CurrentCareSourcePolicy N/A

Lumizyme (alglucosidase alfa) — Coverage Criteria (Arkansas PASSE)

Policy governs prior authorization and medical benefit coverage of Lumizyme (alglucosidase alfa) for treatment of Pompe disease for members served under CareSource (Arkansas PASSE). Affects prescribers and facilities requesting coverage.

Policy Summary

PayerCareSource

PolicyLumizyme (alglucosidase alfa) — Coverage Criteria (Arkansas PASSE)

Policy CodePolicy N/A

Change TypeAnnual review — no material changes noted

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and ensure prescription is by or in consultation with an appropriate specialist and that diagnosis is confirmed by enzyme assay and molecular genetic testing.

SourceLink

POLICY UPDATE CHANGES

Revised date noted as 11/22/2023; annual review entries indicate no changes at those reviews.

20 mg/kgapproved dose per infusion

q2wdosing interval

12 moinitial approval

12 moreauthorization

Coverage Criteria for Lumizyme (alglucosidase alfa)

inv-01: Initial Therapy

Covered when ALL of the following are met for initial authorization

Initial Authorization - All required: 1) Medication prescribed by or in consultation with a geneticist, cardiologist, neurologist, pulmonologist, or metabolic specialist; AND 2) Diagnosis of Pompe disease confirmed by an enzyme activity assay showing GAA deficiency (0% to 40% of normal); AND 3) Molecular genetic testing demonstrates a pathogenic mutation of the GAA gene; AND 4) For late-onset Pompe disease (LOPD), member must have signs or symptoms such as motor weakness or reduced respiratory parameters; AND 5) Dosage does not exceed 20 mg/kg IV infusion every 2 weeks.

If all requirements are met, approve for 12 months.

inv-02: Continuation Therapy / Reauthorization

Covered for reauthorization when ALL of the following are met

Reauthorization - All required: Chart notes document positive clinical response such as improved or stabilized motor function or ambulation, pulmonary function, or cardiomyopathy.

Policy Summary

PayerCareSource

PolicyLumizyme (alglucosidase alfa) — Coverage Criteria (Arkansas PASSE)

Policy CodePolicy N/A

Change TypeAnnual review — no material changes noted

Effective DateN/A

Next Review DateN/A

Key ActionObtain prior authorization and ensure prescription is by or in consultation with an appropriate specialist and that diagnosis is confirmed by enzyme assay and molecular genetic testing.

SourceLink

On This Page

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If met, approve for an additional 12 months.

CareSource considers Lumizyme (alglucosidase alfa) not medically necessary for any indication that is not specifically listed in this policy. Off‑label uses or requests for indications outside those described should be evaluated under the plan’s Off‑Label policy and will generally not be approved under this coverage document.

Treatment of conditions not included in this document is considered not medically necessary. Providers seeking coverage for uses beyond the stated indications must follow the Off‑Label policy and provide supporting clinical justification; absent that, requests for unlisted indications will be denied.

Initial Authorization Requirements

inv-12: Initial Authorization

Initial authorization requirements

Initial authorization: Medication prescribed by or in consultation with an appropriate specialist (geneticist, cardiologist, neurologist, pulmonologist, or metabolic specialist); AND diagnosis of Pompe disease confirmed by enzyme activity assay showing GAA deficiency (0%–40% of normal); AND molecular genetic testing showing a pathogenic GAA mutation; AND for LOPD, presence of signs/symptoms (eg, motor weakness, reduced respiratory parameters); AND dosing at or below 20 mg/kg IV every 2 weeks.

Approve for 12 months if all criteria met.

Reauthorization / Continuation Requirements

inv-13: Reauthorization

Reauthorization requires documentation of clinical benefit.

Reauthorization criteria: Chart notes must document positive clinical response (improved or stabilized motor function or ambulation, pulmonary function, or cardiomyopathy).

Approval for additional 12 months if met.

Provider Requirements & Prior Authorization

Prior Authorization

Prior Authorization & Prescriber Specialty

Prior authorization required. Lumizyme (alglucosidase alfa) is a medical benefit and requires prior authorization before coverage. The medication must be prescribed by, or in consultation with, an appropriate specialist (geneticist, cardiologist, neurologist, pulmonologist, or metabolic specialist).

Benefit type: Medical
Specialist prescriber or documented consultation required: geneticist, cardiologist, neurologist, pulmonologist, or metabolic specialist
Prior authorization duration: up to 12 months for initial approval

Note

Provider Submission Requirements

Providers must submit documentation of diagnosis confirmation (enzyme activity assay showing GAA deficiency and molecular genetic testing demonstrating pathogenic GAA mutation), signs/symptoms for late-onset disease (e.g., motor weakness, reduced respiratory parameters), and proposed dosing (20 mg/kg IV every 2 weeks). Include relevant chart notes, baseline assessments, and specialist consultation notes with the prior authorization request.

Required diagnostic documentation: enzyme activity assay (0–40% of normal) and GAA gene mutation testing
Dosing/quantity limit to request: 20 mg/kg IV every 2 weeks
For LOPD, document signs/symptoms such as motor weakness or reduced respiratory parameters

Documentation Required

Reauthorization Documentation

For reauthorization, chart notes must document a positive clinical response — for example improved or stabilized motor function/ambulation, pulmonary function, or cardiomyopathy — to support continued coverage. Reauthorization approvals are granted in 12-month increments when criteria are met.

Reauthorization documentation: clinical response documented in chart notes (improved or stabilized motor function/ambulation, pulmonary function, or cardiomyopathy)
Reauthorization approval period: 12 months

Denial Risk

Denial Risk for Missing Requirements

Requests that do not meet required clinical criteria may be denied. Common denial risks include missing enzyme assay or genetic test confirmation, lack of specialist prescriber/consultation, failure to document signs/symptoms for late-onset Pompe disease, or absence of documented clinical response at reauthorization.

Denial risk if enzyme activity assay or GAA gene mutation testing not provided
Denial risk if no specialist prescriber or documentation of consultation
Denial risk for missing clinical signs/symptoms in LOPD or missing reauthorization response documentation

Dose and Quantity Limits

Lumizyme (alglucosidase alfa) — Approved Dose and Limits

Approved dose per infusion20 mg/kg IV

Dosing intervalEvery 2 weeks

Quantity limit statementDosage allowed/Quantity limit: 20 mg/kg IV infusion every 2 weeks

Approval duration when criteria metInitial approval: 12 months

Key Definitions

Infantile-onset Pompe disease (IOPD)

DefinitionOnset within the first few months of life; classic form is rapidly progressive with severe cardiomyopathy; non-classic IOPD progresses more slowly

Typical featuresSevere cardiomyopathy (classic IOPD), rapid progression of muscle weakness

Diagnostic relevanceClassified as infantile-onset Pompe disease (IOPD) when symptoms begin in infancy

Late-onset Pompe disease (LOPD)

DefinitionOnset beyond infancy; generally does not present with cardiomyopathy and has variable skeletal muscle weakness and respiratory involvement

Required for coverage (symptoms)Members with late-onset Pompe disease must show signs or symptoms (e.g., motor weakness, reduced respiratory parameters)

Diagnostic testingDiagnosis confirmation requires enzyme assay showing GAA deficiency and molecular genetic testing for pathogenic GAA mutation

Background on Pompe Disease and Therapy

Pompe disease (acid alpha‑glucosidase [GAA] deficiency) is a rare lysosomal storage disorder in which deficient GAA enzyme activity leads to glycogen accumulation and progressive muscle damage. The disease presents across a spectrum from infantile‑onset Pompe disease (IOPD)—often rapidly progressive with cardiomyopathy—to late‑onset Pompe disease (LOPD), which typically manifests as variable skeletal muscle weakness and respiratory involvement. Lumizyme is enzyme replacement therapy (alglucosidase alfa) intended to supply the deficient GAA enzyme and mitigate progressive muscle and cardiopulmonary decline.

Policy Revision History

2023-11-22annual reviewLatest

Annual review completed with no material changes to the clinical policy for Lumizyme.

2021-07-07annual review

Annual review completed with no material changes; policy created for Lumizyme.