ModifiedmhnPolicy CP.PHAR.512

Pegunigalsidase Alfa-iwxj (Elfabrio)

Medical necessity and prior authorization criteria for coverage of Elfabrio (pegunigalsidase alfa-iwxj) in adults with confirmed Fabry disease for members of MHN-affiliated health plans.

Policy Summary

Payermhn

PolicyPegunigalsidase Alfa-iwxj (Elfabrio)

Policy CodePolicy CP.PHAR.512

Change TypeMaterial: coding, duration, documentation

Effective Date05.23

Next Review DateMay 26, 2026

Key ActionSubmit prior authorization with documentation of confirmed Fabry disease, prescriber specialty, member weight (kg), and dosing ≤1 mg/kg every 2 weeks.

SourceLink

POLICY UPDATE CHANGES

Added HCPCS code J2508 for pegunigalsidase alfa-iwxj.

Updated initial approval duration from 6 months to 12 months for Medicaid/HIM and added standard authorization duration language for Commercial.

Added requirement for documentation of member's weight for dose calculation purposes and added/echoed concomitant-use exclusions and examples of positive treatment response in Continued Therapy.

≥18minimum age for coverage

1 mg/kg q2wmaximum labeled dosing

J2508HCPCS code

12 moMedicaid/HIM approval

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Coverage Criteria

inv-01: Initial Therapy

Covered when ALL of the following are met:

Initial Fabry Disease Criteria: 1. Diagnosis of Fabry disease confirmed by one of the following: (a) enzyme assay demonstrating a deficiency of alpha-galactosidase activity; or (b) DNA testing; 2. Prescribed by or in consultation with a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist; 3. Age ≥ 18 years; 4. Elfabrio is not prescribed concurrently with Fabrazyme® or Galafold®; 5. Documentation of member's current weight (in kg); 6. Dose does not exceed 1 mg/kg every 2 weeks.

inv-02: Continuation Therapy

Continued coverage when ALL of the following are met:

Continued Fabry Disease Criteria: 1. Member meets one of the following: (a) currently receiving medication via Centene benefit or previously met initial approval criteria; or (b) member is currently receiving medication and is enrolled in a state/product with continuity of care regulations; 2. Member is responding positively to therapy as evidenced by improvement in the individual's Fabry disease manifestation profile (examples include pain in the extremities; hypohidrosis/anhidrosis; angiokeratomas; diarrhea, abdominal pain, nausea, vomiting, flank pain; renal function; neuropathic pain; heat/cold intolerance; vertigo; diplopia; fatigue; cornea verticillata); 3. Elfabrio is not prescribed concurrently with Fabrazyme® or Galafold®; 4. Documentation of member's current weight (in kg); 5. If request is for a dose increase, new dose does not exceed 1 mg/kg every 2 weeks.

See Appendix D for examples of treatment response.

inv-03: Policy Update Criteria

Policy updates affecting initial and continued therapy include:

Initial Approval Updates: Initial approval duration updated from 6 months to 12 months for Medicaid/HIM; standard authorization duration language added for Commercial; documentation of member weight required for dose calculation.

See initial therapy section for full medical necessity criteria.

Continued Therapy Updates: Added concomitant-use exclusion to Continued Therapy to mirror Initial Approval Criteria and provided explicit examples of positive treatment response (referenced from Appendix D).

See continued therapy section and Appendix D for response examples and exclusion details.

Non‑FDA–approved indications that are not specifically addressed in this policy are not authorized for coverage unless the provider supplies sufficient supporting documentation of efficacy and safety per the applicable off‑label use policies: CP.CPA.09 (Commercial), HIM.PA.154 (Marketplace/ICHRA), or CP.PMN.53 (Medicaid), or unless coverage is provided by the member’s evidence of coverage documents.

A concomitant‑use exclusion was incorporated into the Continued Therapy criteria to mirror the exclusion in Initial Approval. Specifically, Elfabrio may not be prescribed concurrently with excluded therapies (historically including Galafold and Fabrazyme) as described in the policy; this change is documented in the policy update notes and intended to align continued use requirements with initial approval exclusions.

Uses of pegunigalsidase alfa‑iwxj (Elfabrio) for diagnoses or indications that are not FDA‑approved and that lack sufficient supporting documentation per the referenced off‑label policies are considered not authorized for coverage under this policy.

Coding

HCPCS CodesHCPCS

J2508

Injection, pegunigalsidase alfa-iwxj, 1 mg

Covered HCPCS CodesHCPCSCovered

J2508

Injection, pegunigalsidase alfa-iwxj, 1 mg

inv-09: Maximum dose — 1 mg/kg every 2 weeks

Maximum dose1 mg/kg every 2 weeks

Dosing routeIntravenous (IV)

Dose calculation requirementDocumentation of member's current weight (kg) required for dose calculation

Provider Actions & Requirements

Prior Authorization

Prior Authorization Required — Initial Authorization Criteria

Prior authorization is required. Provider must document that the member meets ALL Initial Authorization Criteria for Fabry disease before coverage is approved.

Diagnosis of Fabry disease confirmed by enzyme assay demonstrating deficient alpha-galactosidase activity or by DNA testing
Prescribed by or in consultation with a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist
Member age ≥ 18 years
Elfabrio is not prescribed concurrently with Fabrazyme® or Galafold®
Documentation of member's current weight (kg) for dose calculation
Dose does not exceed 1 mg/kg every 2 weeks
Approval duration: Medicaid/HIM/ICHRA — 12 months; Commercial — 6 months or to the member's renewal date, whichever is longer

Definitions

inv-19: Elfabrio (pegunigalsidase alfa-iwxj) — product definition, mechanism, boxed warnings, and key product details.

Product Name (generic/brand)Pegunigalsidase alfa-iwxj (Elfabrio)

Mechanism / classPEGylated recombinant enzyme replacement therapy — hydrolytic lysosomal neutral glycosphingolipid-specific enzyme

Boxed warning / major safety concernHypersensitivity reactions including anaphylaxis (boxed warning)

Indication (policy context)Treatment of adults with confirmed Fabry disease

Dosage summaryMaximum labeled dosing: 1 mg/kg IV every 2 weeks

inv-20: J2508 — HCPCS J2508: Injection, pegunigalsidase alfa-iwxj, 1 mg.

Initial Therapy Criteria

inv-21: Initial Therapy

Initial approval criteria for Elfabrio use in Fabry disease

Initial Therapy Nodes: Diagnosis of Fabry disease confirmed by enzyme assay demonstrating deficiency of alpha-galactosidase activity or by DNA testing; prescribed by or in consultation with a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist; age ≥ 18 years; not prescribed concurrently with Fabrazyme® or Galafold®; documentation of member's current weight (kg); dose does not exceed 1 mg/kg every 2 weeks.

Prior authorization requires submission of documentation supporting these criteria.

inv-22: Initial Therapy

Initial approval duration and documentation requirements updated.

Initial Therapy Requirements: Initial approval duration: 12 months for Medicaid/HIM (updated from 6 months); Commercial uses standard authorization duration language. Documentation must include member weight for dose calculation purposes.

See initial therapy criteria for full medical necessity requirements and dosing limits (maximum 1 mg/kg every 2 weeks).

Continuation / Continued Therapy Criteria

inv-23: Continued Therapy

Criteria for continued therapy

Continued Therapy Nodes: Member continuing therapy must meet continuity criteria (currently receiving via Centene benefit or previously met initial criteria, or covered under continuity of care rules); demonstrate positive clinical response in individual Fabry disease manifestations (examples listed in Appendix D); not be prescribed Elfabrio concurrently with Fabrazyme® or Galafold®; documentation of current weight (kg); and any dose increase must not exceed 1 mg/kg every 2 weeks.

Provide clinical documentation (office notes, labs) showing treatment response as noted in Appendix D.

inv-24: Continued Therapy

Continued therapy criteria were updated to include examples of positive treatment response and a concomitant-use exclusion.

Continued Therapy Requirements: Demonstrate continued clinical benefit with examples of improvement outlined in Appendix D (e.g., reduction in extremity pain; improvement in gastrointestinal symptoms; improved renal function; reduction in neuropathic symptoms; decreased fatigue; resolution or improvement of cornea verticillata). Must not be receiving excluded concomitant medications (Fabrazyme® or Galafold®). Documentation must include member weight for dose calculation.

Step Therapy & Restrictions

Step	Requirement
1	Concomitant-use exclusion: Elfabrio (pegunigalsidase alfa-iwxj) must not be prescribed concurrently with Fabrazyme® or Galafold® (concomitant-use exclusion added to Continued Therapy to mirror Initial Approval Criteria).

Background

Fabry disease is an X‑linked lysosomal storage disorder caused by deficiency of the enzyme alpha‑galactosidase A, resulting in accumulation of glycosphingolipids and multi‑system manifestations. Pegunigalsidase alfa‑iwxj (Elfabrio) is a PEGylated recombinant enzyme replacement therapy indicated for adults with confirmed Fabry disease; treatment is intended to reduce disease manifestations and requires specialist involvement, documentation of diagnosis, and dosing based on current weight.