Pegunigalsidase Alfa (Elfabrio) — Coverage Criteria
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Defines medical necessity and prior authorization criteria for Elfabrio (pegunigalsidase alfa-iwxj) for treatment of adults with confirmed Fabry disease for Health Net lines of business including Commercial, HIM, and Medicaid.
Added HCPCS code J2508 to coding.
Added concomitant use exclusion to the Continued Therapy section to mirror the Initial Approval Criteria and added requirement for documentation of member's weight for dose calculation.
References reviewed and updated during annual reviews (3Q 2024 and 2Q 2025) with no significant clinical changes otherwise.
Coverage Criteria
inv-01: Initial Therapy — Covered when ALL of the following are met
Covered when ALL of the following are met:
Approval duration: 6 months
inv-02: Continuation Therapy — Covered for continuation when ALL of the following are met
Covered for continuation when ALL of the following are met:
Approval duration: 12 months
inv-03: Continued Therapy (updated) — Continued Therapy criteria including updates
Continued Therapy (updated)
Concomitant use exclusion was added to mirror the Initial Approval Criteria.
Coverage does not authorize use of Elfabrio (pegunigalsidase alfa) for non‑FDA approved indications that are not specifically addressed in this policy. Such off‑label requests must be supported by sufficient documentation of efficacy and safety in accordance with the referenced off‑label use policies (CP.CPA.09 for Commercial, HIM.PA.154 for Health Insurance Marketplace, and CP.PMN.53 for Medicaid) or other evidence of coverage documents.
For Continued Therapy, coverage excludes concomitant use of therapies that are specified in the Initial Approval Criteria. Continued approval requires documentation that the member is not receiving any excluded concomitant therapies; this exclusion was added to the Continued Therapy section to mirror the Initial Approval Criteria and was recorded during the 2Q 2025 update.
Concurrent prescription of Elfabrio with either Fabrazyme or Galafold is explicitly disallowed. Requests where Elfabrio is prescribed simultaneously with Fabrazyme or Galafold are considered not medically necessary and are excluded from coverage.
Coding
Provider Actions and Requirements
Prior Authorization Required
Prior authorization is required for initial therapy. Requests must include documentation confirming the diagnosis of Fabry disease and that the prescriber is a specialist (clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist). Requests that lack required documentation (diagnosis confirmation, specialist prescriber, or member weight in kg) may be denied.
- Prior authorization required for initial therapy with documentation of confirmed Fabry disease
- Prescriber must be a specialist or consult with a listed specialist
- Incomplete requests (missing diagnosis confirmation, specialist prescriber, or weight) may be denied
Non‑FDA Indications
Off‑label (non‑FDA) indications that are not addressed in this policy may be denied unless there is sufficient documentation of efficacy and safety per the applicable off‑label use policy for the line of business. Refer to the commercial, health insurance marketplace, or Medicaid off‑label policies as applicable.
- Non‑FDA indication requests require evidence per off‑label use policies: CP.CPA.09 (commercial), HIM.PA.154 (marketplace), CP.PMN.53 (Medicaid)
- If the use is a recent label change not yet reflected here, follow the relevant line‑of‑business formulary/coverage guidance
Documentation and Concomitant Use
Providers must document absence of excluded concomitant therapies and provide member weight for dose calculation. Failure to document weight or the presence/absence of excluded concomitant use (for example, concurrent Fabrazyme or Galafold) may result in denial.
- Elfabrio (pegunigalsidase alfa) is not to be prescribed concurrently with Fabrazyme or Galafold; providers must document that these therapies are not being used concomitantly
- Documentation of the member's current weight (in kg) is required for dose calculation and must be submitted
Required Clinical Documentation
Submit supporting clinical documentation with the prior authorization request. Acceptable documentation includes office chart notes, laboratory results (e.g., enzyme assay or DNA testing confirming Fabry disease), and any other clinical information that demonstrates the member meets approval criteria.
- Office notes, lab results (enzyme assay demonstrating alpha‑galactosidase deficiency or DNA testing), and specialist consultation notes as applicable
- Documentation must support age, diagnosis confirmation, current weight (kg), dosing, and absence of excluded concomitant therapies
Formulary / Off‑Label Referral
Refer to formulary, non‑formulary, and off‑label policies when the requested use is not covered under this policy. For formulary and PDL items use the appropriate no coverage or non‑formulary policies for the line of business.
- Formulary/no coverage policies: CP.CPA.190 (commercial), HIM.PA.103 or HIM.PA.33 (marketplace), CP.PMN.16 or CP.PMN.255 (Medicaid) as applicable
- Non‑formulary or off‑label escalations should follow the referenced line‑of‑business policies
Background
Fabry disease is an X‑linked lysosomal storage disorder caused by deficiency of the enzyme alpha‑galactosidase A, leading to accumulation of glycosphingolipids in multiple organ systems. Clinical manifestations can include peripheral neuropathic pain, angiokeratomas, hypohidrosis or anhidrosis, gastrointestinal symptoms, renal impairment progressing to proteinuria and chronic kidney disease, and cardiac involvement such as left ventricular hypertrophy and arrhythmia. Diagnosis is established by decreased enzyme activity on assay or by DNA testing, and management may include enzyme replacement therapy such as pegunigalsidase alfa (Elfabrio) for adults with confirmed disease.
Definitions
Initial Therapy Criteria
inv-23: Initial Therapy — Initial coverage criteria (grouped block)
Covered when ALL of the following are met:
Approval duration: 6 months
inv-24: Initial Therapy — Initial Approval Criteria referenced; concomitant use exclusion exists in Initial Approval Criteria
Initial Approval Criteria referenced; concomitant use exclusion exists in Initial Approval Criteria.
Details of initial criteria are in the Initial Approval Criteria section.
Continuation / Continued Therapy Criteria
inv-25: Continuation — Criteria for continued coverage
Criteria for continued coverage
Approval duration: 12 months
inv-26: Continuation Therapy — Continuation therapy updated to include exclusions and weight documentation
Continuation therapy updated to include exclusions and weight documentation
Added during 2Q 2025 annual review.
Step Therapy
| Step | Requirement / Instruction | Reference |
|---|---|---|
| Refer to formulary/non-formulary or off-label policies | When the requested use is not listed in this policy or has recently changed (e.g., new indication, age expansion, or dosing not reflected here), refer to the applicable formulary, non‑formulary, or off‑label use policy for the member's line of business. | CP.CPA.190, HIM.PA.33 or CP.PMN.255 for formulary/no coverage; CP.CPA.09, HIM.PA.154 or CP.PMN.53 for off‑label use |
Quantity Limits
Revision History
3Q 2024 annual review — references reviewed and updated (no significant clinical changes).
HCPCS code J2508 (Injection, pegunigalsidase alfa-iwxj, 1 mg) was added to the policy coding.
2Q 2025 annual review — added concomitant use exclusion to the Continued Therapy section and added requirement for documentation of member weight for dose calculation.
P&T approval recorded for 2Q 2025 updates including the concomitant use exclusion and weight documentation requirement for Continued Therapy.
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