Last Review Date: 02/01/2024

Date of Origin:  11/28/2011

Dates Reviewed: 12/2011, 02/2013, 02/2014, 08/2014, 07/2015, 10/2016, 10/2017, 10/2018, 02/2019, 02/2020, 02/2021, 09/2021, 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]:

• Lumizyme 50 mg single-dose vial: 46 vials every 14 days

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

• 230 billable units every 14 days

3. Initial Approval Criteria ^1,4

         Coverage is provided in the following conditions:

• Documented baseline age-appropriate values for one or more of the following:
  • Infantile-onset disease: muscle weakness, motor function, respiratory function, cardiac involvement, percent predicted forced vital capacity (FVC), and/or 6-minute walk test (6-MWT); OR
  • Late-onset (non-infantile) disease: 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

• Will not be used in combination with other enzyme replacement therapies (i.e., avalglucosidase alfa, cipaglucosidase alfa, etc.); AND
• Patients susceptible to fluid volume overload or those with an acute underlying respiratory illness or compromised cardiac or respiratory function will be closely monitored for exacerbation of their cardiac or respiratory status during infusion; AND

Pompe Disease (Acid Alpha-Glucosidase (GAA) deficiency) † Ф ^1,4

• Diagnosis has been confirmed by one of the following:
  • Deficiency of acid alpha-glucosidase (GAA) enzyme activity**; OR
  • Detection of biallelic pathogenic variants in the GAA gene by molecular genetic testing

**Note: The diagnosis of Infantile-Onset Pompe Disease (IOPD) can be established rapidly after a positive newborn screening (NBS) result when physical examination, echocardiography, and elevated CPK support the diagnosis. It is recommended that the diagnosis be confirmed either by molecular genetic testing or by measurement of GAA activity in another tissue.⁴

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

4. Renewal Criteria ^1,4

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 hypersensitivity reactions, immune-mediated cutaneous reactions, systemic immune-mediated reactions, acute cardiorespiratory failure, cardiac arrhythmia during general anesthesia, etc.; AND
• Patient is being monitored for antibody formation (including neutralizing antibodies); AND
• Patient has demonstrated a beneficial response to therapy compared to pretreatment age-appropriate baseline values in one or more of the following:
  • Infantile-onset disease: stabilization or improvement in muscle weakness, motor function, respiratory function, cardiac involvement, FVC and/or 6-MWT; OR
  • Late-onset (non-infantile) disease: stabilization or improvement in FVC and/or 6-MWT

5. Dosage/Administration ¹

   Indication	Dose
   Pompe Disease	Administer 20 mg/kg body weight as an intravenous (IV) infusion every 2 weeks

6. Billing Code/Availability Information

HCPCS Code:

• J0221 – Injection, alglucosidase alfa, (lumizyme), 10 mg; 1 billable unit = 10 mg

NDC:

• Lumizyme 50 mg single-dose vial for injection: 58468-0160-xx

7. References

1. Lumizyme [package insert]. Cambridge, MA; Genzyme Corporation; May 2023. Accessed December 2023.
2. Cupler EJ, Berger KI, Leshner RT, et al. Consensus treatment recommendations for late-onset Pompe disease. Muscle Nerve. 2012 Mar; 45(3):319-33. doi: 10.1002/mus.22329. Epub 2011 Dec 15.
3. Kishnani PS, Steiner RD, Bali D, et al. Pompe disease diagnosis and management guidelines. Genet Med 2006; 8:267-88.
4. Nancy L, Bailey L. Pompe Disease. GeneReviews. www.ncbi.nlm.nih.gov/books/NBK1261/. Initial Posting: August 31, 2007; Last Update: November 2, 2023.. Accessed on January 02, 2024.
5. Tarnopolsky M, Katzberg H, Petrof BJ, et al. Pompe Disease: Diagnosis and Management. Evidence-Based Guidelines from a Canadian Expert Panel. Can J Neurol Sci. 2016 Jul;43(4):472-85.
6. Kishnani PS, Hwu WL, et al. Introduction to the Newborn Screening, Diagnosis, and Treatment for Pompe Disease Guidance Supplement. Pediatrics 2017 Jul:(1):S1-S3.
7. van der Ploeg AT, Clemens PR, Corzo D, et al. A randomized study of alglucosidase alfa in late-onset Pompe's disease. N Engl J Med. 2010 Apr 15;362(15):1396-406. doi: 10.1056/NEJMoa0909859.
8. Nicolino M, Byrne B, Wraith JE, et al. Clinical outcomes after long-term treatment with alglucosidase alfa in infants and children with advanced Pompe disease. Genet Med. 2009 Mar;11(3):210-9. doi: 10.1097/GIM.0b013e31819d0996.
9. Sawada T, Kido J, Nakamura K. Newborn Screening for Pompe Disease. Int J Neonatal Screen. 2020 Jun; 6(2): 31.Published online 2020 Apr 5. doi: 10.3390/ijns6020031

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