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Enzyme Replacement Therapy for Lysosomal Storage Disorders (MPS I, VI) [Aldurazyme, Naglazyme]

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Enzyme Replacement Therapy for Lysosomal Storage Disorders (MPS I, VI) [Aldurazyme, Naglazyme] Drug and Biologic Coverage Criteria Effective Date: 11/1/2016 Last P&T Approval/Version: 07/28/2021 Next Review Due By: 7/2022 Policy Number: C9997-A Enzyme Replacement Therapy for Lysosomal Storage Disorders (MPS I, VI) [Aldurazyme, Naglazyme] PRODUCTS AFFECTED Aldurazyme SOLN 2.9MG/5ML, Naglazyme SOLN 1MG/ML COVERAGE POLICY Coverage for services, procedures, medical devices, and drugs are dependent upon benefit eligibility as outlined in the member's specific benefit plan. This Coverage Guideline must be read in its entirety to determine coverage eligibility, if any. This Coverage Guideline provides information related to coverage determinations only and does not imply that a service or treatment is clinically appropriate or inappropriate. The provider and the member are responsible for all decisions regarding the appropriateness of care. Providers should provide Molina Healthcare complete medical rationale when requesting any exceptions to these guidelines Documentation Requirements: Molina Healthcare reserves the right to require that additional documentation be made available as part of its coverage determination; quality improvement; and fraud; waste and abuse prevention processes. Documentation required may include, but is not limited to, patient records, test results and credentials of the provider ordering or performing a drug or service. Molina Healthcare may deny reimbursement or take additional appropriate action if the documentation provided does not support the initial determination that the drugs or services were medically necessary, not investigational or experimental, and otherwise within the scope of benefits afforded to the member, and/or the documentation demonstrates a pattern of billing or other practice that is inappropriate or excessive DIAGNOSIS: Naglazyme (galsulfase): indicated for Mucopolysaccharidosis VI (MPS VI); Maroteaux-Lamy syndrome). Naglazyme has been shown to improve walking and stair-climbing capacity. Aldurazyme (laronidase): indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. Limitations of Use: The risks and benefits of treating mildly affected patients with the Scheie form have not been established. E76.01 Hurler’s syndrome E76.02 Hurler-Scheie syndrome E76.03 Scheie’s syndrome E76.29 Other mucopolysaccharidoses REQUIRED MEDICAL INFORMATION: A. MUCOPOLYSACCHARIDOSIS: 1. Diagnosis of Mucopolysaccharidosis I (MPS I) or Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy Syndrome) confirmed by: Documented reduced fibroblast or leukocyte IDUA or ARSB enzyme activity OR Molecular genetic testing of IDUA or ARSB Molina Healthcare, Inc. confidential and proprietary © 2021 This document contains confidential and proprietary information of Molina Healthcare and cannot be reproduced, distributed, or printed without written permission from Molina Healthcare. This page contains prescription brand name drugs that are trademarks or registered trademarks of pharmaceutical manufacturers that are not affiliated with Molina Healthcare. Page 1 of 6 Drug and Biologic Coverage Criteria [Documentation required] AND 2. Documentation that member has at least ONE (1) of the following symptoms of the disease: gait disturbance, growth deficiency, short stature, spinal abnormalities, chest abnormalities, joint abnormalities, respiratory compromise, cardiac valve abnormalities, muscular weakness, visual impairment, hearing loss, or dental abnormalities and oral health challenges AND 3. Prescriber attests that the prescribed ERT will be used as monotherapy: NOT to be used concurrently with other medications for Mucopolysaccharidosis AND 4. Documented baseline values of the following: a) Member’s weight dated within 1 month of the prior authorization request AND b) Urinary glycosaminoglycan (GAG)levels. AND c) Members 6 years of age or older (one of the following): 6-minute walk test (6-MWT) AND/OR percent predicted Forced Vital Capacity (% Predicted FVC) OR Members 3 months to less than 6 years (one of the following): upper airway obstruction during sleep, cardiac status, growth velocity, mental development, FVC, and/or 6-minute walk test NOTE: Member’s weight must be provided at time of prior authorization request and for any subsequent dose increases. Requests for amounts above initially authorized limits will require documentation of an updated member weight for review and authorization. CONTINUATION OF THERAPY: A. MUCOPOLYSACCHARIDOSIS: 1. Prescriber attests (or medical records support) that requested ERT remains for use as monotherapy: NOT to be used concurrently with other MPS drug therapy AND 2. Adherence to therapy at least 85% of the time as verified by Prescriber and member’s medication fill history (review Rx history for compliance), including: Adherent to the prescribed medication regimen, Tolerance to therapy AND No severe adverse reactions or drug toxicity AND 3. Documentation of positive response or disease stability to therapy as compared to baseline (prior to therapy): a) Decreased urinary glycosaminoglycan (GAG) levels AND b) Members 6 years of age or older (one of the following): 6-minute walk test (6- MWT) AND/OR Percent predicted Forced Vital Capacity (% Predicted FVC) OR c) Members 3 months to less than 6 years (one of the following): decreased hepatosplenomegaly improvement in upper airway obstruction during sleep, cardiac status, growth velocity, mental development, FVC, and/or 6-minute walk test 4. Prescriber attests (or medical records support) the absence of severe adverse events or unacceptable toxicity from the drug [e.g., hypersensitivity reactions, anaphylaxis, severe type III immune-mediated reactions (e.g., membranous glomerulonephritis) have occurred] DURATION OF APPROVAL: Initial authorization: 12 months, Continuation of therapy: 12 months Molina Healthcare, Inc. confidential and proprietary © 2021 This document contains confidential and proprietary information of Molina Healthcare and cannot be reproduced, distributed, or printed without written permission from Molina Healthcare. This page contains prescription brand name drugs that are trademarks or registered trademarks of pharmaceutical manufacturers that are not affiliated with Molina Healthcare. Page 2 of 6 Drug and Biologic Coverage Criteria PRESCRIBER REQUIREMENTS: Prescribed by, or in consultation with, a board-certified geneticist, pediatric metabolic specialist, hematologist, or physician experienced in the management of mucopolysaccharidoses (MPS). Submit consultation notes if applicable. Consultation notes must be submitted for initial request AND at least once annually for continuation of treatment requests. AGE RESTRICTIONS: Aldurazyme (laronidase): 6 months of age to 65 years of age Naglazyme (galsulfase): 3 months of age to 29 years of age QUANTITY: Aldurazyme (laronidase) MPS, Type I (Hurler and Hurler-Scheie forms) and Scheie form with moderate to severe symptoms Usual dosage: 0.58 mg/kg of body weight as an IV infusion once a week Only a 1-month supply may be dispensed at a time Naglazyme (galsulfase) MPS, Type VI (Maroteaux-Lamy syndrome) Usual dosage: 1 mg/kg IV once weekly infused over a period of at least 4 hours; initial infusion rate (250 mL infusion) 6 mL/hr for first hour; may increase rate to 80 mL/hr for remaining 3 hours Only a1-month supply may be dispensed at a time PLACE OF ADMINISTRATION: The recommendation is that infused medications in this policy will be for pharmacy or medical benefit coverage administered in a place of service that is a non-hospital facility-based location as per the Molina Health Care Site of Care program. Note: Site of Care Utilization Management Policy applies for Aldurazyme and Naglazyme. For information on site of care, see: https://www.molinamarketplace.com/marketplace/-/media/Molina/PublicWebsite/PDF/Common/Specialty- Medication-Administration-Site-of-Care-Coverage-Criteria-Policy_v2 DRUG INFORMATION ROUTE OF ADMINISTRATION: Intravenous DRUG CLASS: Mucopolysaccharidosis I (MPS I) - Agents FDA-APPROVED USES: Naglazyme (galsulfase): indicated for Mucopolysaccharidosis VI (MPS VI); Maroteaux-Lamy syndrome). Naglazyme has been shown to improve walking and stair-climbing capacity. Aldurazyme (laronidase): indicated for patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms. The risks and benefits of treating mildly affected patients with the Scheie form have not been established. E76.01 Hurler’s syndrome E76.02 Hurler-Scheie syndrome E76.03 Scheie’s syndrome E76.29 Other mucopolysaccharidoses Molina Healthcare, Inc. confidential and proprietary © 2021 This document contains confidential and proprietary information of Molina Healthcare and cannot be reproduced, distributed, or printed without written permission from Molina Healthcare. This page contains prescription brand name drugs that are trademarks or registered trademarks of pharmaceutical manufacturers that are not affiliated with Molina Healthcare. Page 3 of 6 Drug and Biologic Coverage Criteria COMPENDIAL APPROVED OFF-LABELED USES: None APPENDIX APPENDIX: • Forced vital capacity (FVC): In the clinical trials of Aldurazyme in patients ≥ 6 years of age, the mean increase in percent of predicted forced vital capacity (FVC) observed corresponded to a 10% relative improvement over the baseline FVC, which is considered by the American Thoracic Society to be a clinically significant change and not due to
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