ModifiedmhnPolicy CP.PHAR.160

Alglucosidase Alfa (Lumizyme) coverage

Defines medical necessity and authorization criteria for Lumizyme (alglucosidase alfa) for treatment of Pompe disease for Centene-affiliated health plans across commercial, HIM, and Medicaid lines of business.

Policy Summary

Payermhn

PolicyAlglucosidase Alfa (Lumizyme) coverage

Policy CodePolicy CP.PHAR.160

Change TypeMaterial updates to exclusions and diagnostic confirmation options

Effective DateFeb 1, 2016

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming Pompe disease diagnosis, dosing within 20 mg/kg q2w, and absence of excluded concurrent therapies.

SourceLink

POLICY UPDATE CHANGES

Requirement that Lumizyme not be prescribed concurrently with Nexviazyme.

Exclusion for concomitant use with the combination of Pombiliti with Opfolda.

Increased lysosomal glycogen listed as an additional option for confirming a Pompe disease diagnosis.

20 mg/kg q2wmaximum recommended dose

J0220, J0221HCPCS codes referenced

6 monthsinitial approval duration

12 monthscontinued therapy duration

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diagnostic option added

Coverage Criteria

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial Approval - Pompe Disease: Diagnosis of Pompe disease (GAA deficiency) confirmed by one of: a) enzyme assay confirming low GAA activity; b) DNA testing; c) increased lysosomal glycogen.

Concurrent Therapy Exclusion: Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda.

Dosing Limit: Dose does not exceed 20 mg/kg every 2 weeks.<= 20 mg/kg every 2 weeks

inv-02: Continuation Therapy

Covered for continuation when ALL of the following are met:

Eligibility: Member meets one of: a) currently receiving medication via the Centene benefit or previously met initial approval criteria; b) currently receiving medication and enrolled in a state/product with continuity of care regulations.

Refer to state-specific addendums for continuity of care.

Clinical Response: Member is responding positively to therapy as evidenced by improvement in the individual's Pompe disease manifestation profile (see Appendix D for examples such as survival/motor gains for infantile-onset; improved/maintained FVC or 6MWT for late-onset).

See Appendix D for measures of therapeutic response.

Concurrent Therapy Exclusion: Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda.

Dose on Continuation: If request is for a dose increase, new dose does not exceed 20 mg/kg every 2 weeks.<= 20 mg/kg every 2 weeks

Coverage for uses of Lumizyme that are not FDA‑approved is limited. Non‑FDA indications that are not specifically addressed in this policy are not authorized unless the request includes sufficient documentation of efficacy and safety and the use is supported under the applicable off‑label use policy: CP.CPA.09 (Commercial), HIM.PA.154 (Health Insurance Marketplace), or CP.PMN.53 (Medicaid), or is otherwise supported by an evidence of coverage document.

Concurrent prescription of Lumizyme with Nexviazyme is explicitly prohibited. The policy requires that Lumizyme is not prescribed concurrently with Nexviazyme, and this exclusion was added during the 2Q 2022 review cycle.

Lumizyme use is excluded when prescribed at the same time as the combination of Pombiliti with Opfolda. The policy states that Lumizyme must not be used concomitantly with Pombiliti+Opfolda; this exclusion was added to align with Pombiliti criteria in the 2Q 2024 review cycle.

Requests for Lumizyme for indications that are not FDA‑approved and that are not supported by the referenced off‑label use policies or adequate documentation of efficacy and safety are not authorized. Providers seeking coverage for off‑label uses must follow the applicable off‑label policy pathway and submit sufficient supporting clinical evidence.

Initial Therapy Criteria

inv-20: Initial Therapy

Initial approval requires diagnosis confirmation, exclusion of concurrent specified agents, and adherence to dosing limits.

Diagnosis Confirmation: Diagnosis of Pompe disease (GAA deficiency) confirmed by one of: enzyme assay confirming low GAA activity; DNA testing; or increased lysosomal glycogen.

Increased lysosomal glycogen was added as an additional diagnostic option in recent review cycles.

Concurrent Therapy Exclusion: Lumizyme must not be prescribed concurrently with Nexviazyme or with the combination of Pombiliti and Opfolda.

Concurrent-use exclusions were added in 2Q 2022 and 2Q 2024 review updates.

Dosing Limit: Dose does not exceed 20 mg/kg every 2 weeks.<= 20 mg/kg every 2 weeks

Continuation / Ongoing Therapy Criteria

inv-22: Continuation Therapy

Continuation of therapy requires evidence of benefit and non-concurrent prohibited therapies.

Ongoing Eligibility: Member is currently receiving medication via the Centene benefit or previously met initial approval criteria, or is subject to continuity of care provisions.

See state-specific continuity of care addendums as applicable.

Documented Clinical Benefit: Member demonstrates clinical response to therapy as shown by appropriate measures (examples: infantile-onset—survival and motor gains; late-onset—improved/maintained FVC or 6MWT).

Appendix D lists examples of therapeutic response measures.

Concurrent Therapy Exclusion: Lumizyme is not prescribed concurrently with Nexviazyme or the combination of Pombiliti with Opfolda.

Concurrent-use exclusions may lead to denial per policy updates.

Coding and Dosing

HCPCS CodesHCPCS

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

inv-08: Maximum dosing — 20 mg/kg every 2 weeks

Maximum dose20 mg/kg every 2 weeks

Dosing regimen (Pompe disease)20 mg/kg IV every 2 weeks

Reference sectionDosage and Administration (Indication 1 and 2)

inv-25: alglucosidase alfa (Lumizyme) — 20 mg/kg per dose (quantity limits)

Quantity limit per dose20 mg/kg per dose

Dosing intervalEvery 2 weeks

Product presentation relevant to dosing

Provider Actions and Requirements

Prior Authorization

Prior Authorization Required

Prior authorization is required. Requests will be reviewed against the policy criteria and plan prior authorization procedures; initial approval is granted only when all approval criteria are met.

PA required for Lumizyme for Pompe disease
Initial approvals granted when all listed criteria are satisfied
Dose limit: ≤ 20 mg/kg every 2 weeks

Denial Risk

Documentation and Denial Risk

Provider must submit clinical documentation supporting that the member meets all approval criteria. Requests that lack required documentation or fail to demonstrate that listed criteria are met may be denied.

Include office chart notes, relevant lab results (e.g., enzyme assay showing low GAA activity), DNA testing results, or pathology confirming increased lysosomal glycogen

Step Therapy / Formulary Pathway

Scenario	Action / Requirement
Requested indication, age, or dosing regimen is newly changed within the last 6 months and not yet reflected in this policy
Follow the appropriate formulary or non‑formulary policy for the member's line of business: commercial — CP.CPA.190; health insurance marketplace — HIM.PA.33 (formulary) or HIM.PA.103 (non‑formulary); Medicaid — CP.PMN.255 (formulary) or CP.PMN.16 (non‑formulary).
Requested use is not listed in this policy and criterion 1 (recent label change) does not apply
Refer to the off‑label use policy for the member's line of business before approving: commercial — CP.CPA.09; health insurance marketplace — HIM.PA.154; Medicaid — CP.PMN.53.

Site of Care

Note

Site of care not specified

The policy does not specify a site‑of‑care restriction; infusion center/site of care is not defined in this section of the policy.

Background

Pompe disease is caused by deficiency of the enzyme acid alpha‑glucosidase (GAA)

Definitions and Diagnostic Notes

inv-18: acid alpha-glucosidase — GAA definition

Definition (abbreviation)GAA = acid alpha-glucosidase

Clinical contextGAA deficiency causes Pompe disease due to impaired lysosomal glycogen breakdown

FDA indicationLumizyme indicated for Pompe disease (acid alpha-glucosidase [GAA] deficiency)

inv-19: Diagnostic confirmation option — increased lysosomal glycogen as an additional option for confirming Pompe disease diagnosis

Additional diagnostic confirmation optionIncreased lysosomal glycogen as an option to confirm Pompe disease diagnosis

Other accepted confirmation methodsEnzyme assay confirming low GAA activity or DNA testing

Policy Summary

Payermhn

PolicyAlglucosidase Alfa (Lumizyme) coverage

Policy CodePolicy CP.PHAR.160

Change TypeMaterial updates to exclusions and diagnostic confirmation options

Effective DateFeb 1, 2016

Next Review Date

Key ActionObtain prior authorization and submit documentation confirming Pompe disease diagnosis, dosing within 20 mg/kg q2w, and absence of excluded concurrent therapies.

SourceLink

On This Page

Maximum recommended dose per product dosing guidance.

inv-21: INITIAL THERAPY CRITERIA

Discrete initial-therapy criteria are templated; diagnostic confirmation options and exclusions are referenced below.

Templated Criteria Note: This policy's initial-therapy criteria follow a template: diagnosis confirmation (enzyme assay, DNA testing, or increased lysosomal glycogen), explicit concurrent-use exclusions, and dosing limits (<=20 mg/kg q2w).

Refer to the Initial Therapy block for full criteria wording.

Dose within Limits: Dose on continuation (including increases) must not exceed 20 mg/kg every 2 weeks.<= 20 mg/kg every 2 weeks

Approval durations vary by line of business (see policy for details).

inv-23: CONTINUATION CRITERIA

Template notes: specific continuation criteria were templated during reviews but no additional discrete continuation requirements are present in the provided chunks.

Template Notice: Continuation/ongoing therapy considerations were templated during policy reviews; however, no further discrete continuation criteria beyond documented clinical benefit, non-concurrent prohibited therapies, and dosing limits are provided in these source chunks.

Refer to the full policy for any jurisdiction-specific continuation requirements.

Single-use 50 mg vial

Insufficient documentation or failure to meet criteria = denial risk

Documentation Required
Required Clinical Documentation
Required clinical documentation includes office notes, laboratory results, genetic testing reports, or other clinical information that verifies the diagnosis and supports the requested use.
Documentation must confirm Pompe disease by one of: enzyme assay (low GAA activity), DNA testing, or increased lysosomal glycogen
Document absence of concurrent use with Nexviazyme or Pombiliti + Opfolda
Include dosing information to confirm dose does not exceed 20 mg/kg every 2 weeks

Documentation Required
Documentation Procedures
No additional provider documentation beyond standard clinical policy review and P&T approvals is required; however, follow the plan's prior authorization procedures and supply requested supporting records when asked.
Follow plan-specific PA submission procedures
Respond to requests for additional records promptly to avoid delay or denial

Note
Off‑Label / Formulary Pathway
When the requested use falls outside the policy or follows a recent label change, follow the applicable formulary/off-label pathways for the member's line of business as specified in the policy.
For formulary drugs or PDL: consult the no coverage criteria policy (CP.CPA.190 commercial, HIM.PA.33 marketplace, CP.PMN.255 Medicaid) if applicable
For non-formulary drugs or PDL: consult the non-formulary policies (CP.CPA.190 commercial, HIM.PA.103 marketplace, CP.PMN.16 Medicaid)
For off-label uses not listed in exclusions: consult off-label use policies (CP.CPA.09 commercial, HIM.PA.154 marketplace, CP.PMN.53 Medicaid)

Policy history noteDiagnostic option (increased lysosomal glycogen) added during 1Q 2025 annual review