Coverage Policies

Effective Date:

a)    This policy will apply to all services performed on or after the above revision date which will become the new effective date.

b)    For all services referred to in this policy that were performed before the revision date, contact customer service for the rules that would apply.

There are more than 40 diseases classified as lysosomal storage disorders LSDs, each caused by a problem with a particular enzyme in the body. While individually each of these diseases is relatively rare, grouped together they affect 1 in about every 7,700 babies born.

Several new therapies are available for the treatment of inherited forms of lysosomal storage disorders (LSDs). These treatments offer varying abilities for results. Some are not covered. Covered treatment requires preauthorization. This treatment would include:

·         Aldurazyme (laronidase)

·         Ceredase (alglucerase)

·         Cerezyme (imiglucerase)

·         Fabrazyme (agalsidase beta)

·         Myozyme (alglucosidase Alfa)

·         Lumizyme (alglucosidase Alfa)

·         Naglazyme (galsulfase)

·         Elaprase (idursulfase)

·         VPRIV (velaglucerase alfa)

·         Elelyso (taliglucerase alfa)

·         Kanuma (sebelipase alfa)

·         Nexviazyme (avalglucosidase alfa-ngpt)

All medications used in Lysosomal storage disorder therapy require preauthorization and will be approved based on the following criteria:

1. Alglucerase (Ceredase) and imiglucerase (Cerezyme)
   1. Alglucerase (Ceredase) and imiglucerase (Cerezyme) are considered by QualChoice to be medically necessary for children and adolescents less than 18 years of age who are diagnosed with Type 1 Gaucher disease (E75.22)
      
      
   2. Alglucerase (Ceredase) and imiglucerase (Cerezyme) are considered medically necessary for adults with Type 1 Gaucher disease (E75.22) who have any of the following signs and symptoms:
      1. Thrombocytopenia (platelet count less than or equal to 120,000/mm3); or
      2. Moderate to severe anemia (hemoglobin less than or equal to 11.5 g/dL (adult women) or 12.5 g/dL (adult men) or less than or equal to 1.0 g/dL or more below the lower limit of normal for age and sex); or
      3. Skeletal disease beyond mild osteopenia and Erlenmeyer flask deformity; or
      4. Significant hepatomegaly (liver size 1.25 or more times normal (1750 cc in adults)) or splenomegaly (spleen size 5 or more times normal (875 cc in adults)); or
      5. Symptomatic disease, including abdominal or bone pain, fatigue, exertional limitation, weakness, or cachexia.

1. Laronidase (Aldurazyme)

Laronidase (Aldurazyme) is considered medically necessary for members diagnosed with Hurler (E76.01) and Hurler-Scheie (E76.02) forms of mucopolysacchararidoses I (MPS I) and for members diagnosed with the Scheie (E76.03) form who have moderate to severe symptoms.

1. Agalsidase Beta (Fabrazyme)

Agalsidase beta is considered medically necessary for use in members diagnosed with Fabry disease (E75.21).

1. Galsulfase (Naglazyme)

Galsulfase is considered medically necessary for the treatment of members with mucopolysaccharidosis VI (MPS VI) (E76.29).
 

1. Alglucosidase Alfa (Myozyme and Lumizyme)

Alglucosidase alfa is considered medically necessary for the treatment of members with infantile-onset Pompe disease (E74.02).

VI.       Idursulfase (Elaprase)

Elaprase is considered medically necessary for the treatment of Hunter Syndrome (Mucopolysaccharidosis II, MPS II) (E76.1)

VII.      Velaglucerase (VPRIV)

     Velaglucerase is considered medically necessary for the treatment of

     Gaucher’s disease (E75.22) in patients over 4 years of age

VIII.     Taliglucerase Alfa (Elelyso)

Taliglucerase is considered medically necessary for the treatment of   Gaucher’s disease (E75.22) in adults and adolescents.

IX.       Sebelipase alfa (Kanuma)

                Sebelipase alfa is considered medically necessary for the treatment of liposomal acid lipase (LAL) deficiency (Wolman Disease)

X.        Avalglucosidase Alfa-ngpt (Nexviazyme)

            Avalglucosidase alfa-ngpt is considered medically necessary for the treatment of late onset Pompe disease confirmed by either a) enzyme assay confirming low GAA activity or b) DNA testing. Nexviazyme should not be prescribed concurrently with Lumizyme.

Codes Used In This BI:

J0180             Injection, agalsidase, beta, 1 mg

J0205             Injection, alglucerase, per 10 units

J1786             Injection, imiglucerase, per unit

J1931             Injection, laronidase, 0.1 mg

J1458             Injection, galsulfase, per 5 mg

J0220             Injection, alglucosidase alfa

J0221             Injection, alglucosidase alfa (Lumizyme), 10 mg

J1743             Injection, idursulfase

J3385             Injection, velaglucerase 100 units

J3060             Injection, taliglucerase alfa, 10 units

S9357            Home infusion therapy, enzyme-replacement intravenous therapy

J2840             Injection, sebelipase alfa, 1mg

C9085            Injection, avalglucosidase alfa-ngpt, 4mg

1. Laronidase is considered experimental and investigational for treating members with the Scheie form of MPS I who have only mild symptoms, as the risks and benefits of treating mildly affected persons with the Scheie form have not been established.
2. Use of aglucosidase alpha in persons with Pompe disease other than infantile-onset is considered experimental and investigational, because according to the FDA, aglucosidase alpha has not been adequately studied in these other forms of Pompe disease to assure safety and efficacy

Gaucher’s Disease:

Alglucerase (Ceredase), imiglucerase (Cerezyme), velagluceraase (VPRIV), and taliglucerase (Elelyso) are currently the only enzyme-replacement treatments available for Type 1 (non-neuropathic) Gaucher disease. They have not been shown to reverse or ameliorate neurological symptoms associated with Type 2 (acute neuropathic) or Type 3 (sub-acute or chronic neuropathic) Gaucher disease. These products have also not been shown to improve health outcomes in patients with Type 1 Gaucher disease without signs or symptoms of disease.

These products are administered by intravenous infusion over a period of one to two hours.

The ICGG U.S. Regional Coordinators recommend that all children with Gaucher disease be treated with enzyme replacement therapy. Children with Gaucher disease are at high risk for irreversible, morbid complications. The diagnosis of Gaucher disease in the 1st and 2nd decades of life is indicative of a rapidly progressive course. Early intervention is necessary for these children, during the time when the skeleton is immature, to enable them to attain their peak skeletal mass by early adulthood.

Mucopolysaccharidosis I (MPS I)

The mucopolysaccharidoses (MPSs) are a group of inherited lysosomal storage disorders caused by the deficiency of specific enzymes that are required for the degradation of glycosaminoglycan’s (GAGs), or mucopolysaccharides. Mucopolysaccharidosis I disease (MPS I) is a progressive, autosomal recessive genetic disorder resulting from a defect in the gene for the lysosomal enzyme alpha-L-iduronidase.

Laronidase is administered to provide exogenous enzyme for uptake into the lysosomes in order to increase the catabolism of GAG. Enzyme replacement therapy with laronidase has been shown to provide clinically important benefits, such as improved pulmonary function and walking ability and reduction of excess carbohydrates stored in organs.

Laronidase is administered intravenously once every week.

Fabry Disease

Fabry disease is a progressive, X-linked genetic disorder resulting from a defect in the gene for the lysosomal enzyme, alpha-GAL. This enzyme deficiency results in an accumulation of globotriosylceramide (GL-3) and other lipids in tissues throughout the body.

Agalsidase beta reduces GL-3 deposition from the interstitial endothelium of the kidney and certain other cell types. The reduction of GL-3 inclusions suggests that agalsidase beta may ameliorate disease expression of Fabry disease; however, the FDA-approved labeling notes that the relationship of GL-3 inclusion reduction to specific clinical manifestations of Fabry disease has not been established.

Agalsidase beta is administered by intravenous infusion every 2 weeks.

Mucopolysaccharidosis VI

Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome, is a debilitating, life-threatening genetic disease caused by a deficiency of the enzyme N-acetylgalactosamine 4-sulfatase. This deficiency results in the accumulation of glycosaminoglycan’s in the lysosomes, giving rise to progressive cellular, tissue and organ system dysfunction.

On June 1, 2005, galsulfase (Naglazyme, BioMarin Pharmaceutical Inc., Novato, CA) was granted orphan drug status by the FDA for the treatment of MPS VI. Galsulfase has been reported to improve endurance as shown by the 12-minute walk test as well as the 3-minute stair climb. It reduces the urinary excretion of glycosaminoglycan’s, an indication of enzymatic bioactivity, in patients with MPS VI.

Galsulfase is administered by intravenous infusion weekly.

Infantile Pompe Disease

Infantile Pompe disease (IPD), also known as infantile acid maltase deficiency and type 2 glycogen storage disease, is an autosomal recessive muscle-wasting disorder due to a deficiency of the lysosomal enzyme acid alpha-glucosidase. The deficiency results in accumulation of glycogen in lysosomes and is characterized by progressive cardiomyopathy, skeletal muscle weakness and respiratory insufficiency leading to death in early infancy.

On April 28, 2006, the FDA approved alglucosidase alfa, rhGAA (Myozyme), the first treatment for IPD. Alglucosidase alfa had been granted FDA orphan drug status and was approved under a priority review.

Patient survival without needing invasive ventilatory support was substantially greater in the alglucosidase alfa-treated infants than would be expected considering the high mortality rate of untreated patients of similar age and disease severity. According to the FDA, the drug`s safety and effectiveness in other forms of Pompe disease have not been adequately studied.

1.    International Collaborative Gaucher Group (ICGG), U.S. Regional Coordinators. Guidelines on Management of Gaucher Disease. Cambridge, MA: ICGG; 1998.

2.    Vellodi A, Bembi B, de Villemeur TB, et al. Management of neuropathic Gaucher disease: A European Consensus. Neuropathic Gaucher Disease Task Force of the European Working Group on Gaucher Disease. J Inherit Metab Dis. 2001;24:310-327. Available at: http://www.gaucher.org.uk/consen.pdf

3.    Kakkis ED, Muenzer J, Tiller GE. Enzyme-replacement therapy in mucopolysaccharidosis I. N Engl J Med. 2001;344(3):182-188.

4.    Roubicek M, Gehler J, Spranger J. The clinical spectrum of alpha-L-iduronidase deficiency. Am J Med Genet. 1985;20(3):471-481.

5.    Muenzer J, Clarke LA, Kolodny EH, et al. Enzyme replacement therapy for MPS I: 36-week interim results of the Phase 3 open-label extension study [abstract]. Proceeds of the Annual Clinical Genetics Meeting. 2003:34.

6.    Desnick RJ. Fabry disease, an under-recognized multisystemic disorder: Expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med. 2003;138:338-346.

7.    National Horizon Scanning Centre (NHSC). Enzyme replacement therapy for people with Fabry`s disease. Birmingham, UK: NHSC; 2001:5.

8.    Eng CM, Guffon N, Wilcox WR, et al. Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry`s disease. N Engl J Med. 2001:345:9-16.

9.    Canadian Coordinating Office for Health Technology Assessment (CCOHTA). Agalsidase alpha and agalsidase beta. Ottawa, ON: CCOHTA; 2002.

10. Charrow J, Andersson HC, Kaplan P, et al. Enzyme replacement therapy and monitoring for children with type 1 Gaucher disease: Consensus recommendations. J Pediatr. 2004;144(1):112-120.

11. Harmatz P, Whitley CB, Waber L, et al. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr. 2004;144(5):574-580.

12. Wraith JE, Clarke LA, Beck M, et al. Enzyme replacement therapy for mucopolysaccharidosis I: A randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase). J Pediatr. 2004;144(5):581-588.

13. Brady RO, Murray GJ, Moore DF, Schiffmann R. Enzyme replacement therapy in Fabry disease. J Inherit Metabol Dise. 2001;24(Suppl 2) 18-24; discussion 11-12.

14. Hilz MJ, Brys M, Marthol H, et al. Enzyme replacement therapy improves function of C-, Adelta-, and Abeta-nerve fibers in Fabry neuropathy. Neurology. 2004;62(7):1066-1072.

15. International Fabry Disease Study Group. Long-term safety and efficacy of enzyme replacement therapy for Fabry disease. Am J Human Genetics. 2004;75(1):65-74

16. Heitner R, Arndt S, Levin JB. Imiglucerase low-dose therapy for pediatric Gaucher disease--a long-term cohort study. S Afr Med J. 2004;94(8):647-651.

17. Harmatz P, Whitley CB, Waber L, et al. Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr. 2004;144(5):574-80.

18. Andersson HC, Charrow J, Kaplan P, et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet Med. 2005;7(2):105-110.

19. Grewal SS, Wynn R, Abdenur JE, et al. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med. 2005;7(2):143-146.

20. Canadian Coordinating Office for Health Technology Assessment (CCOHTA). Laronidase. Ottawa, ON: CCOHTA; 2004.

21. BioMarin Pharmaceuticals Inc. Naglazyme (galsulfase) solution for intravenous infusion only. Full Prescribing Information. D0624-01P. Rev. 01. Novato, CA; BioMarin; June 2005. Available at: http://www.naglazyme.com/

22. Klinge L, Straub V, Neudorf U, et al. Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study. Neuropediatrics. 2005;36(1):6-11.

23. Klinge L, Straub V, Neudorf U, et al. Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial. Neuromuscul Disord. 2005;15(1):24-31.

24. Scheinfeld, NS. Lysosomal storage disease. eMedicine Pediatric Neurology Topic 668. Omaha, NE: eMedicine.com; updated February 14, 2005. Available at: http://www.emedicine.com/neuro/topic668.htm .

25. Genzyme Corp. Myozyme (alglucosidase alfa). Prescribing Information. Cambridge, MA: Genzyme: April 2006. Available at: http://www.myozyme.com/PDF/mz_pi.pdf .

26. U.S. Food and Drug Administration (FDA). FDA approves first treatment for Pompe disease. FDA News. Rockville, MD: FDA; April 28, 2006. Available at: http://www.fda.gov/bbs/topics/NEWS/2006/NEW01365.html .

27. Clinical Pharmacology. Accessed online 05-23-2018.

28. Nexviazyme Prescribing Information. Cambridge, MA: Genzyme Corporation; August 2021.

Addendum:

Effective 11/01/2018:  Added Kanuma (J2840) to policy.

Effective 01/01/2022: Updated with Nexviazyme (C9085) coverage criteria for late onset Pompe disease.

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