CurrentNeighborhood Health Plan of Rhode IslandPolicy N/A

Enzyme Replacement Therapy (ERT) for Fabry Disease: Fabrazyme (agalsidase beta), Elfabrio (pegunigalsidase alfa)

Policy governing coverage, dosing, and authorization criteria for intravenous enzyme replacement therapies Fabrazyme (agalsidase beta) and Elfabrio (pegunigalsidase alfa) for members of Neighborhood Health Plan of Rhode Island across Medicaid, Commercial, and Medicare lines.

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEnzyme Replacement Therapy (ERT) for Fabry Disease: Fabrazyme, Elfabrio

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJan 1, 2021

Next Review DateApr 22, 2021

Key ActionObtain prior authorization and document diagnostic confirmation (biochemical or genetic) plus baseline biomarkers and dosing per weight for initial approval.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

6 monthsInitial authorization length

115Fabrazyme max units/14d

120Elfabrio max units/14d

E75.21Covered ICD-10

J0180, J2508HCPCS listed

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Coverage Criteria for Fabrazyme and Elfabrio

Initial Therapy — Covered when ALL of the following are met

Covered when ALL of the following are met:

Age and product-specific age limits: Member is at least 2 years of age if request is for Fabrazyme, OR member is at least 18 years of age if request is for Elfabrioage

from policy

Specialist involvement: Medication is prescribed by or in consultation with a medical geneticist, nephrologist, or other physician who specializes in the treatment of Fabry disease

Universal criteria

No combination ERT or migalastat use: Must not be used in combination with migalastat (Galafold) or another enzyme replacement therapy (Fabrazyme or Elfabrio)

Combination therapy prohibited

Diagnostic confirmation: Documented diagnosis of Fabry disease with biochemical or genetic confirmation by either α‑Gal A activity (males) OR detection of a pathogenic GLA mutationlaboratory/genetic

Also requires baseline biomarkers, tissue inclusions, or clinical signs/symptoms

Baseline disease evidence (one required): Baseline of one or more of: tissue Gb3/GL‑3 inclusions; plasma or urinary Gb3/GL‑3; or plasma lysoGb3; OR clinical signs/symptoms consistent with Fabry disease

One required

Prior migalastat requirement for some members: Member has had an inadequate response, intolerance, or contraindication to Galafold (migalastat)

Applies only to Medicaid and Commercial members

General Coverage Criteria — Covered when ALL of the following are met

Coverage renewal requires ALL of the following:

Continued eligibility: Member continues to meet universal and other indication-specific relevant criteria identified in the Initial Approval section

See section IV

Absence of unacceptable toxicity: No unacceptable toxicity from the drug (examples include anaphylaxis, severe hypersensitivity reactions, severe infusion-associated reactions, glomerulonephritis)

Presence of such toxicity may preclude renewal

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The policy explicitly prohibits concurrent administration of enzyme replacement therapy with migalastat or with another ERT formulation. Requests for use of an ERT in combination with migalastat or simultaneous use of two ERT products are not permitted under the coverage criteria.

For Medicare members, the plan defers to applicable Medicare coverage determinations: where a National Coverage Determination (NCD) or Local Coverage Determination (LCD) exists, the NCD/LCD governs and the plan’s coverage criteria do not apply. Providers should consult the CMS coverage database and comply with any relevant NCD/LCD when submitting requests.

Requests for combination therapy with migalastat or concurrent use of two enzyme replacement therapies (for example, Fabrazyme together with Elfabrio) are considered not medically necessary under this policy. The plan’s coverage criteria require single-agent use consistent with FDA labeling and adopted clinical guidance.

Coverage is limited to uses that are consistent with FDA-approved labeling and relevant clinical literature as adopted by the plan. Each request receives individual consideration based on prescriber-submitted information and other plan-available information to ensure use is for a medically accepted indication.

Coding, Dosing, and Unit Thresholds

HCPCS codesHCPCSCovered

J0180	Injection, agalsidase beta, 1 mg
J2508	Injection, pegunigalsidase alfa-iwxj, 1 mg

NDCNDC

58468-0041-xx	Fabrazyme 5 mg single-use vial for injection
58468-0040-xx	Fabrazyme 35 mg single-use vial for injection
10122-0160-xx	Elfabrio 20 mg/10 mL single-use vial for injection

Covered Diagnosis Codes (ICD-10)ICD-10Covered

E75.21

Fabry (-Anderson) disease

Medicare Part B Covered Diagnosis CodesICD-10

N/A	Medicare Part B Covered Diagnosis Codes (applicable to existing NCD/LCD)

Medicare MAC Jurisdictionsmixed

Multiple MAC jurisdictions

Medicare Part B Administrative Contractor (MAC) jurisdictions and contractors listed by region (see Appendix for state/contractor mapping)

Dosing and unit thresholds

Standard dosing1 mg/kg administered intravenously every 2 weeks

Max billable units — Fabrazyme115 billable units per 14 days (HCPCS unit = 1 mg)

Max billable units — Elfabrio120 billable units per 14 days (HCPCS unit = 1 mg)

Prior Authorization, Documentation, and Renewal Requirements

Prior Authorization

Authorization length and Medicare prior‑treatment exception

Coverage is provided for an initial authorization period of 6 months and may be renewed if renewal criteria are met. Medicare members who have previously received this medication within the past 365 days are exempt from Step Therapy / prerequisite therapy requirements (Medicare prior‑treatment exception).

Initial authorization length: 6 months
Medicare prior‑treatment exception: prior receipt within 365 days exempts member from step therapy requirements

Prior Authorization

Prior Authorization Required — FDA‑aligned indications

Prior authorization is required. Coverage is limited to uses consistent with FDA‑approved labeling and relevant clinical literature; requests will be reviewed for medical necessity and alignment with the policy criteria.

Coverage aligned to FDA‑approved indications and evidence-based literature
Individual prior authorization review required

Background and Scope

Fabry disease is a rare X-linked lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A, resulting in accumulation of glycosphingolipids such as Gb3/GL-3 and lyso-Gb3 in multiple organs. Clinical manifestations include renal, cardiac, neurologic, dermatologic and other findings. Enzyme replacement therapies (agalsidase beta and pegunigalsidase alfa) are used to reduce tissue substrate accumulation and may stabilize organ function; infusion-associated and hypersensitivity reactions can occur. Diagnosis is generally confirmed by biochemical or genetic testing (reduced α-Gal A activity in males or pathogenic GLA mutation) with supporting biomarkers or tissue findings.

Definitions and Diagnostic Criteria

Confirmed Fabry disease

Diagnostic confirmation — malesLow α‑Gal A activity measured in plasma, isolated leukocytes, or cultured cells

Diagnostic confirmation — all sexesDetection of a pathogenic GLA gene mutation by molecular genetic testing

Baseline supporting evidenceAt least one of: tissue Gb3/GL‑3 inclusions; plasma or urinary Gb3/GL‑3; or plasma lysoGb3; or clinical signs/symptoms consistent with Fabry disease

ERT for Fabry Disease

DefinitionEnzyme Replacement Therapy (ERT) — intravenous administration of exogenous alpha‑galactosidase A (e.g., agalsidase beta [Fabrazyme] or pegunigalsidase alfa [Elfabrio]) to treat confirmed Fabry disease, used in accordance with FDA‑approved labeling and relevant clinical literature

Purpose

Policy Summary

PayerNeighborhood Health Plan of Rhode Island

PolicyEnzyme Replacement Therapy (ERT) for Fabry Disease: Fabrazyme, Elfabrio

Policy CodePolicy N/A

Change TypeNo material change

Effective DateJan 1, 2021

Next Review DateApr 22, 2021

Key ActionObtain prior authorization and document diagnostic confirmation (biochemical or genetic) plus baseline biomarkers and dosing per weight for initial approval.

SourceLink

On This Page

Denial Risk

Medicare compliance and NCD/LCD precedence

For Medicare members, noncompliance with applicable Medicare National Coverage Determinations (NCDs) or Local Coverage Determinations (LCDs) may result in denial of coverage. Where an NCD/LCD exists, those determinations supersede plan criteria.

Medicare NCD/LCDs take precedence over plan criteria when applicable
Noncompliance with NCD/LCD requirements may lead to denial

Documentation Required

Provider submission guidance

Prescribers should submit documentation supporting the FDA‑approved indication or relevant clinical literature and any information needed for individual consideration. Neighborhood may request additional documentation to determine medical necessity.

Required submission: diagnosis confirmation, baseline biomarkers or tissue findings, prior treatment history, and rationale for therapy
Neighborhood may request additional records to support the request

Step Therapy

Step therapy and cost‑effectiveness intent

The criteria are intended to ensure clinically appropriate and cost‑effective options are used first, when applicable and appropriate. Step therapy and prerequisite therapy requirements apply except where Medicare prior‑treatment exceptions or other policy provisions exempt the member.

Aim: encourage use of cost‑effective therapeutic options before higher‑cost therapies when clinically appropriate

Documentation Required

Required diagnostic, baseline, and drug documentation

Documented diagnosis confirmation (for example, α‑Gal A activity in males or a pathogenic GLA mutation), baseline biomarkers or tissue findings (e.g., GL‑3/ Gb3, lyso‑Gb3, or tissue inclusions), and other supporting clinical evidence should be provided with the prior authorization request.

Diagnosis confirmation: α‑Gal A activity in males or pathogenic GLA mutation
Baseline evidence: tissue GL‑3/Gb3 inclusions, plasma/urinary Gb3, plasma lyso‑Gb3, or clinical signs/symptoms

Denial Risk

Renewal requirements and toxicity-related denial risk

Coverage renewal requires continued meeting of the policy’s renewal criteria and absence of unacceptable toxicity (e.g., anaphylaxis, severe hypersensitivity, severe infusion‑associated reactions).

Renewal contingent on documented disease response or stabilization compared to baseline and no unacceptable toxicity

Reduce tissue substrate accumulation (Gb3/GL‑3, lysoGb3) and stabilize organ function in patients with confirmed Fabry disease

Coverage contextNeighborhood aligns ERT use with FDA labeling and relevant clinical literature; individual prior authorization review is performed