ModifiedmhnPolicy CP.PHAR.160

Alglucosidase Alfa (Lumizyme)

Coverage and authorization criteria for alglucosidase alfa (Lumizyme) for treatment of Pompe disease across commercial, HIM, and Medicaid lines of business.

Policy Summary

Payermhn

PolicyAlglucosidase Alfa (Lumizyme)

Policy CodePolicy CP.PHAR.160

Change TypeMaterial updates to exclusions, diagnostic confirmation, and authorization duration

Effective Date02.01.16

Next Review Date

Key ActionSubmit prior authorization with documentation confirming Pompe disease and ensure no concurrent use with Nexviazyme or Pombiliti+Opfolda.

SourceLink

POLICY UPDATE CHANGES

Updated initial authorization duration from 6 months to 12 months for Medicaid/HIM.

Added increased lysosomal glycogen as an additional option for confirming a Pompe disease diagnosis.

Added exclusion for concomitant use with Pombiliti+Opfolda to align with the Pombiliti criteria.

Requirement that Lumizyme not be prescribed concurrently with Nexviazyme.

12 moinitial auth (Medicaid/HIM)

6 moinitial auth (Commercial)

20 mg/kgmaximum dose

J0220/J0221HCPCS

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Coverage Criteria

inv-01: Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

Initial Approval - Pompe Disease: 1) Diagnosis of Pompe disease (GAA deficiency) confirmed by one of the following: (a) enzyme assay confirming low GAA activity; (b) DNA testing; (c) increased lysosomal glycogen; 2) Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda; 3) Dose does not exceed 20 mg/kg every 2 weeks.

inv-02: Continuation Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

Continued Therapy - Pompe Disease: 1) Member meets one of the following: (a) currently receiving medication via Centene benefit or previously met initial approval criteria; (b) currently receiving medication and enrolled in a state/product with continuity of care regulations; 2) Member is responding positively to therapy as evidenced by improvement in the individual's Pompe disease manifestation profile (see Appendix D for examples); 3) Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda; 4) If request is for a dose increase, new dose does not exceed 20 mg/kg every 2 weeks.

See Appendix D for examples of therapeutic response.

Non–FDA approved indications that are not specifically addressed in this policy are excluded from coverage unless the provider supplies sufficient documentation of efficacy and safety in accordance with the applicable off‑label use policy. See CP.CPA.09 for commercial, HIM.PA.154 for health insurance marketplace, and CP.PMN.53 for Medicaid for the required supporting information and review criteria.

Lumizyme must not be prescribed concurrently with Nexviazyme. In addition, Lumizyme is excluded for concomitant use with the combination of Pombiliti and Opfolda. These concurrent‑use prohibitions are part of the coverage criteria and were added to align with related product criteria during policy review.

Requests that seek authorization for concurrent administration of Lumizyme with Nexviazyme, or for Lumizyme given at the same time as the combination of Pombiliti plus Opfolda, are not authorized under this policy and should be denied.

Coding and Dosing

HCPCS CodesHCPCS

J0220	Injection, alglucosidase alfa, 10 mg, not otherwise specified
J0221	Injection, alglucosidase alfa, (Lumizyme), 10 mg

Maximum dose — 20 mg/kg every 2 weeks

Maximum dose20 mg/kg every 2 weeks

Dosing routeIV infusion

Indication tied to dosePompe disease dosing regimen: 20 mg/kg IV every 2 weeks

Provider Actions and Authorization

Prior Authorization

Prior Authorization Required

Prior authorization required. Initial and continued therapy criteria must be met for approval.

Prior authorization required for Lumizyme
Initial and continuation criteria must be documented and met

Documentation Required

Required Documentation

Provider must submit documentation (office chart notes, lab results, genetic testing, enzyme assay results, or other clinical information) supporting that the member has met all approval criteria.

Office chart notes
Laboratory results (including enzyme assay)
Genetic testing reports
Any other clinical information supporting criteria

Initial Therapy Criteria

inv-19: Initial Therapy — top-level initial therapy criteria node

Initial Therapy Criteria: Confirmed Pompe disease by enzyme assay, DNA testing, or increased lysosomal glycogen; Lumizyme is not prescribed concurrently with Nexviazyme or Pombiliti+Opfolda; dose does not exceed 20 mg/kg every 2 weeks.

inv-20: Initial authorization duration — authorization duration for initial therapy

Initial authorization duration for Medicaid/HIM is 12 months.

Updated 1Q 2026

Continuation Therapy Criteria

inv-21: Continued Therapy — top-level continued therapy criteria node

Continued Therapy Criteria: Member currently receiving medication or previously met initial criteria (or continuity of care as applicable); evidence of positive response demonstrated by improvement in the member's Pompe disease manifestation profile (see Appendix D for examples); Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda; if request is for dose increase, new dose does not exceed 20 mg/kg every 2 weeks.

See Appendix D for response measures (e.g., ventilator independence, motor function gains, maintained/improved FVC or 6MWT).

Quantity Limits

Alglucosidase alfa (Lumizyme) quantity limit — 20 mg/kg

Quantity limit (per dosing event)20 mg/kg (maximum dosing) every 2 weeks

Vial availabilitySingle-use vial: 50 mg

Administration settingIV infusion per dosing regimen (hospital outpatient appropriate)

Site of Care

Billing Rule

Hospital outpatient administration and vial availability

For hospital outpatient administration, Lumizyme is given by IV infusion per the dosing regimen; a single-use 50 mg vial is available for preparation.

Administer IV infusion according to dosing (20 mg/kg every 2 weeks)
Single-use vial available: 50 mg

Definitions

Lumizyme / GAA — GAA definition and Lumizyme as enzyme replacement therapy

Definition - GAAGAA = acid alpha-glucosidase (enzyme deficient in Pompe disease)

DrugAlglucosidase alfa (Lumizyme)

Mechanism / roleLumizyme is a lysosomal glycogen-specific enzyme replacement therapy for Pompe disease

Diagnostic marker — increased lysosomal glycogen

Diagnostic markerIncreased lysosomal glycogen (listed as an option to confirm Pompe disease)

Other confirmatory testsEnzyme assay confirming low GAA activity; DNA testing

Background

Pompe disease is caused by deficiency of the lysosomal enzyme acid alpha‑glucosidase (GAA), resulting in accumulation of glycogen within lysosomes. Clinical presentation spans a spectrum from infantile‑onset disease—characterized by cardiomyopathy and rapid clinical decline—to late‑onset forms with progressive skeletal muscle weakness and respiratory insufficiency. Alglucosidase alfa (Lumizyme) is a lysosomal glycogen‑specific enzyme replacement indicated for treatment of Pompe disease; diagnostic confirmation for treatment eligibility may include enzyme assay demonstrating low GAA activity, DNA testing, or increased lysosomal glycogen.