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Horizon BCBSNJ
Uniform Medical Policy ManualSection:Drugs
Policy Number:027
Effective Date: 09/11/2020
Original Policy Date:12/19/2003
Last Review Date:09/08/2020
Date Published to Web: 10/10/2018
Subject:
Laronidase (Aldurazyme), Galsulfase (Naglazyme), and Idursulfase (Elaprase), Elosulfase alfa (Vimizim), and Vestronidase alfa (Mepsevii)

Description:
_______________________________________________________________________________________

IMPORTANT NOTE:

The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.

Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.

__________________________________________________________________________________________________________________________

Mucopolysaccharidoses (MPS) are a rare group of inherited storage disorders that are caused by the deficiency of specific lysosomal enzymes required for catabolism of glycosaminoglycans, which are long chains of carbohydrates. Glycosaminoglycans are used to help build bone, cartilage, tendons, corneas, skin, and connective tissues. There are seven different types of MPS discovered and they include:
  • MPS I has a wide spectrum of clinical severity and has been subdivided into three phenotypes: Hurler Syndrome (severe), Hurler-Scheie syndrome (intermediate), and Scheie syndrome (mild). This disease is caused by a deficiency of a-L-iduronidase enzyme. Laronidase (Aldurazyme) is FDA approved for this condition.
  • MPS II or Hunter syndrome is an X-linked recessive disease caused by insufficient levels of lysosomal enzyme iduronate-2-sulfatase. There are 2 clinical subtypes of Hunter syndrome: MPS IIA and MPS IIB. Idursulfase (Elaprase) is FDA approved for this condition.
  • MPS III or Sanfilippo syndrome is caused by deficient enzyme heparin N-sulfatase (Sanfilippo A), alpha-N- acetylglucosaminidase (Sanfilippo B), acetyl-CoAlpha-glucosaminide acetyltransferase (Sanfilippo C), or N-acetylglucosamine 6-sulfatase (Sanfilippo D).
  • MPS IV or Morquio syndrome has two subtypes as a result of deficient enzymes N-acetylgalactosamine 6-sulfatase (Type A) or beta-galactosidase (Type B). Elosulfase alfa (Vimizim) is FDA approved to for patients with MPS IVA or Morquio A syndrome.
  • MPS VI or Maroteaux Lamy is characterized by the absence or marked reduction in N-acetylgalactosamine 4-sulfatase. Galsulfase (Naglazyme) is FDA approved for this condition.
  • MPS VII or Sly syndrome is deficiency in enzyme beta-glucuronidase. Vestronidase alfa (Mepsevii) is FDA approved for this condition.

[INFORMATIONAL NOTE:
The FDA-approved package inserts have the following BOXED WARNINGS or warnings & precautions:
    • Laronidase (Aldurazyme): Risk of anaphylaxis. Life-threatening anaphylactic reactions have been observed in some patients during laronidase infusions. Therefore, appropriate medical support should be readily available when laronidase is administered. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions, and require additional monitoring.
    • Idursulfase (Elaprase): Risk of anaphylaxis. Life-threatening anaphylactic reactions have been observed in some patients during idursulfase infusions. Therefore, appropriate medical support should be readily available when idursulfase is administered. Biphasic anaphylactic reactions have also been observed after idursulfase administration and patients who have experienced anaphylactic reactions may require prolonged observation. Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions, and require additional monitoring.
    • Elosulfase alfa (Vimizim): Risk of anaphylaxis. Life-threatening anaphylactic reactions have occurred in some patients during Vimizim infusions. Anaphylaxis, presenting as cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, and gastrointestinal symptoms in conjunction with urticaria, have been reported to occur during infusions, regardless of duration of the course of treatment. Closely observe patients during and after Vimizim administration and be prepared to manage anaphylaxis. Inform patients of the signs and symptoms of anaphylaxis and have them seek immediate medical care should symptoms occur. Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring
    • The FDA-approved package insert for Naglazyme (galsufase) has warnings and precautions regarding the risk of anaphylaxis, allergic reactions, acute cardiorespiratory failure, and acute respiratory complications. Caution should be exercised when administering Naglazyme to patients susceptible to fluid volume overload, and appropriate respiratory support should be available during infusion.
    • Vestronidase alfa (Mepsevii): Risk of anaphylaxis: Anaphylaxis has occurred with vestronidase alfa administration, as early as the first dose, therefore appropriate medical support should be readily available when vestronidase alfa is administered. Closely observe patients during and for 60 minutes after vestronidase alfa infusion. Immediately discontinue the vestronidase alfa infusion if the patient experiences anaphylaxis.]

Policy:
[(NOTE: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance.)

The requirements of the Horizon BCBSNJ Laronidase (Aldurazyme), Galsulfase (Naglazyme), and Idursulfase (Elaprase), elosulfase alfa (Vimizim) and vestronidase alfa (Mepsevii) Program may require a precertification/prior authorization via MagellanRx Management. These requirements are member-specific: please verify member eligibility and requirements through the Horizon Provider Portal (www.horizonblue.com/provider). Ordering clinicians should request pre-certification from MagellanRx Management at ih.magellanrx.com or call 1-800-424-4508 (when applicable).]
1. Please refer to a separate policy on Site of Administration for Infusion and Injectable Prescription Medications (Policy #142) under the Drug Section.

2. The following drugs are considered medically necessary based on the FDA-approved product information:
    • Laronidase (Aldurazyme) for members with documented diagnosis of Hurler or Hurler-Scheie forms of MPS I OR for those with the Scheie form of MPS I who have moderate to severe symptoms
      • Diagnosis is confirmed by (documentation of medical records required):
        • Detection of pathogenic mutations in the IDUA gene by molecular genetic testing; or
        • Deficiency of alpha-L-iduronidase activity in fibroblasts, leukocytes, serum, or blood spots; AND
      • Member is 6 months of age or older; AND
      • Member has absence of severe cognitive impairment; AND
      • Baseline value for urinary glycosaminoglycan (uGAG) (documentation of medical records required); AND
      • Baseline values for one or more of the following (documentation of medical records required):
        • Members 6 years or greater: percent predicted forced vital capacity (FVC), 6-minute walk test, joint range of motion, left ventricular hypertrophy, growth, quality of life (CHAQ/HAQ/MPS HAQ); or
        • Members 6 months to less than 6 years: cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC, and/or 6-minute walk test; AND
      • The prescriber is a specialist (e.g. endocrinologist, geneticist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
    [INFORMATIONAL NOTE: The quality of life scales are as defined follows:
    • MPS HAQ: MPS Health Assessment Questionnaire
      • Used to evaluate the functional capabilities and performance in children and adults with MPS
      • 10-point Likert Scale– 0 to 10 with higher scores representing greater difficulty performing the basic activities of daily living
      • Consists of Self Care Domain and Proficiency Scores ( Eating/Drinking – Dressing – Bathing – Grooming – Toileting) Mobility Domain and Proficiency Scores(Transfers – Walking – Stairs) Caregiver Assistance Domain Score
    • CHAQ:Childhood Health Assessment Questionnaire
      • To measure functional status in children diagnoses with Juvenile Idiopathic Arthritis
      • The CHAQ is composed of disability and discomfort indexes.
        • Disability index measures functional ability in eight activities of daily living; dressing and grooming, arising, eating, walking, hygiene, reach, grip and activities. Three components are assessed in each area: 1) the degree to which daily functions were difficult to perform; 2) the use of special aids and devices; and 3) activities for which the assistance of another person was required. Each question had a four level scale between 0 (no difficulty), 1 (with some difficulty), 2 (with much difficulty) and 3 (unable to perform).
    • HAQ:Health Assessment Questionnaire
      • The HAQ is based on five patient-centered dimensions: disability, pain, medication effects, costs of care, and mortality
      • Patients report that they want: 1) to avoid disability; 2) to be free of pain and discomfort; 3) to avoid adverse effects of treatment; 4) to keep medical costs low; 5) and to postpone death]
      • Idursulfase (Elaprase) for members with documented diagnosis of Hunter syndrome (MPS II)
        • Diagnosis is confirmed by (documentation of medical records required):
          • Deficiency of iduronidate 2-sulfatase activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase; or
          • Detection of pathogenic mutations in the IDS gene by molecular genetic testing; AND
        • Member is 16 months of age or older; AND
        • Member has absence of severe cognitive impairment; AND
        • Baseline value for urinary glycosaminoglycan (uGAG) (documentation of medical records required); AND
        • Baseline values for one or more of the following (documentation of medical records required):
            • Members 5 years or greater: 6-minute walk test (6-MWT) and/or percent predicted forced vital capacity (FVC), joint range of motion, left ventricular hypertrophy, quality of life (CHAQ/HAQ/MPS HAQ); or
            • Members < 5 years: spleen volume, liver volume, FVC, and/or 6-minute walk test; AND
        • The prescriber is a specialist (e.g. endocrinologist, geneticist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
      • Elosulfase alfa (Vimizim) for members with documented diagnosis of MPS IVA (Morquio A syndrome)
        • Diagnosis is confirmed by (documentation of medical records required):
            • Absence or marked reduction in N-acetylgalactosamine 6-sulfatase (GALNS) enzyme activity; or
            • Sequence analysis and/or deletion/duplication analysis of the GALNS gene for biallelic mutation; AND
        • Member is 5 years of age or older; AND
        • Baseline values for one or more of the following (documentation of medical records required): 6-minute walk test (6-MWT), timed 25-foot walk test (T25FW) and/or percent predicted forced vital capacity (FVC); AND
        • The prescriber is a specialist (e.g. endocrinologist, geneticist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
      • Galsulfase (Naglazyme) for members with documented diagnosis of MPS VI
        • Patient aged 5 years or older; AND
        • Diagnosis is confirmed by (documentation of medical records required):
          • Detection of pathogenic mutations in the ARSB gene by molecular genetic testing; or
          • Arylsulfatase B (ASB) enzyme activity of <10% of the lower limit of normal in cultured fibroblasts or isolated leukocytes; and
            • Member has normal enzyme activity of a different sulfatase (excluding Multiple Sulfatase Deficiency [MSD]); and
            • Member has an elevated urinary GAG level as defined as being above the upper limit of normal by the reference laboratory; AND
        • Baseline value for urinary glycosaminoglycan (uGAG) (documentation of medical records required); AND
        • Baseline values for one or more of the following (documentation of medical records required):
            • 12-minute walk test (12-MWT) , FEV1 and/or 3-minute stair climb test; AND
        • The prescriber is a specialist (e.g. endocrinologist, geneticist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis
      • Vestronidase alfa (Mepsevii) for members with documented diagnosis of MPS VII (Sly syndrome)
        • Diagnosis is confirmed by BOTH of the following (documentation of medical records required):
          • Beta-glucuronidase enzyme deficiency in peripheral blood leukocytes; and
          • Detection of pathogenic mutations in the GUSB gene by molecular genetic testing; AND
        • Member is 5 months of age or older; AND
        • Baseline value for one or more of the following (documentation of medical records required):
            • Six minute walk test (6MWT), motor function [i.e., Bruininks-Oseretsky Test of Motor Proficiency (BOT-2)], liver and/or spleen volume, urinary excretion of glycosaminoglycans (GAGs) such as chondroitin sulfate and dermatan sulfate, skeletal involvement, pulmonary function tests, shoulder flexion, visual acuity, etc.; AND
        • The prescriber is a specialist (e.g. endocrinologist, geneticist) or the prescriber has consulted with a specialist in the area of the patient’s diagnosis

    [INFORMATIONAL NOTE:
      • In clinical trials, Aldurazyme reduced hepatosplenomegaly, the number of apnea and hypopnea episodes during sleep, progression of heart failure or cor pulmonale, and urinary GAG excretion. The rate of growth in height and weight had increased, along with mean maximal range of motion of shoulder flexion and elbow extension. It has been reported that laronidase improves pulmonary function and functional capacity as well. In addition, NYHA functional class improved by one or two classes. The medical literature suggests that Aldurazyme reduces lysosomal storage of glycoaminoglycans in the liver and ameliorates some clinical manifestations of the disease. Aldurazyme has not been evaluated for effects on the central nervous system manifestations of the disorder. The safety and effectiveness of ALDURAZYME was assessed in a 52-week, open-label, uncontrolled clinical study in 20 patients with MPS I, ages 6 months to 5 years old, and was found to be similar to the safety and effectiveness of ALDURAZYME in pediatric patients 6 to 18 years, and adults.
      • In clinical trials, individuals on idursulfase showed improvement in 6-minute walk test capacity, improvement in percent predicted forced vital capacity, and reduced organ size. The urinary glycoaminoglycans were markedly reduced in the idursulfase treatment arm. In patients 16 months to 5 years of age, there is no data available to demonstrate improvement in disease-related symptoms or long term clinical outcome; however, treatment with idursulfase has reduced spleen volume similarly to that of adults and children 5 years of age and older. The safety and efficacy of idursulfase have not been established in pediatric patients less than 16 months of age.
      • In clinical trials, individuals on galsulfase showed improvement in 12-minute walk test and stair climbing capacity compared to members on placebo. Urinary glycoaminoglycans levels decreased in members receiving galsulfase. The safety and efficacy have not been established in children less than 5 years.
      • In clinical trials, elosulfase alfa demonstrated improvement in the 6-minute walk test compared to placebo. The reduction in urinary keratin sulfate (KS) levels from baseline was greater in patients receiving elosulfase alfa. The relationship between urinary KS and other measures of clinical response has not been established. The safety and efficacy have not been established in children less than 5 years. Use of Vimizim in patients 5 years of age and older is supported by an adequate and well-controlled trial in pediatric and adult patients. Clinical trials with Vimizim were conducted in 176 patients (median age 12 years, range 5 to 57 years old) with the majority of patients in the pediatric age group (53% aged 5 to 11 years, 27% aged 12 to 17 years).
      • In clinical trials, vestronidase alfa was studied in 12 subjects with MPS VII aged 5 to 35 years. At 24 weeks there was a reduction in urinary glycosaminoglycans (GAGs), improvements in Multi-domain Responder Index (MDRI) score with 6 of 12 subjects having improvements of +1 or more, reduction in fatigue, and an increase in 6 minute walking test in those able to complete this assessment. The safety and efficacy of vestronidase alfa has not been established in pediatric patients less than 18 years of age.]

    3. When these drugs are medically necessary, initial will be eligible for a period of 1 year at the following FDA recommended doses:
      • Laronidase (Aldurazyme) - 0.58 mg/kg body weight infused once weekly as an IV infusion.
      • Idursulfase (Elaprase) – 0.5 mg/kg body weight infused once weekly as IV infusion.
      • Galsulfase (Naglazyme) – 1 mg/kg body weight infused once weekly as IV infusion.
      • Elosulfase alfa (Vimizim) – 2 mg/kg body weight infused once weekly as IV infusion.
      • Vestronidase alfa (Mepsevii) – 4 mg/kg body weight infused once every two weeks as an IV infusion
    4. Continued therapy will be approved annually if the following criteria are met:
      • Absence of unacceptable toxicity (severe hypersensitivity reactions, acute respiratory complications, acute cardiorespiratory failure, severe infusion reactions, etc.) from the drug and the patient has demonstrated a beneficial response to therapy compared to pretreatment baseline as shown by the following:
          • Laronidase (Aldurazyme)
            • Patient does not have progressive/irreversible severe cognitive impairment; and
            • There is a reduction in uGAG levels from baseline and improvement/stabilization in one or more of the baseline tests (eg 12MWT, FEV1, etc) (documentation of medical records required).
          • Idursulfase (Elaprase)
            • Patient does not have progressive/irreversible severe cognitive impairment; and
            • There is a reduction in uGAG levels from baseline and improvement/stabilization in one or more of the baseline tests (eg 12MWT, FEV1, etc) (documentation of medical records required).
          • Elosulfase alfa (Vimizim)
            • Patient has shown a response to therapy as evidenced by one or more of the following markers (documentation of medical records required):
              • Stability or improvement on endurance tests
              • Stability or improvement in pulmonary function tests
          • Galsulfase (Naglazyme)
            • Patient does not have progressive/irreversible severe cognitive impairment
            • There is a reduction in uGAG levels from baseline and improvement/stabilization in one or more of the baseline tests (eg 12MWT, FEV1, etc) (documentation of medical records required).
          • Vestronidase alfa (Mepsevii):
            • Patient has responded to therapy compared to pre-treatment baseline in one or more of the following (documentation of medical records required):
                • Stability or improvement in 6MWT, shoulder flexion, visual acuity, and/or other motor functions
                • Reduction in liver and/or spleen volume
                • Reduction in urinary excretion of GAGs
                • Stability of skeletal disease
                • Stability or improvement in pulmonary function tests


    [INFORMATIONAL NOTE:
      • It is recommended that laronidase be pretreated with antipyretics and/or antihistamines 60 minutes prior to the start of infusion to reduce the risk of infusion reactions.
      • For laronidase, if anaphylactic or other severe allergic reactions occur, immediately discontinue the infusion and initiate appropriate medical treatment. Caution should be exercised if epinephrine is being considered for use in patients with MPS I due to the increased prevalence of coronary artery disease in these patients. Interventions have included resuscitation, mechanical ventilator support, emergency tracheotomy, hospitalization, and treatment with inhaled beta-adrenergic agonists, epinephrine, and IV corticosteroids.
      • It is recommended that galsulfase be pretreated with antipyretics and/or antihistamines 30 to 60 minutes prior to the start of infusion to reduce the risk of infusion reactions.
      • It is recommended that elosulfase alfa be pretreated with antihistamines with or without antipyretics 30 to 60 minutes prior to the start of infusion to reduce the risk of infusion reactions
      • It is recommended that vestronidase alfa be pretreated with non-sedating antihistamine with or without an anti-pyretic medication 30 to 60 minutes prior to the start of infusion ]

    5. Other uses of laronidase (Aldurazyme), idursulfase (Elaprase), galsulfase (Naglazyme), elosulfase alfa (Vimizim), and vestronidase alfa (Mepsevii) are considered investigational.

    Medicare Coverage:
    There is no National Coverage Determination (NCD or Local Coverage Determination (LCD) for jurisdiction JL for this service. Therefore, Medicare Advantage Products will follow the Horizon BCBSNJ Medical Policy.

    **Note: Bullet 1 of the policy section referring to Site of Administration for Infusion and Injectable Prescription Medications (Policy #142) does not apply for Medicare Advantage Products.

    Medicaid Coverage:
    For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf

    ________________________________________________________________________________________

    Horizon BCBSNJ Medical Policy Development Process:

    This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.

    ___________________________________________________________________________________________________________________________

    Index:
    Laronidase (Aldurazyme), Galsulfase (Naglazyme), Idursulfase (Elaprase), Elosulfase alfa (Vimizim), and Vestronidase alfa (Mepsevii)
    Laronidase (Aldurazyme)
    Aldurazyme (Laronidase)
    Idursulfase (Elaprase)
    Elaprase (Idursulfase)
    Elosulfase alfa (Vimizim)
    Vimizim (Elosulfase alfa)
    Galsulfase (Naglazyme)
    Naglazyme (Galsulfase)
    Vestronidase alfa (Mepsevii)
    Mepsevii (Vestronidase alfa)

    References:

    1. Aldurazyme Product Information. BioMarin Pharmaceutical Inc./Genzyme Corp. April 2013.
    2. Kakkis ED, Muenzer J, Tiller GE, et al. Enzyme-Replacement Therapy in Mucopolysaccharidosis I. N Engl J Med 2001;344:182-88.
    3. Genzyme General Therapeutics. Mucopolysaccharidosis I: A Complex Multisystemic Lysosomal Storage Disorder. Genzyme Corporation. 2002.
    4. Genzyme General Therapeutics. Mucopolysaccharidosis I: An overview of symptoms, diagnosis, and management. Genzyme Corporation. 2003.
    5. Naglazyme Product Information. BioMarin Pharmaceutical Inc. March 2013.
    6. Elaprase Product Information. Shire Human Genetic Therapies, Inc. November 2018.
    7. Giugliani R, Harmatz P, Wraith JE. Management guidelines for mucopolysaccharidosis VI. Pediatrics. 2007;120:405‐418
    8. Harmatz P, Giugliani R, Schwartz I, et al. Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human n-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase b or rhasb) and follow-on, open-label extension study. J Pediatr 2006;148:533-539.
    9. Harmatz P, Whitley CB, Waber L, et al. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr 2004;144:574-580.
    10. Harmatz P, Ketteridge D, Giugliani R, et al. Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-acetylgalactosamine 4-sulfatase. Pediatrics 2005;115(6):681-689.
    11. ClinicalTrials.gov [7/2008]. Available at: http://www.clinicaltrials.gov
    12. Pastores GM. Laronidase (Aldurazyme): enzyme replacement therapy for mucopolysaccharidosis type I. Expert Opin Biol Ther. 2008 Jul;8(7):1003-9.
    13. Wraith JE. The first 5 years of clinical experience with laronidase enzyme replacement therapy for mucopolysaccharidosis I. Expert Opin Pharmacother. 2005 Mar;6(3):489-506.
    14. Wilcox WR. Lysosomal storage disorders: the need for better pediatric recognition and comprehensive care. J Pediatr. 2004;144(5 Suppl):S3–S14.
    15. Vimizim Product Information. BioMarin Pharmaceutical Inc. February 2014.
    16. Muenzer J, Wraith JE, Clarke LA, et al. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009 Jan;123(1):19-29.
    17. Wang RY, Bodamer OA, Watson MS, et al. Lysosomal storage diseases: diagnostic confirmation and management of presymptomatic individuals. Genet Med. 2011 May;13(5):457-84.
    18. Tomatsu S, Yasuda E, Patel P, et al. Morquio A syndrome: diagnosis and current and future therapies. Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:141-51.
    19. Mepsevii Product Information. UltraGenyx Pharmaceuticals. December 2019.
    20. A Phase 3 study of UX03 rhGUS Enzyme Replacement Therapy in Patients with MPS 7. Clinicaltrial.gov. Accessed November 29 2017. Available at: https://clinicaltrials.gov/ct2/show/NCT02230566?term=NCT02230566&rank=1
    21. Hartmatz P. Whitley CB, et. Al. A novel, Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 study to assess the efficacy and safety of UX003 (rhGUS) Enzyme Replacement Therapy in Patients with MPS VII.
    22. Martins AM, Dualibi AP, Norato D, et al. Guidelines for the management of mucopolysaccharidosis type I. J Pediatr. 2009;155(4 Suppl):S32-46.
    23. Scarpa M, Almássy Z, Beck M, et al. Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease. Orphanet J Rare Dis. 2011;6:72.
    24. Orphanet J Rare Dis. 2011;6:72.
    25. Clark LA. Mucopolysaccharidosis Type I. GeneReviews. www.ncbi.nlm.nih.gov/books/NBK1162/
    26. Giugliani R, Villareal MLS, Valdez CAA, et al. Guidelines for diagnosis and treatment of Hunter Syndrome for clinicians in Latin America. Genet Mol Biol. 2014 Jun; 37(2): 315–329.
    27. “Mucopolysaccharidosis Type VII.” NORD (National Organization for Rare Disorders), rarediseases.org/rare-diseases/sly-syndrome/.
    28. "Mucopolysaccharidosis Type VII | Genetic And Rare Diseases Information Center (GARD) – An NCATS Program". Rarediseases.Info.Nih.Gov, 2019, https://rarediseases.info.nih.gov/diseases/7096/mucopolysaccharidosis-type-vii. Accessed 6 Jan 2019.
    29. Martins AM, Dualibi AP, Norato D, et al. 1.Guidelines for the Management of Mucopolysaccharidosis Type I. JPeds. 155 (4): S32 - S46.
    30. Table 11, Descriptive Summary of MPS HAQ Domain Scores at Baseline and Week 24 - Elosulfase Alfa (Vimizim) - NCBI Bookshelf.” Current Neurology and Neuroscience Reports., U.S. National Library of Medicine, www.ncbi.nlm.nih.gov/books/NBK362596/table/T18/.
    31. Hendriksz CJ, Berger KI, Lampe C, et al. Health-related quality of life in mucopolysaccharidosis: looking beyond biomedical issues. Orphanet J Rare Dis. 2016 Aug 26;11(1):119. https://www.pediatrics.umn.edu/sites/pediatrics.umn.edu/files/health-related_quality_of_life_in_mucopolysaccharidosis_looking_beyond_biomedical_issues.pdf.
    32. Gümüº, Duygu. "Evaluation Of Childhood Health Assessment Questionnaire In Juvenile Idiopathic Arthritis: A Single Center Experience From Turkey". Archives Of Rheumatology, vol 30, no. 1, 2015, pp. 57-62. Baycinar Tibbi Yayincilik, doi:10.5606/archrheumatol.2015.5130.
    33. Bruce, B, and J F Fries. “The Health Assessment Questionnaire (HAQ).” Current Neurology and Neuroscience Reports., U.S. National Library of Medicine, www.ncbi.nlm.nih.gov/pubmed/16273780.
    34. Muenzer J, Bodamer O, Burton B, et al. The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. Eur J Pediatr. 2012 Jan;171(1):181-.
    35. Burrow T, Leslie ND. Review of the use of idursulfase in the treatment of mucopolysaccharidosis II. Biologics. 2008 Jun; 2(2): 311–320.
    36. Regier DS, Oetgen M, Tanpaiboon P. Mucopolysaccharidosis Type IVA. 2013 Jul 11 [Updated 2016 Mar 24]. In: Adam MP, Ardinger HH, Pagon RA, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2019. Available from: https://www.ncbi.nlm.nih.gov/books/NBK148668/
    37. Clinicaltrials.gov. A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7). NCT02230566. Available at: https://clinicaltrials.gov/ct2/show/NCT02230566
    38. Clinicaltrials.gov. An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7). NCT01856218. Available at: https://clinicaltrials.gov/ct2/show/NCT01856218


    Codes:
    (The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)

    CPT*

    HCPCS
      J1458
      J1743
      J1931
      J1322
      J3397

    * CPT copyright only 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.

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    Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.

    The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy

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