Last Review Date: 02/01/2022

Date of Origin: 04/29/2014

Dates Reviewed: 02/2015, 01/2016, 01/2017, 01/2018, 02/2019, 02/2020, 02/2021, 02/2022

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]:

• Vimizim 5mg/5ml: 184 vials every 28 days

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

• 230 billable units (230 mg) every 7 days

3. Initial Approval Criteria ¹

Coverage is provided in the following conditions:

• Patient is at least 5 years of age; AND
• Documented baseline value for one or more of the following: endurance tests (e.g., six-minute walk test [6-MWT], timed 25-foot walk test [T25FW], three-minute stair climb test [3-MSCT]), and/or pulmonary function tests (e.g., percent predicted forced vital capacity [FVC]), etc.; AND

Mucopolysaccharidosis IVA (MPS IVA; Morquio A Syndrome) † Ф ^1,3-5

• Documented diagnosis of Mucopolysaccharidosis IVA with biochemical/genetic confirmation by one of the following:

• Absence or marked reduction in N-acetylgalactosamine 6-sulfatase (GALNS) enzyme activity in cultured fibroblasts or leukocytes ; OR
• Detection of biallelic pathogenic mutations in the GALNS gene by genetic molecular testing (i.e., sequence analysis and/or deletion/duplication analysis)

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

4. Renewal Criteria ^1,3.5

• 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: anaphylaxis and hypersensitivity reactions, acute respiratory complications, spinal/cervical cord compression, etc.; AND
• Patient has shown a response to therapy as evidenced by the following when compared to pretreatment baseline values:
  • Stability or improvement in endurance tests (e.g., six-minute walk test [6-MWT], timed 25-foot walk test [T25FW], three-minute stair climb test [3-MSCT]); OR
  • Stability or improvement in pulmonary function tests (e.g., FVC, etc.)

5. Dosage/Administration ¹  

6. Billing Code/Availability Information

HCPCS Code:

• J1322 – Injection, elosulfase alfa, 1 mg: 1 billable unit = 1 mg 

NDC:

• Vimizim 5mg per5ml solution; single-dose vial: 68135-0100-xx

7. References

1. Vimizim [package insert].  Novato, CA; Biomarin Pharmaceutical Inc.; December 2019.  Accessed January 2022.
2. Hendriksz CJ, Berger KI, Giugliani R, et al. International Guidelines for the Management and Treatment of Morquio A Syndrome. Am J Med Genet A. 2015 Jan; 167(1): 11–25. Published online 2014 Oct 24.  doi:  10.1002/ajmg.a.36833
3. Regier DS, Oetgen M, Tanpaiboon P. Mucopolysaccharidosis Type IVA. GeneReviews® https://www.ncbi.nlm.nih.gov/books/NBK148668/. Initial Posting: July 11, 2002; Last Update: June 17, 2021. Accessed on January 21, 2022.
4. Schweighardt B, Tompkins T, Lau K, et al. Immunogenicity of Elosulfase Alfa, an Enzyme Replacement Therapy in Patients With Morquio A Syndrome: Results From MOR-004, a Phase III Trial. Clin Ther. 2015 May 1;37(5):1012-1021.e6. doi: 10.1016/j.clinthera.2014.11.005. Epub 2014 Dec 6.
5. Hendriksz CJ, Burton B, Fleming TR, et al. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014 Nov;37(6):979-90. doi: 10.1007/s10545-014-9715-6. Epub 2014 May 9.

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