ModifiedmhnPolicy CP.PHAR.158

Clinical Policy: Agalsidase Beta (Fabrazyme)

Defines medical necessity and authorization criteria for use of Fabrazyme (agalsidase beta) in members with Fabry disease for commercial, ICHRA/HIM, and Medicaid lines of business. Affects prescribers and providers requesting coverage for Fabrazyme.

Policy Summary

Payermhn

PolicyClinical Policy: Agalsidase Beta (Fabrazyme)

Policy CodePolicy CP.PHAR.158

Change TypeMultiple material clarifications & updates

Effective Date02.16

Next Review Date

Key ActionSubmit prior authorization with documentation including confirmed diagnosis and the member's current weight for dose calculation.

SourceLink

POLICY UPDATE CHANGES

Updated initial approval duration from 6 months to 12 months.

Added requirement for documentation of member's weight for dose calculation.

Added explicit exclusion for concomitant use with Elfabrio (and Galafold).

For continued therapy, added examples of positive treatment response (previously in Appendix D) into the criteria section.

Added ICHRA line of business.

1 mg/kg q2wMaximum dosing regimen

≥2 yrsMinimum covered age

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Coverage and Medical Necessity Criteria

Non–FDA approved indications that are not specifically addressed in this policy are not authorized unless the provider supplies sufficient documentation of efficacy and safety consistent with the applicable off‑label use policy: CP.CPA.09 (commercial), HIM.PA.154 (health insurance marketplace/ICHRA), or CP.PMN.53 (Medicaid).

Concomitant prescribing of Fabrazyme with Elfabrio is explicitly excluded and may result in denial of the request. This exclusion was added to align this policy with the Elfabrio criteria and is enforced for both initial and continued therapy requests.

Use of Fabrazyme for non–FDA approved indications without adequate supporting documentation is considered not authorized under the policy. Providers should follow the referenced off‑label and formulary/non‑formulary policies for the applicable line of business prior to requesting coverage for off‑label uses.

Initial Therapy Requirements

inv-21: Initial Therapy

Initial approval requires all criteria be met:

Initial therapy nodes: See Coverage.criteria_sets Initial Therapy

Includes prescriber specialty, age, diagnosis confirmation, weight documentation, concomitant therapy exclusions, dose limit

inv-22: Initial therapy duration

Initial approval duration updated.

Initial approval duration: Initial authorization duration: Medicaid/HIM/ICHRA — 12 months; Commercial — 6 months or to member renewal (updated initial approval duration to 12 months where applicable)

See prior authorization section

Criteria for Continued Therapy

inv-23: Continuation Therapy

Criteria required for continued coverage of Fabrazyme:

Continuation nodes: See Coverage.criteria_sets Continued Therapy

Includes evidence of positive response and weight/dose limits

inv-24: Continued therapy requirements

Continued therapy requires documentation of positive treatment response; examples were added.

Documentation of response: Documentation must show positive clinical response to therapy; examples of improvement include reduction in extremity pain, improved gastrointestinal symptoms (diarrhea, abdominal pain, nausea, vomiting), improved renal function, reduction in neuropathic symptoms, improved fatigue, or resolution of cornea verticillata

Examples align with Appendix D and were added into the continued therapy criteria

Billing and Coding

HCPCS CodesHCPCSCovered

J0180

Injection, agalsidase beta, 1 mg

Covered HCPCS CodesHCPCSCovered

J0180

Injection, agalsidase beta, 1 mg

inv-09: Maximum dosing frequency/dose

Maximum dosing frequency/dose1 mg/kg every 2 weeks

Administration routeIntravenous (IV)

Dose contextMaximum dose specified for Fabry disease treatment

inv-20: J0180 — HCPCS J0180 represents injection of agalsidase beta, 1 mg.

HCPCS codeJ0180

DescriptionInjection, agalsidase beta, 1 mg

Billing unit

Prior Authorization, Documentation, and Denial Triggers

Prior Authorization

Prior Authorization Required

Prior authorization required. Initial approval duration: Medicaid/HIM/ICHRA — 12 months; Commercial — 6 months or to the member's renewal date, whichever is longer. Initial approval duration updated to 12 months for all applicable lines of business per 2Q 2026 review.

Medicaid/HIM/ICHRA: 12 months
Commercial: 6 months or to member's renewal date, whichever is longer

Documentation Required

Required Documentation

Provider must submit documentation (such as office chart notes, lab results, or other clinical information) supporting that the member has met all approval criteria. Documentation of the member's current weight (in kg) is required for dose calculation purposes.

Office chart notes
Laboratory results (including enzyme assay or DNA testing when applicable)

Clinical Background

Fabry disease is an X‑linked lysosomal storage disorder caused by deficiency of the enzyme alpha‑galactosidase A, leading to accumulation of globotriaosylceramide and multi‑system manifestations. Fabrazyme (agalsidase beta) is a recombinant human alpha‑galactosidase A enzyme indicated for treatment of confirmed Fabry disease in patients aged ≥ 2 years. The recommended dosing regimen is 1 mg/kg administered intravenously every 2 weeks, and clinical responses that may support continued therapy include improvements in neuropathic pain, gastrointestinal symptoms, renal function measures, dermatologic findings, and other disease‑specific manifestations.

Product and Coding Definitions

inv-19: Fabrazyme / agalsidase beta — Product definition and indication summary.

Product name (generic)Agalsidase beta (Fabrazyme)

DescriptionRecombinant human alpha-galactosidase A enzyme

FDA indicationTreatment of confirmed Fabry disease in patients aged ≥ 2 years

Key initial therapy requirementsConfirmed diagnosis by enzyme assay or DNA testing; prescribed by or in consultation with an appropriate specialist; documentation of current weight; not used concurrently with Galafold or Elfabrio

inv-20: J0180 — HCPCS code definition for agalsidase beta 1 mg.

HCPCS codeJ0180

Step Therapy and Off-Label Guidance

Requirement	Action/Policy
Use not listed in criteria or follows recent label change	Refer to applicable formulary/non‑formulary or off‑label policies before coverage (e.g., CP.CPA.190, HIM.PA.103, CP.PMN.16 for non‑formulary; CP.CPA.09, HIM.PA.154, CP.PMN.53 for off‑label).

Dose Limits and Administration

inv-26: agalsidase beta (Fabrazyme) — Dose limited to 1 mg/kg every 2 weeks.

Quantity limitDose limited to 1 mg/kg every 2 weeks

Dose intervalEvery 2 weeks (14 days)

Weight-based dosingDose calculated using member's current weight (kg)

Administration Site

Note

IV administration per dosing instructions (site not restricted)

Intravenous administration per the dosing instructions is required; the policy does not restrict the infusion site within this section.

Administer IV at recommended dose: 1 mg/kg every 2 weeks.

Policy Summary

Payermhn

PolicyClinical Policy: Agalsidase Beta (Fabrazyme)

Policy CodePolicy CP.PHAR.158

Change TypeMultiple material clarifications & updates

Effective Date02.16

Next Review Date

Key ActionSubmit prior authorization with documentation including confirmed diagnosis and the member's current weight for dose calculation.

SourceLink

On This Page

Denial Risk

Triggers for Denial

Requests may be denied if the diagnosis of Fabry disease is not confirmed by enzyme assay demonstrating alpha-galactosidase deficiency or by DNA testing, if the request is for concomitant use with Elfabrio or Galafold, if member weight is not documented for dose calculation, or if prescribed by a provider who is not a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist.

Diagnosis not confirmed by enzyme assay or DNA testing
Concurrent prescription with Elfabrio or Galafold
Failure to document member weight for dose calculation
Prescribed by an inappropriate provider specialty

Note

Policy Cross-Reference for Non-Listed Uses

If the requested use (e.g., diagnosis, age, dosing regimen) is not listed in the policy or the drug is not on the formulary/PDL for the applicable line of business, refer to the relevant non-formulary or off-label use policies for coverage determination.

Non-formulary policies: CP.CPA.190 (commercial), HIM.PA.103 (HIM/ICHRA), CP.PMN.16 (Medicaid)
Off-label use policies: CP.CPA.09 (commercial), HIM.PA.154 (HIM/ICHRA), CP.PMN.53 (Medicaid)