Background

Lysosomal Storage Disorders (LSDs) are a group of inherited metabolic disorders in which harmful amounts of fatty materials accumulate in various cells and tissues in the body. People with these disorders either do not produce enough of one of the enzymes needed to metabolize or they produce enzymes that do not work properly. Although there is no cure, enzyme replacement therapy is available for some of the LSDs including Gaucher Disease.

Gaucher disease is a genetic disorder resulting in the malfunction or absence of the enzyme glucocerebrosidase. The deficiency of this enzyme causes accumulation of glucosylceramide in the liver, spleen, bone marrow, and lymph nodes. Treatment for this disease focuses on either replacement of the missing or ineffective enzyme (enzyme replacement therapy [ERT]), or reduction of the substrate upon which it acts (substrate reduction therapy [SRT]).

Policy (Criteria)

Cerezyme (imiglucerase)
Elelyso (taliglucerase)
Vpriv (velaglucerase)

Coverage of these drugs is provided when the criteria below is met and for non–preferred products there has been a trial and failure of preferred therapy (if applicable).

Gaucher Disease Agents may be considered MEDICALLY NECESSARY for the following condition(s):

1.  Diagnosis of Type 1 Gaucher disease
2. Member has evidence of symptomatic disease (e.g., moderate to severe anemia, thrombocytopenia, bone disease, hepatomegaly, or splenomegaly)
3. Member meets FDA approved age requirement

Authorization duration: 12 months

References

1. Elelyso prescribing information. Pfizer. October 2019. Last accessed August 2020.
2. Vpriv prescribing information. Shire. November 2019. Last accessed August 2020.
3. Cerezyme prescribing information. Genzyme. March 2020. Last accessed August 2020.
4. Lysosomal Storage Disorders. Blue Cross & Blue Shield of Mississippi. October 2024. Last accessed November 2025.
5. Polinski JM, Kowal MK, Gagnon M, et al. Home infusion: safe clinically effective, patient preferred, and cost saving. Healthcare. 2016. Accessed November 9, 2017.
6. ASHP Guidelines on Home Infusion Pharmacy Services, 2013. Accessed September 2, 2017.
7. MCGTM Care Guidelines, 22nd edition, 2018, Home Infusion Therapy, CMT: CMT-0009(SR).
8. Micromedex DrugDex Compendium® 2024. Imiglucerase.
9. Alfa.Micromedex DrugDex Compendium® 2024.Taliglucerase alfa.
10. Clinical PharmacologyTM Compendium. 2024.Tampa FL: Gold Standard, Inc. Imiglucerase..
11. Clinical PharmacologyTM Compendium. 2024.Tampa FL: Gold Standard, Inc. Velaglucerase Alfa.
12. Stirnemann J, Belmatoug N, Camou F, et al. A review of gaucher disease pathophysiology, clinical presentation and treatments. Int J Mol Sci. 2017;18(2):441.
13. Gary SE, Ryan E, Steward AM, Sidransky E. Recent advances in the diagnosis and management of Gaucher disease. Expert Rev Endocrinol Metab. 2018;13(2):107‐118.
14. El-Beshlawy A, Tylki-Szymanska A, Vellodi A, et al. Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry. Mol Genet Metab 2017;120(1-2);47-56.
15. Pastores GM, Hughes DA. Gaucher disease. GeneReviews. 2018;1-35.
16. Cerezyme (imiglucerase) for injection, for intravenous use [package insert]. Genzyme
17. Corporation. Cambridge, MA. Revised 7/2024.
18. Elelyso (taliglucerase alfa) for injection, for intravenous use [package insert]. Pfizer Labs. New York, New York. Revised 7/2024.
19. VPRIV (velaglucerase alfa) for injection, for intravenous use [package insert]. Shire Human Genetic Therapies, Inc. Lexington, MA. Revised 7/2024
20. National Institute of Health. National Organization for Rare Disorders. Gaucher Disease. Last published: 2018.
21. Lau H, Belmatoug N, Deegan P, et al. Reported outcomes of 453 pregnancies in patients with Gaucher disease: An analysis from the Gaucher outcome survey. Blood Cells Mol Dis.2018;68:226-231.
22. Weinreb NJ, Camelo JS Jr, Charrow J, et al. International Collaborative Gaucher Group (ICGG) Gaucher Registry (NCT00358943) investigators. Gaucher disease type 1 patients from the ICGG Gaucher Registry sustain initial clinical improvements during twenty years of imiglucerase treatment. Mol Genet Metab. 2021;132(2):100-111.
23. Imiglucerase In: AHFS Drug Information Online Electronic Medical Library. Bethesda, MD: American Society of Health-System Pharmacists. Updated March 21, 2017.
24. Taliglucerase alfa In: AHFS Drug Information Online Electronic Medical Library. Bethesda, MD: American Society of Health-System Pharmacists. Updated March 22, 2017.
25. Velaglucerase alfa In: AHFS Drug Information Online Electronic Medical Library. Bethesda, MD: American Society of Health-System Pharmacists. Updated March 22, 2017

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each Policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, technology assessment program (TEC) and other non-affiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines.

"Current Procedural Terminology © American Medical Association. All Rights Reserved" 

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