IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Joenja (leniolisib)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Joenja, approved by the FDA in 2023, is a small molecule kinase inhibitor indicated for the treatment of activated phosphoinositide 3-kinase delta (PI3K) syndrome (APDS) in adult and pediatric patients 12 years of age and older . It selectively targets PI3K signaling by inhibiting its hyperactive subunit . APDS is also known as p110-activating mutation causing senescent Tcells, lymphadenopathy, and immunodeficiency (PASLI) . It is an ultra-rare disease of primary immunodeficiency caused by variants in either of the genes encoding the PI3K heterodimer ( PIK3CD in APDS1 or PIK3R1 in APDS2 ) which leads to hyperactive PI3K signaling. This results in disrupted immune cell development and function. Clinical manifestations include infections, nonmalignant lymphoproliferation, autoimmunity (e.g., cytopenias), enter opathy , bronchiectasis, and increased risk of lymphoma. In a Phase 3 study , Joenja met the coprimary endpoints of reducing lymphadenopathy and normalizing immune cell subsets . It is the first FDA-approved drug for APDS.Joenja (leniolisib) will be considered for coverage when the following criteria are met:A ctivated Phosphoinositide 3-K inase D elta (PI3K) syndrome (APDS)For initial authorization: 1. Member is at least 12 years of age ; AND 2. Medication must be prescribed by or in consultation with an immunologist or hematologist; AND 3. Member has a diagnosis of APDS confirmed by an APDS-associated genetic PI3K mutation with a documented variant in either PIK3CD or PIK3R1 ; AND 4. Chart notes must show clinical findings/manifestations of APDS (e.g., history of repeated oto-sino-pulmonary infections , at least 1 measurable nodal lesion by CT or MRI ). 5. Dosage allowed/Quantity limit: Weight 45 kg or greater: 70 mg orally twice daily. (QL: 60 tablets per 30 days) If all the above requirements are met , the medication will be approved for 6 months. For reauthorization : 1. Chart notes must show positive clinical response such as reduced lymph node size, increased nave Bcell percentage (out of total Bcells) , reduced spleen volume, or improved cytopenias . If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Joenja (leniolisib) 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/DESCRIPTION04/24/2023 New policy for Joenja created.03/07/2024 Added reference. Moved measurable nodal lesion to list of clinical findings rather than an additional requirement. Removed restriction for concurrent use with an immunosuppressive medication. References: 1. Joenja [prescribing information]. Pharming Technologies B.V.; 2023. 2. Rao VK, Webster S, ediv A, et al. A randomized, placebo-controlled phase 3 trial of the PI3K inhibitor leniolisib for activated PI3K syndrome. Blood . 2023;141(9):971-983. doi:10.1182/blood.2022018546 3. Rao VK, Webster S, Dalm VASH, et al. Effective "activated PI3K syndrome" -targeted therapy with the PI3K inhibitor leniolisib. Blood . 2017;130(21):2307-2316. doi:10.1182/blood-2017-08-801191 4. Coulter TI, Cant AJ. The Treatment of Activated PI3K Syndrome. Front Immunol . 2018;9:2043. Published 2018 Sep 7. doi:10.3389/fimmu.2018.02043 5. Singh A, Joshi V, Jindal AK, Mathew B, Rawat A. An updated review on activated PI3 kinase delta syndrome (APDS). Genes Dis . 2019;7(1):67-74. Published 2019 Oct 14. doi:10.1016/j.gendis.2019.09.015 6. Rao VK, Kulm E, ediv A, et al. Interim analysis: Open-label extension study of leniolisib for patients with APDS. JAllergy Clin Immunol . 2024;153(1):265-274.e9. doi:10.1016/j.jaci.2023.09.032 Effective date: 10/01/2024 Revised date: 03/07/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Scenesse ( afamelanotide)BENEFIT TYPE Medical STATUS Prior Authorization Required Scenesse, approved by the FDA in 2019, is a melanocortin 1 receptor (MC1-R) agonist indicated to increase pain free light exposure in adult patients with a history of phototoxic reactions from erythropoietic protoporphyria (EPP) . It was the first drug approved for the treatment of EPP. Scenesse is a structural analog of alpha-melanocyte stimulating hormone (alpha-MSH). It increases production of photoprotective eumelanin (a type of melanin pigment) in the ski n to help reduce sensitivity to light. EPP, a subtype of a broader group of disorders known as porphyrias, is a rare inherited metabolic disorder caused by deficiency of the enzyme ferrochelatase (FECH) due to mutations in the FECH gene. FECH has a role in the biosynthesis of heme. Low levels of FECH cause excess protoporphyrin to accumulate in certain tissues, which leads to the characteristic symptoms of EPP . The major symptom is skin hypersensitivity to sunlight and some types of artificial light. Complications may include gallbladder dysfunc tion and liver damage. Less commonly, EPP can be caused by a mutation in the ALAS2 gene. In these cases, it is referred to as X-linked protoporphyria (XLP).Scenesse (afamelanotide) will be considered for coverage when the following criteria are met:Erythropoietic Protoporphyria (EPP)For initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a dermatologist , hepatologist, gastroenterologist , or hematologist; AND 3. Member has a documented diagnosis of EPP confirmed with biochemical testing that shows elevated total erythrocyte protoporphyrin concentration > 3x ULN and majority (>50%) metal-free vs. zinc-bound; AND 4. Member exhibits characteristic symptoms of EPP phototoxicity (e.g., intolerance to light including pain, swelling, burning, itching, and redness of the skin during or after exposure to sunlight) which interferes with their quality of life (i.e., interference with work, activities of daily living, lifestyle choices, etc.); AND 5. Sun avoidance and use of protective measures ( i.e., sunscreen, protective clothing, etc. ) have been inadequate in controlling EPP phototoxic reactions; AND 6. Member does NOT have any of the following: a) EPP with severe hepatic involvement b) Untreated malignant or premalignant skin lesions . 7. Dosage allowed/Quantity limit: One 16 mg subcutaneous implant every 2 months (medical justification is required for requests beyond 3 implants a year for seasonal coverage) . QL: 1 implant per 60 days; 3 implants per year. 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 improvement of signs and symptoms of disease: Increased tolerance to sunlight exposure and/or decreased phototoxic pain; AND 2. Member is receiving skin exams as recommended in the prescribing information. If all the above requirements are met , the medication will be approved for an additional 12 months. CareSource considers Scenesse (afamelanotide) 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/DESCRIPTION06/17/2020 New policy for Scenesse created.10/24/2022 Updated/added references. Added GI, hepatology, hematology to specialists. Removed pain medications from criteria. Reworded renewal criteria and added skin exam.07/02/2024 Updated references. Changed elevated free protoporphyrin in peripheral erythrocytes to total erythrocyte protoporphyrin concentration > 3x ULN and majority (>50%) metal-free vs. zinc-bound. Removed genetic testing option as it is not recommended in the absence of biochemical testing (Dickey 2023). References: 1. Scenesse [package insert]. Clinuvel, Inc.; 2023 . 2. Langendonk JG, Balwani M, Anderson KE, et al. Afamelanotide for Erythropoietic Protoporphyria. NEngl JMed. 2015;373(1):48 59. doi:10.1056/NEJMoa1411481 3. Wensink D, Wagenmakers MAEM, Langendonk JG. Afamelanotide for prevention of phototoxicity in erythropoietic protoporphyria. Expert Rev Clin Pharmacol . 2021;14(2):151-160. doi:10.1080/17512433.2021.1879638 4. American Porphyria Foundation. Erythropoietic protoporphyria (EPP) and X-Linked Protoporphyria (XLP), https://porphyriafoundation.org/for-patients/types-of-porphyria/epp-xlp/5. Balwani M. Erythropoietic Protoporphyria and X-Linked Protoporphyria: pathophysiology, genetics, clinical manifestations, and management. Mol Genet Metab. 2019;128(3):298-303. doi:10.1016/j.ymgme.2019.01.020 6. Ahmed jan N, Masood S. Erythropoietic Protoporphyria. [Updated 2023 Feb 16]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan- . Available from: https://www.ncbi.nlm.nih.gov/books/NBK563141/ 7. Leaf RK, Dickey AK. How I treat erythropoietic protoporphyria and X-linked protoporphyria. Blood . 2023;141(24):2921-2931. doi:10.1182/blood.2022018688 8. Dickey AK, Naik H, Keel SB, et al. Evidence-based consensus guidelines for the diagnosis and management of erythropoietic protoporphyria and X-linked protoporphyria. JAm Acad Dermatol . 2023;89(6):1227-1237. doi:10.1016/j.jaad.2022.08.036 Effective date: 01/01/2025 Revised date: 07/02/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Palynziq (pegvaliase-pqpz)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Palynziq, approved by the FDA in 2018, is a phenylalanine (Phe) -metabolizing enzyme indicated to reduce blood Phe concentrations in adult patients with phenylketonuria (PKU) who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Palynziq is only available through a REMS program due to a risk of anaphylaxis. PKU results from a deficiency of phenylalanine hydroxylase (PAH) enzyme, leading to increased concentrations of Phe. If untreated, this excess accumulation causes neuropsychiatric and neurocognitive symptoms. Palynziq is a PEGylated phenylalanine ammonia lyase (PAL) enzyme that converts phenylalanine to ammonia and trans-cinnamic acid. It works as an enzyme substitution therapy as PAL substitutes for the deficient PAH enzyme activity. Standard of care for PKU is a Phe-restricted diet.Palynziq (pegvaliase-pqpz) will be considered for coverage when the following criteria are met:Phenylketonuria (PKU)For initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with specialist experienced in metabolic or genetic diseases ; AND 3. Member has a diagnosis of phenylketonuria; AND 4. Member has uncontrolled blood phenylalanine (Phe) concentrations greater than 600 micromol/L on existing management with Kuvan* (requires prior authorization) in conjunction with following recommended dietary modifications; AND 5. Palynziq will not be prescribed in combination with Kuvan. 6. Dosage allowed/Quantity limit: Initial, 2.5 mg subQ once weekly x 4 weeks. Titrate over at least 5 weeks to 20 mg once daily. May increase to 40 mg daily after 24 weeks on 20 mg/day if control not achieved. May increase to 60 mg daily if control not achieved with 40 mg/day after 16 weeks. Discontinue after 16 weeks of 60 mg/day if adequate response not achieved. (Max dose 60 mg/day). QL: 90 syringes per 30 days *Note : A trial of Kuvan is not necessary if there is documentation of 2 null mutations. However, a trial and failure of compliant diet management is still required. 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 at least one of the following: a) Member has achieved at least a 20% reduction in blood phenylalanine concentration from pretreatment baseline b) Member has achieved a blood phenylalanine concentration of 600 micromol/L or less. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Palynziq (pegvaliase-pqpz) 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/DESCRIPTION07/27/2018 New policy for Palynziq created.04/30/2021 Updated references. Added requirements for dietary management and Kuvan. Removed exclusion criteria that were from clinical trial. Abbreviated dosing information and updated to reflect label change with new max. Amended renewal criteria.10/31/2022 Transferred to new template. Added QL. Changed initial auth duration from 12 months to 6 months. Split renewal criteria into 2 bullets for readability. 06/14/2024 Updated references. References: 1. Palynziq [package insert]. Novato, CA: BioMarin Pharmaceutical Inc.; November 2020. 2. Vockley J, Andersson HC, Antshel KM, et al. Phenylalanine hydroxylase deficiency: diagnosis and management guideline [published correction appears in Genet Med. 2014 Apr;16(4):356]. Genet Med. 2014;16(2):188-200. doi:10.1038/gim.2013.157 3. van Wegberg AMJ, MacDonald A, Ahring K, et al. The complete European guidelines on phenylketonuria: diagnosis and treatment. Orphanet JRare Dis. 2017;12(1):162. Published 2017 Oct 12. doi:10.1186/s13023-017-0685-2 4. van Spronsen FJ, van Wegberg AM, Ahring K, et al. Key European guidelines for the diagnosis and management of patients with phenylketonuria. Lancet Diabetes Endocrinol. 2017;5(9):743-756. doi:10.1016/S2213-8587(16)30320-5 5. Thomas J, Levy H, Amato S, et al. Pegvaliase for the treatment of phenylketonuria: Results of a long-term phase 3 clinical trial program (PRISM). Mol Genet Metab. 2018;124(1):27-38. doi:10.1016/j.ymgme.2018.03.006 6. Harding CO, Amato RS, Stuy M, et al. Pegvaliase for the treatment of phenylketonuria: A pivotal, double-blind randomized discontinuation Phase 3 clinical trial. Mol Genet Metab. 2018;124(1):20-26. doi:10.1016/j.ymgme.2018.03.003 7. Longo N, Dimmock D, Levy H, et al. Evidence-and consensus-based recommendations for the use of pegvaliase in adults with phenylketonuria. Genet Med. 2019;21(8):1851-1867. doi:10.1038/s41436-018-0403-z 8. Adams AD, Fiesco-Roa M, Wong L, et al. Phenylalanine hydroxylase deficiency treatment and management: A systematic evidence review of the American College of Medical Genetics and Genomics (ACMG). Genet Med . 2023;25(9):100358. doi:10.1016/j.gim.2022.12.005 Effective date: 01/01/2025 Revised date: 06/14/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Xolremdi (mavorixafor )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Xolremdi, approved by the FDA in 2024, is a CXC chemokine receptor 4 (CXCR4) antagonist indicated in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes . WHIM syndrome is a primary immunodeficiency characterized by retention of leukocytes in the bone marrow (myelokathexis), resulting in neutropenia, leukopenia, and sometimes hypogammaglobulinemia or warts, as well as recurrent infections and predisposition to malignancy. It is caused by mutations in CXCR4, resulting in hyperactivation of the CXCR4-CXCL12 pathway and myelokathexis . Xolremdi , the first approved treatment for WHIM syndrome, inhibits the hyperresponsiveness to the CXCR4 ligand CXCL12 to increase mobilization of neutrophils and lymphocytes from the bone marrow into peripheral circulation . This results in improvement in absolute neutrophil counts (ANC), improvement in absolute lymphocyte counts (ALC), and a reduction of infection frequency, severity, and duration.Xolremdi (mavorixafor) will be considered for coverage when the following criteria are met:WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis)For initial authorization: 1. Member is at least 12 years of age ; AND 2. Medication must be prescribed by or in consultation with an immunologist; AND 3. Member has a diagnosis of WHIM syndrome confirmed by genetic testing that shows mutation of the CXCR4 gene; AND 4. Member s labs show absolute neutrophil count (ANC) 400 cells/L in the absence of infection; AND 5. Members chart notes show baseline ALC and infection history . 6. Dosage allowed/Quantity limit: Weight more than 50 kg: 400 mg orally once daily (4 capsules) Weight less than or equal to 50 kg: 300 mg orally once daily (3 capsules) QL: 120 capsules per 30 days (1 capsule = 100 mg) If all the above requirements are met , the medication will be approved for 6 months. For reauthorization : 1. Chart notes must show clinically significant improve ment of ANC, ALC, and/or reduced infections since starting Xolremdi. 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 Xolremdi (mavorixafor) 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/DESCRIPTION05/01/2024 New policy for Xolremdi created. References: 1. Xolremdi [prescribing information]. X4 Pharmaceuticals, Inc.; 2024. 2. Badolato R, Alsina L, Azar A, et al. Phase 3 randomized trial of mavorixafor, CXCR4 antagonist, in WHIM syndrome. Blood. Published online April 21, 2024. doi:10.1182/blood.2023022658 3. Badolato R, Donadieu J; WHIM Research Group. How I treat warts, hypogammaglobulinemia, infections, and myelokathexis syndrome. Blood. 2017;130(23):2491-2498. doi:10.1182/blood-2017-02-708552 Effective date: 01/01/2025 Revised date: 05/01/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Xenpozyme (olipudase alfa-rpcp )BENEFIT TYPE Medical STATUS Prior Authorization Required Xenpozyme, approved by the FDA in 2022, is a hydrolytic lysosomal sphingomyelin-specific enzyme indicated for treatment of non central nervous system manifestations of acid sphingomyelinase deficiency (ASMD) inadult and pediatric patients . Xenpozyme provides the enzyme that is deficient or absent in ASMD. Without the enzyme acid sphingomyelinase (ASM), a complex lipid called sphingomyelin builds up in cells which can lead to multiorgan symptoms such as decreased lung function, enlarged liver and spleen, decreased platelet count, and growth delay in children, among many o ther manifestations. ASMD , a lysosomal storage disorder, is also known as Niemann-Pick disease and can be differentiated as type A, A/B, and B. Xenpozyme has not been studied in patients with ASMD type A. It is not expected to cross the blood-brain barrier or improve CNS manifestations. ASMD is an extremely rare genetic disease with fewer than 120 ASMD diagnoses in the U.S. Signs and symptoms may present in infancy, childhood, or adulthood with about two-thirds of diagnoses in the U.S. in pediatrics. Xenpozyme was the first approved treatment for ASMD. Xenpozyme ( olipudase alfa-rpcp ) will be considered for coverage when the following criteria are met: Acid Sphingomyelinase D eficiency (ASMD)For initial authorization: 1. Medication must be prescribed by or in consultation with a physician knowledgeable in the management of ASMD , such as an endocrinologist, hepatologist, or pulmonologist ; AND 2. Member has a confirmed diagnosis of ASMD with documentation of at least one of the following: a) ASM enzyme activity
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Voydeya (danicopan )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Voydeya, approved by the FDA in 2024, is a small molecule complement factor Dinhibitor indicated as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH) . 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. C5 inhibitors greatly reduce intravascular hemolysis (IVH; occurring within blood vessels) and thrombosis, however, EVH can be a mechanistic consequence of therapy and may lead to remaining dependent on transfusions. Voydeya acts proximally in the alternative pathway of the complement cascade to control EVH while the co-administered C5 inhibitor maintains control over IVH .Voydeya (danicopan) 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; AND 4. Member has been treated with a C5 inhibitor ( e.g., ravulizumab or eculizumab) for at least 6 months and will continue; AND 5. Member has clinically evident EVH with both of the following: a) Hemoglobin 9.5 g/dL or less, and b) Absolute reticulocyte count (ARC) 120 x 10 9/L or greater; AND 6. Member has been or will be vaccinated against encapsulated bacteria (Streptococcus pneumoniae, Neisseria meningitidis types A, C, W, Y, and B) . 7. Dosage allowed/Quantity limit: Start 150 mg 3 times a day orally. If hemoglobin has not increased by greater than 2 g/dL after 4 weeks or a transfusion was required, may increase to 200 mg 3 times a day . QL: 180 tablets per 30 days 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 clinical evidence of positive response to therapy such as increased hemoglobin level, decreased need for transfusions, improved fatigue; AND 2. Member is continuing treatment with a C5 inhibitor and not using Voydeya as monotherapy. If all the above requirements are met , the medication will be approved for an additional 12 months . CareSource considers Voydeya (danicopan ) 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/DESCRIPTION04/03/2024 New policy for Voydeya created. References: 1. Voydeya [prescribing information]. Alexion Pharmaceuticals, Inc.; 2024. 2. Lee JW, Griffin M, Kim JS, et al. Addition of danicopan to ravulizumab or eculizumab in patients with paroxysmal nocturnal haemoglobinuria and clinically significant extravascular haemolysis (ALPHA): a double-blind, randomised, phase 3 trial. Lancet Haematol. 2023;10(12):e955-e965. doi:10.1016/S2352-3026(23)00315-0 3. Risitano AM, Marotta S, Ricci P, et al. Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT. Front Immunol. 2019;10:1157. Published 2019 Jun 14. doi:10.3389/fimmu.2019.01157 4. Oliver M, Patriquin CJ. Paroxysmal Nocturnal Hemoglobinuria: Current Management, Unmet Needs, and Recommendations. JBlood Med. 2023;14:613-628. Published 2023 Dec 6. doi:10.2147/JBM.S431493 5. Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016;2016(1):208-216. doi:10.1182/asheducation-2016.1.208 6. Patriquin CJ, Kiss T, Caplan S, et al. How we treat paroxysmal nocturnal hemoglobinuria: A consensus statement of the Canadian PNH Network and review of the national registry. Eur JHaematol . 2019;102(1):36-52. doi:10.1111/ejh.13176 7. Devos T, Meers S, Boeckx N, et al. Diagnosis and management of PNH: Review and recommendations from a Belgian expert panel. Eur JHaematol. 2018;101(6):737-749. doi:10.1111/ejh.13166 8. Bod I, Amine I, Boban A, et al. Complement Inhibition in Paroxysmal Nocturnal Hemoglobinuria (PNH): A Systematic Review and Expert Opinion from Central Europe on Special Patient Populations. Adv Ther. 2023;40(6):2752-2772. doi:10.1007/s12325-023-02510-4 9. Sahin F, Akay OM, Ayer M, et al. Pesg PNH diagnosis, follow-up and treatment guidelines. Am JBlood Res. 2016;6(2):19-27. Published 2016 Aug 5. 10. Canado RD, Arajo ADS, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348. doi:10.1016/j.htct.2020.06.006 Effective date: 10/01/2024 Revised date: 04/03/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Ryplazim (plasminogen, human-tvmh )BENEFIT TYPE Medica l STATUS Prior Authorization Required Ryplazim is a plasma-derived human plasminogen indicated for the treatment of patients with plasminogen deficiency type 1 (hypoplasminogenemia). It was approved by the FDA on June 4, 2021 and is the first approved treatment for plasminogen deficiency type 1. Individuals with this disease lack a protein called plasminogen, which is responsible for the ability of the body to break down fibrin clots. Plasminogen deficiency leads to an accumulation of fibrin, causing the development of growths (lesions) that can impair normal tissue and organ function and may lead to blindness when these lesions affect the eyes. Ligneous conjunctivitis (LC) appears to be the most common clinical manifestation and is characterized by inflamed, woody growths on the conjunctival membr anes that, if left untreated, can result in visual impairment or blindness. Treatment with Ryplazim temporarily increases plasminogen levels in blood. The effectiveness and safety of Ryplazim (plasminogen) is primarily based on one single-arm, open-label (unblinded) clinical trial enrolling 15 adult and pediatric patients with plasminogen deficiency type 1. All patients received Ryplazim administered every two to four days for 48 weeks. The effectiveness of Ryplazim was demonstrated by at least 50% improvement of their lesions in all 11 patients who had lesions at baseline, and abs ence of recurrent or new lesions in any of the 15 patients through the 48 weeks of treatment.Ryplazim (plasminogen, human-tvmh) will be considered for coverage when the following criteria are met:HypoplasminogenemiaFor initial authorization: 1. Member must be at least 11 months old; AND 2. Medication must be prescribed by or in consultation with a hematologist; AND 3. Member has a documented history of disease-related lesions and symptoms consistent with a diagnosis of hypoplasminogenemia; AND 4. Documentation of baseline plasminogen activity level 45%. 5. Dosage allowed/Quantity limit: 6.6 mg/kg body weight given intravenously every 2 to 4 days If all the above requirements are met , the medication will be approved for 12 weeks . For reauthorization : Ryplazim will be reauthorized when chart notes show at least ONE of the following: a) Absence of recurrent or new lesions ; b) Decrease in the lesion number and/or size ; c) Increase in trough plasminogen activity level by at least 10% from baseline. If all the above requirements are met , the medication will be approved for an additional 12 months. IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023 CareSource considers Ryplazim (plasminogen, human-tvmh) 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/DESCRIPTION10/06 /20 21 Ryplazim policy creation 05/17/2024 Added reference (Shapiro, et al 2023); removed geneticist as prescriber option; added in consultation with for prescriber. References: 1. Ryplazim [package insert]. Laval, Quebec, CA; Prometric Bioproduction, Inc.; 2021. 2. Shapiro, Amy D. et al. Plasminogen replacement therapy for the treatment of children and adults with congenital plasminogen deficiency . Blood. 2018 Mar 22;131(12):1301-1310 3. Shapiro AD, Nakar C, Parker JM, Thibaudeau K, Crea R, Sandset PM. Plasminogen, human-tvmh for the treatment of children and adults with plasminogen deficiency type 1. Haemophilia . 2023;29(6):1556-1564. doi:10.1111/hae.14849 4. Shapiro AD, et al. An international registry of patients with plasminogen deficiency (HISTORY). Haematologica. 2020;105(3):554-561Effective date: 01/01/2025 Revised date: 05/17/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Rezurock (belumosudil)BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Rezurock , approved by the FDA in 2021, is a small molecule kinase inhibitor indicated for patients 12 years of age and older with chronic graft-versus-host disease (c GVHD) after failure of at least 2 prior lines of systemic therapy. GVHD, a common complication following allogeneic hematopoietic stem cell transplant (HSCT), occurs in about 50% of HSCT patients. Prednisone is the mainstay of initial therapy but at least half of pati ents require at least 2 lines of therapy. Rezurock is the first rho-associated, coiled-coil kinase 2 (ROCK2) inhibitor. The ROCK2 pathway modulates inflammatory response and fibrotic processes. ROCK2 inhibition is thought to both restore immune homeostasis and reduce fibrotic processes, which make s Rezurock unique from other pharmacologic treatment options. Approval was based on the phase 2 ROCKstar study.Rezurock (belumosudil) will be considered for coverage when the following criteria are met:Chronic Graft-Versus-Host Disease (cGVHD)For initial authorization: 1. Member is at least 12 years of age; AND 2. Medication must be prescribed by or in consultation with a transplant or hematology/oncology specialist ; AND 3. Member has a diagnosis of cGVHD following allogeneic hematopoietic cell transplant ; AND 4. Member has failed at least 2 prior lines of systemic therapy , i.e., systemic corticosteroid and another systemic treatment (calcineurin inhibitor, Jakafi, mycophenolate mofetil, sirolimus, methotrexate, Imbruvica) ; AND 5. If the member is on a chronic proton pump inhibitor (e.g., omeprazole) , the member must attempt to discontinue it or switch to an alternate agent such as an H2 blocker (e.g. , famotidine). 6. Dosage allowed/Quantity limit: 200 mg orally once daily. (QL: 30 tablets per 30 days). NOTE: Patients who must remain on a proton pump inhibitor will require 200 mg twice daily (and a QL override). If all the above requirements are met , the medication will be approved for 6 months. For reauthorization : 1. Chart notes must show improvement of signs and symptoms of disease in at least 1 organ/site, without progression in any other organ/site. 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 Rezurock (belumosudil) 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/DESCRIPTION09/29/2021 New policy created for Rezurock . 03/07/2024 Updated references. Removed persistent manifestations and added following allogen eic hematopoietic cell transplant. References: 1. Rezurock [prescribing information]. Kadmon Pharmaceuticals, LLC; 2023 . 2. National Comprehensive Cancer Network. Hematopoietic Cell Transplantation (HCT). Version 3.2023. https://www.nccn.org/professionals/physician_gls/pdf/hct.pdf. Accessed March 8, 2024. 3. Jagasia M, Lazaryan A, Bachier CR, et al. ROCK2 Inhibition With Belumosudil (KD025) for the Treatment of Chronic Graft-Versus-Host Disease. JClin Oncol . 2021;39(17):1888-1898. doi:10.1200/JCO.20.02754 4. Wolff D, Fatobene G, Rocha V, Krger N, Flowers ME. Steroid-refractory chronic graft-versus-host disease: treatment options and patient management. Bone Marrow Transplant . 2021;56(9):2079-2087. doi:10.1038/s41409-021-01389-5 5. Penack O, Marchetti M, Aljurf M, et al. Prophylaxis and management of graft-versus-host disease after stem-cell transplantation for haematological malignancies: updated consensus recommendations of the European Society for Blood and Marrow Transplantation. Lancet Haematol . 2024;11(2):e147-e159. doi:10.1016/S2352-3026(23)00342-3 6. Cutler C, Lee SJ, Arai S, et al. Belumosudil for chronic graft-versus-host disease after 2 or more prior lines of therapy: the ROCKstar Study [published correction appears in Blood. 2022 Mar 17;139(11):1772]. Blood . 2021;138(22):2278-2289. doi:10.1182/blood.2021012021 Effective date: 10/01/2024 Revised date: 03/07/2024
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Pyrukynd (mitapivat )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Pyrukynd , approved by the FDA in 2022, is a pyruvate kinase activator indicated for treatment of adults with hemolytic anemia caused by pyruvate kinase (PK) deficiency . PK deficiency is caused by mutations in the PKLR gene that encodes for the PK enzyme. PK catalyzes the production of ATP which is essential for RBC function. Deficiency of PK activity results in inadequate ATP and premature RBC destruction (hemolytic anemia) . Standard t reatments include RBC transfusions and splenectomy. Pyrukynd increases PK activity , addressing the underlying cause of hemolysis .Pyrukynd (mitapivat ) will be considered for coverage when the following criteria are met:Pyruvate K inase (PK) deficiencyFor 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 diagnosis of PK deficiency confirmed by genetic testing (two or more documented mutant PKLR alleles, at least one of which is a missense mutation); AND 4. Member s hemoglobin level is 10 g/dL or less OR member has had a minimum of 6 transfusions in the past 52 weeks ; AND 5. Member is currently taking at least 0.8 mg oral folic acid; AND 6. Member does NOT have any of the following: a) Homozygous R479H mutation or have 2 non-missense mutations, without the presence of another missense mutation, in the PKLR gene b) Moderate to severe hepatic impairment c) Prior bone marrow or stem cell transplant . 7. Dosage allowed/Quantity limit s: a) Starting Dose : 5 mg orally twice daily for the first 4 weeks b) Maintenance Doses: Titrate to 20 mg twice daily, and then to the maximum recommended dose of 50 mg twice daily, with these dose increases occurring every 4 weeks, based on assessments of Hb and transfusion requirement s, as directed in prescribing information. c) Quantity Limit: 5 6 tablets /28 days . 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 hemoglobin increase of at least 1.5 g/dL OR clinically significant decrease in transfusion burden. If all the above requirements are met , the medication will be approved for an additional 6 months.CareSource considers Pyrukynd (mitapivat ) 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/DESCRIPTION 04/16/2022 New policy for Pyrukynd created. 04/23/2024 Updated references. Removed geneticist as prescriber. Corrected QL from 58/28 to 56/28. Removed LFTs from reauth. Corrected hemoglobin from
IN-MED-P -366647a; Issued Date: 6/1/2023 OMPP Approved: 5/16/2023PHARMACY POLICY STATEMENTIndiana Medicaid DRUG NAME Iqirvo ( elafibranor )BENEFIT TYPE Pharmacy STATUS Prior Authorization Required Iqirvo, approved by the FDA in 2024, is a dual peroxisome proliferator-activated receptor (PPAR) agonist indicated for the treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA. PBC is a progressive, autoimmune liver disease that leads to scarring and inflammation of the small bile ducts. It primarily affects women and is characterized by fatigue, pruritis, and jaundice. Antimitochondrial antibody (AMA) is found in 95% of PBC pati ents. Activation of PPAR-alpha and PPAR-delta inhibits bile acid synthesis . Ursodiol (ursodeoxycholic acid [UDCA]) is the first-line treatment for PBC. It improves biochemical indices, delays histologic progression, and improves survival. Accelerated approval of Iqirvo for PBC was based on reduction of alkaline phosphatase (ALP). Improvement in survival or prevention of liver decompensation events have not been demonstrated .Iqirvo (elafibranor) will be considered for coverage when the following criteria are met:Primary Biliary Cholangitis (PBC)For initial authorization: 1. Member is at least 18 years of age ; AND 2. Medication must be prescribed by or in consultation with a hepatologist or gastroenterologist ; AND 3. Member has a diagnosis of PBC confirmed by at least 2 of the following : a) Biochemical evidence of cholestasis based on ALP elevation b) Presence of AMA or other PBC-specific antibodies, including sp100 or gp210 c) Histologic evidence of nonsuppurative cholangitis and destruction of small or medium-sized bile ducts on biopsy; AND 4. Member had an inadequate response to UDCA after 1 year of treatment OR the member has documentation of intolerance to UDCA; AND 5. UDCA will be continued in combination with Iqirvo unless the patient has documented intolerance; AND 6. Member does NOT have any of the following: a) Decompensated cirrhosis (e.g., ascites, variceal bleeding, hepatic encephalopathy ) b) Complete biliary obstruction. 7. Dosage allowed/Quantity limit: 80 mg orally once daily . QL: 30 tablets/30 days. 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 improved (decreased) ALP and/or total bilirubin compared to baseline. If all the above requirements are met , the medication will be approved for an additional 12 months CareSource considers Iqirvo (elafibranor) 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/DESCRIPTION06/20/2024 New policy for Iqirvo created. References: 1. Iqirvo [prescribing information]. Ipsen Biopharmaceuticals, Inc.; 2024. 2. Lindor KD, Bowlus CL, Boyer J, Levy C, Mayo M. Primary Biliary Cholangitis: 2018 Practice Guidance from the American Association for the Study of Liver Diseases . Hepatology. 2019;69(1):394-419. doi:10.1002/hep.30145 3. Lindor KD, Bowlus CL, Boyer J, Levy C, Mayo M. Primary biliary cholangitis: 2021 practice guidance update from the American Association for the Study of Liver Diseases. Hepatology. 2022;75(4):1012-1013. doi:10.1002/hep.32117 4. Hirschfield GM, Dyson JK, Alexander GJM, et al. The British Society of Gastroenterology/UK-PBC primary biliary cholangitis treatment and management guidelines. Gut . 2018;67(9):1568-1594. doi:10.1136/gutjnl-2017-315259 5. European Association for the Study of the Liver. Electronic address: easloffice@easloffice.eu; European Association for the Study of the Liver. EASL Clinical Practice Guidelines: The diagnosis and management of patients with primary biliary cholangitis. JHepatol . 2017;67(1):145-172. doi:10.1016/j.jhep.2017.03.022 Effective date: 01/01/2025 Revised date: 06/20/2024
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