Last Review Date: 02/02/2023

Date of Origin: 05/31/2016

Dates Reviewed: 05/2016, 04/2017, 04/2018, 02/2019, 02/2020, 02/2021, 12/2021, 02/2022, 02/2023

1. Length of Authorization

Initial coverage will be provided for 6 months and may be renewed annually thereafter.

2. Dosing Limits

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

• Kanuma 20 mg/10 mL single-dose vials: 112 vials per 28 day supply

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

• 560 billable units once weekly

3. Initial Approval Criteria ¹

Coverage is provided in the following conditions:

• Patient is at least 1 month of age; AND

Lysosomal Acid Lipase (LAL) Deficiency † Ф ^1-7

• Diagnosis has been confirmed by either biallelic pathogenic variants in LIPA or deficient LAL enzyme activity in peripheral blood leukocytes, fibroblasts, or dried blood spots

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

4. Renewal Criteria ^1,6,7

Coverage can be renewed based upon the following criteria:

• Patient continues to meet indication-specific relevant criteria such as concomitant therapy requirements (not including prerequisite therapy), performance status, etc. identified in section III; AND
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include:  hypersensitivity reactions including anaphylaxis, etc.; AND
  • Treatment has resulted in clinical benefit as evidenced in one or more of the following:

• Improvement in weight-for-age z-scores for patients exhibiting growth failure
• Improvement in LDL
• Improvement in HDL
• Improvement in triglycerides
• Improvement of AST or ALT; OR
  • Dose escalation in pediatric and adult patients with a suboptimal clinical response to the 1 mg/kg dose defined by at least one of the following:

• • Poor growth
  • Deteriorating biochemical markers [e.g., alanine aminotransferase (ALT), aspartate aminotransferase (AST)], and/or parameters of lipid metabolism [e.g., low-density lipoprotein cholesterol (LDL-c), triglycerides (TG)]; OR

• • Dose escalation for infants with rapidly progressive disease presenting within the first 6 months of life who have a suboptimal clinical response to the 1 mg/kg dose or 3 mg/kg dose defined by at least one of the following:

• • Poor growth
  • Deteriorating biochemical markers [e.g., alanine aminotransferase (ALT), aspartate aminotransferase (AST)]
  • Persistent or worsening organomegaly

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS Code:

J2840 - Injection, sebelipase alfa, 1 mg: 1 billable unit = 1 mg

NDC:

Kanuma 20 mg/10 mL single-dose vials: 25682-0007-xx

7. References

1. Kanuma [package insert]. Boston, MA; Alexion Pharmaceuticals, Inc; November 2021. Accessed January 2023.
2. Porto AF. Lysosomal acid lipase deficiency: diagnosis and treatment of Wolman and Cholesteryl Ester Storage Diseases. Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:125-32.
3. Zhang B, Porto AF. Cholesteryl ester storage disease: protean presentations of lysosomal acid lipase deficiency. Pediatr Gastroenterol Nutr. 2013;56(6):682. 
4. Reiner Z, Guardamagna O, Nair D, et al. Lysosomal acid lipase deficiency--an under-recognized cause of dyslipidaemia and liver dysfunction. Atherosclerosis. 2014 Jul;235(1):21-30. doi: 10.1016/j.atherosclerosis.2014.04.003.
5. Hamilton J, Jones I, Srivastava R. A new method for the measurement of lysosomal acid lipase in dried blood spots using the inhibitor Lalistat 2. Clin Chim Acta. 2012 Aug 16;413(15-16):1207-10. doi: 10.1016/j.cca.2012.03.019.
6. Burton BK, Balwani M, Feillet F, et al. A Phase 3 Trial of Sebelipase Alfa in Lysosomal Acid Lipase Deficiency. 2015 Sep 10;373(11):1010-20. doi: 10.1056/NEJMoa1501365.
7. Jones SA, Rojas-Caro S, Quinn AG, et al. Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study. Orphanet J Rare Dis. 2017 Feb 8;12(1):25. doi: 10.1186/s13023-017-0587-3.

Appendix 1 – Covered Diagnosis Codes

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

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 Determination (NCD), Local Coverage Determinations (LCDs), and Local Coverage Articles (LCAs) may exist and compliance with these policies is required where applicable. They can be found at: https://www.cms.gov/medicare-coverage-database/search.aspx. Additional indications may be covered at the discretion of the health plan.

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