599. Wendt, Suzanne [corrected to Wendt, Susanne]; Puga, Antonio [corrected to Puga, Ana Cristina]; Conway, Ann Marie [corrected to Conway, Anne Marie]]. Genet Med . 2006;8(8):465-473. doi:10.1097/01.gim.0000232477.37660.fb 4. 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 5. Giugliani R, Federhen A, Rojas MV, et al. Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. Genet Mol Biol . 2010;33(4):589-604. doi:10.1590/S1415-47572010005000093 6. Scarpa M. Mucopolysaccharidosis Type II. 2007 Nov 6 [Updated 2018 Oct 4]. 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/NBK1274/7. Scarpa M, Almssy Z, Beck M, et al. Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease. Orphanet JRare Dis . 2011;6:72. Published 2011 Nov 7. doi:10.1186/1750-1172-6-72 8. Wraith JE, Scarpa M, Beck M, et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur JPediatr . 2008;167(3):267-277. doi:10.1007/s00431-007-0635-4 9. Muenzer J, Bodamer O, Burton B, et al. The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. Eur JPediatr . 2012;171(1):181-188. doi:10.1007/s00431-011-1606-3 10. McBride KL, Berry SA, Braverman N; ACMG Therapeutics Committee. Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG). Genet Med. 2020;22(11):1735-1742. doi:10.1038/s41436-020-0909-z Effective date: 07/01/2024 Revised date: 12/29/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Pombiliti (c ipaglucosidase alfa-atga ) andOpfolda (miglustat) BENEFIT TYPE Medical STATUS Prior Authorization Required Pombiliti and Opfolda, approved by the FDA in 2023, are indicated to be used in combination for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing 40 kg and who are not improving on their current enzyme replacement therapy (ERT) . Pombiliti provides an exogenous source of the deficient GAA enzyme , and Opfolda is an enzyme stabilizer. Pompe disease is a rare, genetic lysosomal storage disorder that results in the buildup of glycogen in cell lysosomes causing serious and life-threatening muscle damage and weakness . It can be broadly classified as infantile onset within the first few months of life (IOPD) or late onset beyond infancy (LOPD). Classic IOPD is rapidly progressive with severe cardiomyopathy. Non-classic IOPD progresses slower with less severe cardiomyopathy. LO PD does not typically present with cardiomyopathy and has more vari able symptoms, especially skeletal muscle weakness. Pombiliti and Opfolda are only indicated to treat LOPD. Of note, miglustat is also the active ingredient in Zavesca, which is approved for Gaucher disease.Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) will be considered for coverage when the following criteria are met:Pompe DiseaseFor initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a geneticist, neurologist, pulmonologist, or metabolic specialist ; AND 3. Pombiliti and Opfolda are being prescribed in combination; AND 4. Member has a diagnosis of late onset Pompe disease confirmed by an enzyme activity assay showing GAA deficiency (2% to 40% of normal); AND 5. Molecular genetic testing shows pathogenic mutation of the GAA gene; AND 6. Member is not improving on their current enzyme replacement therapy (i.e., Lumizyme or Nexviazyme); AND 7. Member must show signs or symptoms (i.e., motor weakness, reduced respiratory parameters) ; AND 8. Documentation of baseline percent-predicted forced vital capacity (FVC) and 6-minute walk test (6MWT) ; AND 9. If female, attestation that the member is not pregnant. 10. Dosage allowed/Quantity limit: Starting 2 weeks after the last ERT dose: Pombiliti: 20 mg/kg every other week IV infusion Opfolda: Orally every other week; 260 mg for patients weighing 50 kg or 195 mg for patients weighing 40 kg to
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Nexviazyme (avalglucosidase alfa-ngp )BENEFIT TYPE Medical STATUS Prior Authorization Required Nexviazyme is an enzyme replacement therapy for the treatment of late-onset Pompe disease, also known as acid alpha-glucosidase (GAA) deficiency or glycogen storage disease type II. Pompe disease is a rare, genetic lysosomal storage disorder that results in the buil dup of glycogen in cell lysosomes causing serious and life-threatening muscle damage and weakness. Nexviazyme provides an exogenous source of the deficient GAA enzyme to cleave glycogen and reduce its accumulation. In the COMET trial, Nexviazyme was found to be non-inferior to Lumizyme. Pompe disease can be broadly classified as infantile-onset within the first few months of life (IOPD) or late-onset beyond infancy (LOPD). Classic IOPD is rapidly progressive with severe cardiomyopathy. Non-classic IOPD progresses slower with less severe c ardiomyopathy. LOPD does not typically present with cardiomyopathy and has more variable symptoms, especially skeletal muscle weakness. Nexviazyme is only indicated to treat late-onset Pompe disease.Nexviazyme (avalglucosidase alfa-ngp) will be considered for coverage when the following criteria are met:Pompe disease ( acid -glucosidase [GAA ] deficiency) For initial authorization: 1. Member is at least 1 year of age; AND 2. Medication must be prescribed by or in consultation with a geneticist, neurologist, pulmonologist, or metabolic specialist ; AND 3. Member has a diagnosis of late onset Pompe disease confirmed by an enzyme activity assay showing GAA deficiency (2% to 40% of normal); AND 4. Molecular genetic testing shows pathogenic mutation of the GAA gene; AND 5. Member must show signs or symptoms (i.e., motor weakness, reduced respiratory parameters). 6. Dosage allowed/Quantity limit: Actual body weight 30 kg or greater: 20 mg/kg IV infusion every 2 weeks Actual body weight less than 30 kg: 40mg/kg IV infusion every 2 weeks If all the above requirements are met , the medication will be approved for 12 months. For reauthorization :1. Chart notes must document positive clinical response such as improved or stabilized respiratory muscle strength (i.e., forced vital capacity (FVC)) or functional endurance (e.g., 6-minute walk test). 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 Nexviazyme (avalglucosidase alfa-ngp) 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/DESCRIPTION08/20/2021 New policy for Nexviazyme created. 05/24/2022 Updated Jcode. 11/22/2023 Annual review; updated reference. References: 1. Nexviazyme [package insert]. Genzyme Corporation; 2023. 2. Kushlaf H, Attarian S, Borges JL, et al. Efficacy and Safety Results of the Avalglucosidase alfa Phase 3 COMET Trial in Late-Onset Pompe Disease Patients (4195) . Neurology . 2021;96(15 Supplement). https://n.neurology.org/content/96/15_Supplement/4195/tab-article-info3. Diaz-Manera J, Kishnani PS, Kushlaf H, et al. Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial [published correction appears in Lancet Neurol. 2022 Apr;21(4):e4]. Lancet Neurol . 2021;20(12):1012-1026. doi:10.1016/S1474-4422(21)00241-6 4. Tarnopolsky M, Katzberg H, Petrof BJ, et al. Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel. Can JNeurol Sci . 2016;43(4):472-485. doi:10.1017/cjn.2016.37 5. Burton BK, Kronn DF, Hwu WL, Kishnani PS; Pompe Disease Newborn Screening Working Group. The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease. Pediatrics . 2017;140(Suppl 1):S14-S23. doi:10.1542/peds.2016-0280D 6. Kronn DF, Day-Salvatore D, Hwu WL, et al. Management of Confirmed Newborn-Screened Patients With Pompe Disease Across the Disease Spectrum. Pediatrics. 2017;140(Suppl 1):S24-S45. doi:10.1542/peds.2016-0280E 7. van der Ploeg AT, Kruijshaar ME, Toscano A, et al. European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. Eur JNeurol . 2017;24(6):768-e31. doi:10.1111/ene.13285 8. 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 9. Cupler EJ, Berger KI, Leshner RT, et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve. 2012;45(3):319-333. doi:10.1002/mus.22329 Effective date: 04/01/2024 Revised date: 11/22/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Sohonos (palovarotene)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Sohonos , initially approved by the FDA in 2023, is a retinoid indicated for reduction in the volume of new heterotopic ossification in adults and children aged 8 years and older for females and 10 years and older for males with fibrodysplasia ossificans progressi va (FOP). Through binding to RAR, Sohonos decreases the BMP/ALK2 downstream signaling pathway by inhibiting the phosphorylation of SMAD1/5/8, which reduces ALK2/SMAD-dependent chondrogenesis and osteocyte differentiation resulting in reduced endochondral bone formation. FOP is an ultra-rare condition that causes abnormal bone growth in areas outside of the skeleton such as ligaments, tendons, and muscles. The disease progresses with flare-up episodes that lead to rapid heterotopic ossification (HO), severely restricting m obility and function as well as quality of life. Approval was based on the phase 3 MOVE trial which did not meet the primary endpoint of annualized volume of new HO measured by low-dose whole-body computed tomography (WBCT). However, a post hoc 18-month interim analysis showed that Sohonos reduced annual ized HO volume by 54% compared with standard of care.Sohonos (palovarotene) will be considered for coverage when the following criteria are met: Fibrodysplasia Ossificans Progressiva (FOP)For initial authorization: 1. If member is female, member is at least 8 years of age; OR 2. If member is male, member is at least 10 years of age; AND 3. Medication must be prescribed by or in consultation with an orthopedic, orthopedic surgeon, genetic specialist, pediatric endocrinologist or rheumatologist; AND 4. Member has a diagnosis of FOP with the ACVR1 R206H mutation confirmed by genetic testing; AND 5. If member has not reached skeletal maturity or final adult height, chart notes must include BOTH of the following: a) Radiological evidence of baseline bone age (x-ray results must be included); b) Baseline linear growth chart; AND, 6. If member is of reproductive potential, attestation that member is NOT pregnant. 7. Dosage allowed/Quantity limit: Adults and Pediatric Patients 14 Years and Older a) Daily dose: 5 mg daily b) Flare-up dose: 20 mg daily for 4 weeks, followed by 10 mg daily for 8 weeks (for a total of 12 weeks of flare-up treatment), even if symptoms resolve earlier, then return to daily dosing of 5 mg. Pediatric Patients Aged 8 to 13 Years for Females and Aged 10 to 13 Years for Males a) Daily dose: weight based (see table below). b) Flare-up dose: weight based (see table below). Administer the initial flare-up dosage once daily for 4 weeks, then administer the lower flare-up dosage once daily for 8 weeks (for a total of 12 weeks of flare-up treatment), even if symptoms resolve earlier , then return to daily dosing. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023If all the above requirements are met, the medication will be approved for 12 months.For reauthorization :1. Chart notes have been provided showing improvement of signs and symptoms of disease (such as reduced volume of new heterotopic ossifications, decreased flare ups, decreased pain or increased mobility); AND 2. If member has not reached skeletal maturity or final adult height, chart notes must include radiological evidence of appropriate bone age (x-ray results must be included) and linear growth.If all the above requirements are met, the medication will be approved for an additional 12 months. CareSource considers Sohonos (palovarotene) 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/DESCRIPTION11/9/2023 New policy for Sohonos created. References: 1. Sohonos [prescribing information]. Cambridge, MA: Ipsen Biopharmaceuticals, Inc.; 2023. 2. Pignolo RJ, Hsiao EC, Al Mukaddam M, et al. Reduction of New Heterotopic Ossification (HO) in the Open-Label, Phase 3 MOVE Trial of Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP). JBone Miner Res. 2023;38(3):381-394. doi:10.1002/jbmr.4762. 3. Kaplan FS, et al. The medical management of fibrodysplasia ossificans progressiva: current treatment considerations. Proc Intl Clin Council FOP 2: 1-127, 2022. 4. Smilde BJ, Botman E, de Ruiter RD, et al. Monitoring and Management of Fibrodysplasia Ossificans Progressiva: Current Perspectives [published correction appears in Orthop Res Rev. 2022 May 04;14:147-148]. Orthop Res Rev . 2022;14:113-120. Published 2022 Apr 20. doi:10.2147/ORR.S337491 Effective date: 04/ 01/2024 Revised date: 11/09/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Myalept (metreleptin )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Myalept was approved in 2014 to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. The defining feature of lipodystrophy is the selective loss of subcutaneous adipose tissue. Leptin is a hormone secreted by adipose tissue that informs the brain of the status of energy stores in the body. The leptin deficiency resulting from adipose tissue loss contributes to metabolic abnormalities. The replacement of leptin with metreleptin, which mimics native leptin, helps improve metabolic parameters. It must be used as an adjunct to diet, the fundamental treatment for lipodystrophy. It is only available through the Myalept REMS program.Myalept (metreleptin) will be considered for coverage when the following criteria are met:LipodystrophyFor initial authorization: 1. Medication must be prescribed by or in consultation with an endocrinologist; AND 2. Member has a diagnosis of congenital or acquired generalized lipodystrophy (CGL or AGL) ; AND 3. Member has a metabolic abnormality (e.g. diabetes, hypertriglyceridemia, insulin resistance) that has not responded to optimized standard therapy (e.g. metformin, insulin, statins, fibrates); AND 4. Member has failed dietary/lifestyle modifications and medication is prescribed in conjunction with ongoing diet management; AND 5. Member does not have any of the following: a) HIV-associated lipodystrophy ; b) General obesity (without leptin deficiency) . 6. Dosage allowed/Quantity limit: Administer as a subcutaneous injection once daily. See package insert for adult and pediatric dosing charts. The maximum dose is 10mg/day (2mL). Quantity Limit: 30 vials per 30 days. If all the above requirements are met , the medication will be approved for 6 months. For reauthorization : 1. Chart notes must demonstrate improved signs and symptoms of leptin deficiency, such as reductions in HbA1c, fasting glucose, or triglycerides. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Myalept (metreleptin) not medically necessary for the treatment of conditions that are not listed in this document. For any other indication, please refer to the Off-Label policy. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023DATE ACTION/DESCRIPTION01/08/2021 New policy for Myalept created. 09/25/2023 Updated references; added quantity limit; added dosing information; removed initial criteria requirement from reauthorization. References: 1. Myalept (metreleptin) [package insert]. Dublin, Ireland: Amryt Pharmaceuticals DAC; 2022. 2. Brown RJ, Araujo-Vilar D, Cheung PT, et al. The Diagnosis and Management of Lipodystrophy Syndromes: A Multi-Society Practice Guideline. JClin Endocrinol Metab. 2016;101(12):4500-4511. doi:10.1210/jc.2016-24663. Handelsman Y, Oral EA, Bloomgarden ZT, et al. The clinical approach to the detection of lipodystrophy-anAACE consensus statement. Endocr Pract . 2013;19(1):107-116. doi:10.4158/endp.19.1.v767575m65p5mr06 4. Meehan CA, Cochran E, Kassai A, Brown RJ, Gorden P. Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy. Expert Rev Clin Pharmacol . 2016;9(1):59-68. doi:10.1586/17512433.2016.1096772 5. Arajo-Vilar D, Santini F. Diagnosis and treatment of lipodystrophy: a step-by-step approach. JEndocrinol Invest . 2019;42(1):61-73. doi:10.1007/s40618-018-0887-z Effective date: 04/01/2024 Revised date: 09/25/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Lumizyme (alglucosidase alfa)BENEFIT TYPE Medical STATUS Prior Authorization Required Lumizyme is an enzyme replacement therapy for the treatment of Pompe disease, also known as acid alpha-glucosidase (GAA) deficiency or glycogen storage disease type II. Pompe disease is a rare, genetic lysosomal storage disorder that results in the buildup of glycogen in cell lysosomes causing serious and life-threatening muscle damage and weakness. Lumizyme replaces the deficient GAA enzyme to reduce the glycogen accumulation. Pompe disease can be broadly classified as infantile-onset within the first few months of life (IOPD) or late-onset beyond infancy (LOPD). Classic IOPD is rapidly progressive with severe cardiomyopathy. Non-classic IOPD progresses slower with less severe c ardiomyopathy. LOPD does not typically present with cardiomyopathy and has more variable symptoms, especially skeletal muscle weakness . Lumizyme (alglucosidase alfa) will be considered for coverage when the following criteria are met:Pompe disease ( acid -glucosidase [GAA ] deficiency) For initial authorization: 1. Medication must be prescribed by or in consultation with a geneticist, cardiologist, neurologist, pulmonologist, or metabolic specialist ; AND 2. Member has a diagnosis of Pompe disease confirmed by an enzyme activity assay showing GAA deficiency (0% to 40% of normal); AND 3. Molecular genetic testing shows pathogenic mutation of the GAA gene; AND 4. Members with late onset Pompe disease must show signs or symptoms (i.e., motor weakness, reduced respiratory parameters). 5. Dosage allowed/Quantity limit: 20 mg/kg IV infusion every 2 weeks If all the above requirements are met , the medication will be approved for 12 months. For reauthorization : 1. Chart notes must document positive clinical response such as improved or stabilized motor function or ambulation, pulmonary function, or cardiomyopathy. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Lumizyme (alglucosidase 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. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023DATE ACTION/DESCRIPTION07/0 7/2021 New policy for Lumizyme created. 11/09/2022 Annual review; no changes. 11/22/2023 Annual review; no changes. References: 1. Lumizyme [prescribing information]. Cambridge, MA: Genzyme Corporation; 2023 . 2. Chen M, Zhang L, Quan S. Enzyme replacement therapy for infantile-onset Pompe disease. Cochrane Database Syst Rev. 2017;11(11):CD011539. Published 2017 Nov 20. doi:10.1002/14651858.CD011539.pub2 3. Schoser B, Stewart A, Kanters S, et al. Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis. JNeurol . 2017;264(4):621-630. doi:10.1007/s00415-016-8219-8 4. 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 5. Tarnopolsky M, Katzberg H, Petrof BJ, et al. Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel. Can JNeurol Sci . 2016;43(4):472-485. doi:10.1017/cjn.2016.37 6. van der Ploeg AT, Kruijshaar ME, Toscano A, et al. European consensus for starting and stopping enzyme replacement therapy in adult patients with Pompe disease: a 10-year experience. Eur JNeurol . 2017;24(6):768-e31. doi:10.1111/ene.13285 7. Cupler EJ, Berger KI, Leshner RT, et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve. 2012;45(3):319-333. doi:10.1002/mus.22329 8. Burton BK, Kronn DF, Hwu WL, Kishnani PS; Pompe Disease Newborn Screening Working Group. The Initial Evaluation of Patients After Positive Newborn Screening: Recommended Algorithms Leading to a Confirmed Diagnosis of Pompe Disease. Pediatrics . 2017;140(Suppl 1):S14-S23. doi:10.1542/peds.2016-0280D 9. Kronn DF, Day-Salvatore D, Hwu WL, et al. Management of Confirmed Newborn-Screened Patients With Pompe Disease Across the Disease Spectrum. Pediatrics. 2017;140(Suppl 1):S24-S45. doi:10.1542/peds.2016-0280E 10. Marques JS. The Clinical Management of Pompe Disease: A Pediatric Perspective. Children (Basel) . 2022;9(9):1404. Published 2022 Sep 16. doi:10.3390/children9091404 Effective date: 04/01/2024 Revised date: 11/22/2023
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Empaveli (pegcetacoplan)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Empaveli is the first and only FDA-approved drug for PNH that controls both intravascular and extravascular hemolysis. In contrast to Soliris and Ultomiris, C5 inhibitors which only impact intravascular hemolysis, Empaveli is a C3 inhibitor. The phase 3 PEGASUS st udy concluded Empaveli was superior to Soliris in terms of improving hemoglobin levels and freedom from transfusion. PNH is a hematopoietic stem cell disorder in which activation of the complement system destroys red blood cells because of an acquired mutation in the PIGA gene. Common manifestations can include hemolytic anemia and fatigue. Thrombosis and bone marrow suppression may also occur .Empaveli (pegcetacoplan) will be considered for coverage when the following criteria are met:Paroxysmal nocturnal hemoglobinuria (PNH)For initial authorization: 1. Member is at least 18 years of age; AND 2. Medication must be prescribed by or in consultation with a hematologist; AND 3. Member has a documented diagnosis of PNH as confirmed by flow 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., fatigue, hemoglobin
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