Last Review Date: 02/01/2024

Date of Origin: 11/28/2011

Dates Reviewed: 12/2011, 02/2013, 02/2014, 12/2014, 10/2015, 10/2016, 10/2017, 10/2018, 02/2020, 02/2021, 02/2022, 02/2023, 02/2024

1. Length of Authorization

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

2. Dosing Limits

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

• Aldurazyme 2.9 mg/5 mL vial: 92 vials every 28 days

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

• 667 billable units every 7 days

3. Initial Approval Criteria ^1-6

Coverage is provided in the following conditions:

• Patient is at least 6 months of age; AND
• Documented baseline value for urinary glycosaminoglycan (uGAG) has been obtained; AND
• Documented baseline age-appropriate values for one or more of the following have been obtained:
  • Patients 6 years of age or greater: percent predicted forced vital capacity (FVC), 6-minute walk test (6-MWT), joint range of motion, left ventricular hypertrophy, growth, and/or quality of life (CHAQ/HAQ/MPS HAQ); OR
  • Patients 6 months to less than 6 years of age: cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC, and/or 6-MWT; AND

**NOTE: For very young patients in which FVC or 6-MWT are not suitable for measuring, requests will be reviewed on a case-by case basis.

Universal Criteria ^1,7,8

• Therapy is being used to treat non-central nervous system manifestations of the disease and patient does not have severe, irreversible cognitive impairment; AND

Mucopolysaccharidosis I (MPS I) † Ф ^1,2

• Patient has a definitive diagnosis of MPS I as confirmed by one of the following:
  • Detection of biallelic pathogenic mutations in the α-L-iduronidase (IDUA) gene by molecular genetic testing; OR
  • Detection of deficient activity of the IDUA lysosomal enzyme; AND
• Patient has one of the following diagnoses:
  • Hurler (severe) or Hurler-Scheie (attenuated) forms of disease; OR
  • Scheie (attenuated) form of disease with moderate to severe symptoms

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

4. Renewal Criteria ^1,2,5,6

Coverage may 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
• Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include: anaphylaxis and severe hypersensitivity reactions, acute respiratory complications, acute cardiorespiratory failure, severe infusion reactions, etc.; AND
• Patient has a documented reduction in uGAG levels compared to pretreatment baseline; AND
• Patient has demonstrated a beneficial response to therapy compared to pretreatment age-appropriate baseline values in one or more of the following:
  • Patients 6 years of age or greater: stability or improvement in percent predicted FVC and/or 6-MWT, increased joint range of motion, decreased left ventricular hypertrophy, improved growth, and/or improved quality of life (clinically meaningful change in the CHAQ/HAQ/MPS HAQ disability index); OR
  • Patients 6 months to less than 6 years of age: stability or improvement in cardiac status, upper airway obstruction during sleep, growth velocity, mental development, FVC and/or 6-MWT

5. Dosage/Administration ¹

6. Billing Code/Availability Information

HCPCS Code:

• J1931 – Injection, laronidase, 0.1 mg; 1 billable unit = 0.1 mg

NDC:

• Aldurazyme 2.9 mg/5 mL single-dose vial: 58468-0070-xx

7. References

1. Aldurazyme [package insert].  Cambridge, MA; Genzyme Corporation.; December 2023.   Accessed December 2023.
2. Clark LA. Mucopolysaccharidosis Type I. GeneReviews®. www.ncbi.nlm.nih.gov/books/NBK1162/. Initial Posting: October 31, 2002; Last Update: February 25, 2021. Accessed on December 27, 2023.
3. Muenzer J, Wraith JE, Clarke LA; International Consensus Panel on Management and Treatment of Mucopolysaccharidosis I. Mucopolysaccharidosis I: management and treatment guidelines. Pediatrics. 2009 Jan; 123(1):19-29. doi: 10.1542/peds.2008-0416.
4. Martins AM, Dualibi AP, Norato D, et al. Guidelines for the Management of Mucopolysaccharidosis Type I. JPeds. 2009 Oct; 155 (4): S32 - S46. doi: 10.1016/j.jpeds.2009.07.005.
5. Clarke LA, Wraith JE, Beck M, et al. Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I. Pediatrics. 2009 Jan;123(1):229-40. doi: 10.1542/peds.2007-3847.
6. Wraith JE, Beck M, Lane R, et al. Enzyme replacement therapy in patients who have mucopolysaccharidosis I and are younger than 5 years: results of a multinational study of recombinant human alpha-L-iduronidase (laronidase). Pediatrics. 2007 Jul;120(1):e37-46. Epub 2007 Jun 4.
7. Zhou J, Lin J, Leung WT, Wang L. A basic understanding of mucopolysaccharidosis: Incidence, clinical features, diagnosis, and management. Intractable Rare Dis Res. 2020 Feb;9(1):1-9. doi: 10.5582/irdr.2020.01011.
8. Shapiro EG, Eisengart JB. The natural history of neurocognition in MPS disorders: A review. Mol Genet Metab. 2021 May;133(1):8-34. doi: 10.1016/j.ymgme.2021.03.002. Epub 2021 Mar 11. PMID: 33741271.

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