Last Review Date: 09/05/2024

Date of Origin:  03/07/2013

Dates Reviewed: 12/2013, 02/2014, 09/2014, 07/2015, 07/2016, 08/2016, 08/2017, 07/2018, 07/2019, 07/2020, 09/2021, 09/2022, 09/2023, 09/2024

1. Length of Authorization

Coverage will be provided for 12 months and may be renewed.

2. Dosing Limits

1. Quantity Limit (max daily dose) [NDC Unit]:

• Elelyso 200 unit powder for injection: 35 vials per 14 days

2. Max Units (per dose and over time) [HCPCS Unit]:

• 700 billable units every 14 days

3. Initial Approval Criteria ¹

Coverage is provided in the following conditions:

• Patient is at least 4 years of age; AND

Universal Criteria ¹

• Used as a single agent; AND

Type 1 Gaucher Disease † Ф ^1,6,13-17

• Patient has a documented diagnosis of Type 1 Gaucher Disease confirmed by one of the following:

• Significantly reduced or absent glucocerebrosidase enzyme activity as measured by a beta-glucosidase leukocyte (BGL) test
• Detection of mutations in the glucocerebrosidase (GBA) gene; AND

• Patient’s disease results in one or more of the following:

• Anemia-related symptoms [i.e., blood transfusion dependency and/or hemoglobin ≤ 11 g/dL (women and children) or ≤ 12 g/dL (men)]

• Thrombocytopenia (platelet count ≤ 120,000/mm³)
• Hepatomegaly or splenomegaly
• Skeletal disease (e.g., lesions, remodeling defects and/or deformity of long bones, osteopenia/osteoporosis, etc.)
• Symptomatic disease (e.g., bone pain, fatigue dyspnea, abdominal distension, diminished quality of life, etc.)

† FDA Approved Indication(s); ‡ Compendia Recommended Indication(s); Ф Orphan Drug

4. Renewal Criteria ^1,6,12,14-17

Coverage can be renewed based on the following criteria:

• Patient continues to meet the universal and other indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in section III; AND
• Disease response with treatment as defined by one or more of the following (compared to pre-treatment baseline):

• Improvement in anemia-related symptoms (i.e., improvement in hemoglobin and/or decrease in blood transfusion dependency)
• Improvement in platelet counts
• Reduction in size of liver or spleen
• Improvement in skeletal disease (e.g., increase in lumbar spine and/or femoral neck BMD, no bone crises or bone fractures, etc.)
• Improvement in symptoms (e.g., bone pain, fatigue, dyspnea, abdominal distension, quality of life, etc.); AND

• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: hypersensitivity reactions, including anaphylaxis, etc.

5. Dosage/Administration¹

6. Billing Code/Availability Information

HCPCS Code:

• J3060 – Injection, taliglucerase alfa, 10 units; 1 billable unit = 10 units

NDC:

• Elelyso 200 unit powder for injection, single-dose vial: 00069-0106-xx

7. References

1. Elelyso [package insert]. New York, NY; Pfizer Inc; July 2024. Accessed August 2024.
2. Anderson HC, et al. Consensus Statement by the International Collaborative Gaucher Group (ICGG) U.S. Coordinators on Individualization of ERT for Type-1 Gaucher Disease. September 2000.
3. Charrow, et al. Gaucher Disease: Recommendations on Diagnosis, Evaluation and Monitoring (Special Article). Archives of Internal Medicine 1998; 158:1754-1760.
4. Pastores GM, Weinreb NJ, Aerts H, et al. Therapeutic goals in the treatment of Gaucher disease. Semin Hematol 2004; 41:4.
5. Baldellou A, Andria G, Campbell PE, et al. Paediatric non-neuronopathic Gaucher disease: recommendations for treatment and monitoring. Eur J Pediatr 2004; 163:67.
6. Charrow J, Andersson HC, Kaplan P, et al. The Gaucher Registry: Demographics and disease characteristics of 1698 patients with Gaucher disease. Arch Intern Med 2000; 160:2835.
7. Martins AM, Valadares ER, Porta G, et al. Recommendations on diagnosis, treatment, and monitoring for Gaucher disease. J Pediatr. 2009 Oct;155(4 Suppl):S10-8.
8. Kaplan P, Baris H, De Meirleir L, et al. Revised recommendations for the management of Gaucher disease in children. Eur J Pediatr. 2013 Apr;172(4):447-58: doi: 10.1007/s00431-012-1771-z. Epub 2012 Jul 8.
9. Zimran A, Duran G, Mehta A, et al. Long-term Efficacy and Safety Results of Taliglucerase Alfa Up to 36 Months in Adult Treatment-Naïve Patients With Gaucher Disease. Am J Hematol. 2016 Jul;91(7):656-60. doi: 10.1002/ajh.24369. Epub 2016 Apr 24.
10. Zimran A, Almon E, Chertkoff R, et al. Pivotal Trial With Plant Cell-Expressed Recombinant Glucocerebrosidase, Taliglucerase Alfa, a Novel Enzyme Replacement Therapy for Gaucher Disease. Blood 2011 Nov 24;118(22):5767-73. doi: 10.1182/blood-2011-07-366955. Epub 2011 Sep 6.
11. Pastores G, Petakov M, Giraldo P, et al. A Phase 3, Multicenter, Open-Label, Switchover Trial to Assess the Safety and Efficacy of Taliglucerase Alfa, a Plant Cell-Expressed Recombinant Human Glucocerebrosidase, in Adult and Pediatric Patients With Gaucher Disease Previously Treated With Imiglucerase Blood Cells Mol Dis 2014 Dec;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18.
12. Biegstraaten M, Cox TM, Belmatoug N, et al. Management goals for type 1 Gaucher disease: An expert consensus document from the European working group on Gaucher disease. Blood Cells, Molecules and Diseases 68 (2018) 203-208.
13. DuaPuri R, Kapoor S, Kishnani PS, et al. Diagnosis and Management of Gaucher Disease in India – Consensus Guidelines of the Gaucher Disease Task Force of the Society for Indian Academy of Medical Genetics and the Indian Academy of Pediatrics. Indian Pediatrics. 5;2018: 143-153.
14. Kaplan P, Andersson HC, Kacena KA, Yee JD. The clinical and demographic characteristics of nonneuronopathic Gaucher disease in 887 children at diagnosis. Arch Pediatr Adolesc Med. 2006 Jun;160(6):603-8.
15. Pastores GM, Hughes DA. Gaucher Disease. GeneReviews®. www.ncbi.nlm.nih.gov/books/NBK1269/. Initial Posting: July 27, 2000; Last Revision: December 7, 2023. Accessed on August 2, 2024.
16. Hughs D, Sidransky E. (2024). Gaucher disease: Pathogenesis, clinical manifestations, and diagnosis. In Hahn S, TePas E (Eds.), UptoDate. Last updated: August 6, 2024. Accessed on August 13, 2024. Available from https://www.uptodate.com/contents/gaucher-disease-pathogenesis-clinical-manifestations-and-diagnosis?search=gaucher%20disease%20type%201&source=search_result&selectedTitle=1~150&usage_type=default&display_rank=1#H11.
17. Hughs D, Sidransky E. (2022). Gaucher disease: Treatment. In Hahn S, TePas E (Eds.), UptoDate. Last updated: August 02, 2022. Accessed on August 2, 2024. Available from https://www.uptodate.com/contents/gaucher-disease-treatment?sectionName=ENZYME%20REPLACEMENT%20THERAPY&search=gaucher%20disease%20type%201&topicRef=2918&anchor=H4&source=see_link#H15131002.

Appendix 1 – Covered Diagnosis Codes

Appendix 2 – Centers for Medicare and Medicaid Services (CMS)

The preceding information is intended for non-Medicare coverage determinations. Medicare coverage for outpatient (Part B) drugs is outlined in the Medicare Benefit Policy Manual (Pub. 100-2), Chapter 15, §50 Drugs and Biologicals. In addition, National Coverage Determinations (NCDs) and/or Local Coverage Determinations (LCDs) may exist and compliance with these policies is required where applicable. Local Coverage Articles (LCAs) may also exist for claims payment purposes or to clarify benefit eligibility under Part B for drugs which may be self-administered. The following link may be used to search for NCD, LCD, or LCA documents: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications, including any preceding information, may be applied at the discretion of the health plan.

Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD/LCA): N/A