CurrentCignaPolicy N/A

Enzyme Replacement Therapy - Fabrazyme

This policy governs prior authorization, coverage criteria, dosing, and exclusions for Fabrazyme (agalsidase beta) intravenous therapy for Fabry disease for members of Cigna-administered health benefit plans.

Policy Summary

PayerCigna

PolicyEnzyme Replacement Therapy - Fabrazyme

Policy CodePolicy N/A

Change TypeTitle updatedosing addedreauthorization removedexclusion added

Effective DateAug 1, 2024

Next Review DateApr 23, 2026

Key ActionObtain prior authorization and document diagnostic confirmation (deficient α-galactosidase A activity or pathogenic GLA variant) and specialist prescribing/consultation.

SourceLink

POLICY UPDATE CHANGES

Updated coverage policy title from Agalsidase to Enzyme Replacement Therapy - Fabrazyme.

Fabry disease dosing was added: each dose must not exceed 1 mg/kg IV no more frequently than every 2 weeks.

Reauthorization criteria were removed.

Concurrent use with Elfabrio (pegunigalsidase alfa) was added as a condition not covered.

1 yearapproval duration

1 mg/kgmax dose

every 2 weeksmin interval

J0180covered code

Elfabrioexplicit exclusion

Fabrazyme Coverage Criteria

Initial therapy (FDA-Approved Indication)

Approve for Fabry disease when ALL of the following are met

FDA-Approved Indication for Fabrazyme: A) Diagnosis is established by ONE of: (i) laboratory test demonstrating deficient α-galactosidase A activity in leukocytes or fibroblasts OR (ii) molecular genetic test demonstrating a pathogenic variant in the galactosidase alpha gene (GLA); AND B) Fabrazyme is prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub-specialist, or physician who specializes in lysosomal storage disorders.Each dose must not exceed 1 mg/kg administered IV no more frequently than once every 2 weeks.

Approve for 1 year if criteria are met.

Diagnostic confirmation of Fabry disease to support Fabrazyme coverage

Coverage requires either deficient α-galactosidase A activity or a pathogenic GLA variant.

ONE of the following diagnostic criteria

Policy Summary

PayerCigna

PolicyEnzyme Replacement Therapy - Fabrazyme

Policy CodePolicy N/A

Change TypeTitle updatedosing addedreauthorization removedexclusion added

Effective DateAug 1, 2024

Next Review DateApr 23, 2026

Key ActionObtain prior authorization and document diagnostic confirmation (deficient α-galactosidase A activity or pathogenic GLA variant) and specialist prescribing/consultation.

SourceLink

On This Page

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Option 1: Laboratory test demonstrating deficient α-galactosidase A activity in leukocytes or fibroblasts.

Option 2: Molecular genetic test demonstrating a pathogenic variant in the galactosidase alpha gene (GLA).

Fabrazyme (agalsidase beta) is covered only for the FDA‑approved indication of Fabry disease when the specific coverage criteria are met. Fabrazyme for any use other than Fabry disease is not medically necessary. Concurrent use of Fabrazyme with certain other Fabry therapies is explicitly excluded (see concurrent use exclusions).

Concurrent administration of Fabrazyme with either Galafold (migalastat) or Elfabrio (pegunigalsidase alfa) is considered not medically necessary. The policy cites limited evidence on safety and efficacy for combined use — for example, a small study (n=23) evaluated a single dose of Galafold given with Fabrazyme but did not establish long‑term safety or effectiveness — and Galafold is not FDA‑approved for concurrent use with Fabrazyme. For these reasons, concurrent therapy with these agents is excluded from coverage.

Covered Indications and Diagnostic Requirements

Diagnostic confirmation of Fabry disease to support Fabrazyme coverage

Coverage requires either deficient α-galactosidase A activity or a pathogenic GLA variant.

ONE of the following diagnostic criteria

Option 1: Laboratory test demonstrating deficient α-galactosidase A activity in leukocytes or fibroblasts.
Option 2: Molecular genetic test demonstrating a pathogenic variant in the galactosidase alpha gene (GLA).

Billing Codes and Dose Limits

Considered Medically Necessary when criteria metHCPCSCovered

J0180

Injection, agalsidase beta, 1 mg

Dose limit (inv-05)

Dose limitEach dose must not exceed 1 mg/kg administered intravenously no more frequently than once every 2 weeks.

Administration routeIntravenous (IV).

Maximum frequencyNo more frequently than once every 2 weeks (minimum interval = 14 days).

Per-dose calculationDose calculated by patient weight (mg/kg).

Approval contextDosing limit applies when approving Fabrazyme for Fabry disease under this policy (FDA‑approved indication).

Provider Actions, Prior Authorization, and Documentation

Prior Authorization

Prior Authorization Required

Prior Authorization is required for Fabrazyme. Approvals may be granted for up to 1 year when the patient meets the policy criteria and dosing. Fabrazyme must be prescribed by or in consultation with an appropriate specialist (geneticist, endocrinologist, metabolic disorder sub-specialist, or physician who specializes in lysosomal storage disorders). Requests for doses outside the dosing specified in the policy are reviewed case-by-case by a clinician (Medical Director or Pharmacist).

Prior authorization required — Approve for up to 1 year when criteria are met
Prescriber requirement — Must be prescribed by or in consultation with a specialist (geneticist, endocrinologist, metabolic disorder sub-specialist, or lysosomal storage disorder specialist)
Dose exceptions — Requests for dosing outside policy dosing reviewed case-by-case

Denial Risk

Concurrent Therapy Exclusions

Concurrent use of Fabrazyme with certain other Fabry therapies is considered not medically necessary.

Concurrent use with Galafold (migalastat) is not medically necessary — long-term safety and efficacy of concurrent use with Fabrazyme has not been established; Galafold is not FDA approved for concurrent use with Fabrazyme
Concurrent use with Elfabrio (pegunigalsidase alfa) is not medically necessary

Documentation Required

Required Clinical Documentation

Documentation must demonstrate confirmation of the diagnosis and supporting clinical information. For males, diagnosis can be confirmed by deficient α-galactosidase A activity in leukocytes or fibroblasts; for all patients, a pathogenic GLA variant on molecular genetic testing is acceptable. Include evidence of specialist involvement as required by the policy.

Required diagnostic documentation — laboratory demonstration of deficient α-galactosidase A activity OR molecular genetic test showing a pathogenic GLA variant
Prescribing clinician documentation — note that Fabrazyme was prescribed by or in consultation with an appropriate specialist

Billing Rule

Billing and Coding Denial Risk

Claims submitted without covered diagnosis or procedure codes (see Coding Information) will be denied as not covered.

Billing and coding denial risk — Claims without covered diagnosis or procedure codes will be denied as not covered
Covered HCPCS for medically necessary use: J0180 (Injection, agalsidase beta, 1 mg)

Member Eligibility Requirements

No top-level eligibility requirement nodes are specified in this policy.

Exclusions and Not Medically Necessary Uses

Concurrent use of Fabrazyme with Galafold (migalastat) or with Elfabrio (pegunigalsidase alfa) is explicitly not covered. The policy identifies insufficient evidence of long‑term safety and efficacy for these combinations and therefore excludes concurrent therapy from coverage.

Background

Fabry disease is an X‑linked lysosomal storage disorder caused by deficient α‑galactosidase A (α‑Gal) activity, resulting in accumulation of GL‑3 in multiple cell types and progressive multisystem disease affecting the kidney, heart, and nervous system. The disorder can present as a severe classical phenotype in males with absent enzyme activity or a milder nonclassical phenotype in persons with residual activity. Diagnosis is confirmed in males by demonstration of deficient α‑Gal activity and in all patients by identification of a pathogenic GLA gene variant. Treatment with Fabrazyme aims to reduce GL‑3 accumulation and slow progression of organ damage.

Definitions

Diagnosis confirmation (inv-11)

Definitive diagnostic options (males)Diagnosis can be confirmed by demonstrating deficient α‑galactosidase A enzyme activity in leukocytes or fibroblasts.

Molecular genetic confirmation (all patients)Identification of a pathogenic variant in the galactosidase alpha gene (GLA) by molecular genetic testing confirms diagnosis.

Sample types for enzyme testingEnzyme activity testing performed on leukocytes or fibroblasts.

Specialist involvementFabrazyme must be prescribed by or in consultation with a geneticist, endocrinologist, metabolic disorder sub‑specialist, or physician specializing in lysosomal storage disorders to meet coverage criteria.

Purpose of confirmationDiagnostic confirmation (enzyme assay or pathogenic GLA variant) is required to support prior authorization and coverage of Fabrazyme.

Revision History

2026-05-15effective_dateLatest

Policy effective date updated (no criteria changes noted).

2025-07-15effective_date

Policy effective date updated (no criteria changes noted).

2024-08-01effective_date

Policy effective date set and material changes implemented: Fabry disease dosing added (each dose ≤1 mg/kg IV no more frequently than every 2 weeks), reauthorization criteria removed, and concurrent use with Elfabrio added as not covered.

2024-06-20review_date

Policy reviewed; documented changes include title update, addition of dosing limits, removal of reauthorization criteria, and addition of Elfabrio as a not-covered concurrent therapy.