CurrentBlue Cross Blue Shield - IowaPolicy 05.02.53

Galafold (migalastat) — Coverage and Prior Authorization Criteria

Defines coverage and prior authorization requirements for Galafold (migalastat) for treatment of Fabry disease for Wellmark/Blue Cross Blue Shield members, including initial and continuation criteria, documentation, dosing limits, and billing guidance.

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyGalafold (migalastat) — Coverage and Prior Authorization Criteria

Policy CodePolicy 05.02.53

Change TypeRevised (Jan 2025)

Effective DateMar 1, 2025

Next Review Date

Key ActionObtain prior authorization with documentation of age ≥16, confirmed Fabry disease with an amenable GLA variant, prescriber specialty, and evidence of no concurrent enzyme replacement therapy.

SourceLink

POLICY UPDATE CHANGES

Policy reviewed and revised in January 2025 with current effective date March 1, 2025.

16+min. age

14 capsqty limit/28d

Amenable GLAgenetic req

Prohibitedconcurrent ERT

Coverage Criteria for Galafold (migalastat)

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Initial Therapy

Covered when ALL of the following are met for initial approval:

Initial Approval Criteria: Prescribed by or in consultation with a geneticist, nephrologist, or a physician who specializes in the management of Fabry disease.

From policy initial approval requirements.

Initial Approval Criteria: Patient is 16 years of age or older.

Minimum age requirement per policy.

Initial Approval Criteria: Diagnosis of Fabry disease confirmed by enzyme assay demonstrating deficiency of alpha-galactosidase A activity, or by genetic testing, or the member is a symptomatic obligate carrier.

Diagnostic confirmation required.

Initial Approval Criteria: Patient has an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

Genetic eligibility requirement.

Initial Approval Criteria: Patient will not receive Galafold in combination with enzyme replacement therapy (ERT) for Fabry disease (e.g., Fabrazyme [agalsidase beta], Elfabrio [pegunigalsidase alfa]).

Concurrent ERT is excluded.

Initial Approval Criteria: Documented renal disease requiring dialysis, when applicable, is noted in the policy (placement in list is per source).

Renal dialysis stated in source; include as criterion where clinically relevant.

Continuation Therapy

Covered when ALL of the following are met for continuation:

Continuation Criteria: Patient has a documented positive clinical response to therapy (e.g., lab results demonstrating a reduction in plasma GL-3/Gb3 or GL-3/Gb3 inclusions; progress notes documenting improvement or stabilization in clinical symptoms; improvement or stabilization in renal function; reduction in pain).

Objective clinical or laboratory evidence required for continuation; medical records must be provided.

Continuation Criteria: Patient is not receiving Galafold in combination with enzyme replacement therapy (e.g., Fabrazyme [agalsidase beta]).

Concurrent ERT is excluded for continuation as well.

Use of Galafold (migalastat) in combination with enzyme replacement therapy (ERT) is specifically excluded. The policy requires that the patient will not be receiving Galafold in combination with enzyme replacement therapy (ERT), for example Fabrazyme (agalsidase beta) or Elfabrio (pegunigalsidase alfa). Requests where Galafold is to be used concurrently with ERT are not consistent with the coverage criteria and may be denied.

Coverage is limited to patients with a confirmed diagnosis of Fabry disease and evidence that the patient’s GLA gene variant is amenable to migalastat. Initial approval requires confirmation of diagnosis by enzyme assay demonstrating alpha-galactosidase A deficiency or by genetic testing (or documentation that the member is a symptomatic obligate carrier), and that the patient has an amenable GLA variant based on in vitro assay data. Continuation of therapy likewise depends on documented clinical benefit and the absence of concurrent ERT.

Initial Therapy Requirements

Initial Therapy

Covered when ALL of the following are met for initial approval:

Initial Approval Criteria: Prescribed by or in consultation with a geneticist, nephrologist, or a physician who specializes in the management of Fabry disease.

From policy initial approval requirements.

Initial Approval Criteria: Patient is 16 years of age or older.

Minimum age requirement per policy.

Diagnostic confirmation required.

Initial Approval Criteria: Patient has an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.

Genetic eligibility requirement.

Concurrent ERT is excluded.

Initial Approval Criteria: Documented renal disease requiring dialysis, when applicable, is noted in the policy (placement in list is per source).

Renal dialysis stated in source; include as criterion where clinically relevant.

Continuation / Renewal Requirements

Continuation criteria

Continuation of Galafold for Fabry disease

Continuation: Documented positive clinical response to therapy (e.g., reduction in plasma GL-3/Gb3 or GL-3/Gb3 inclusions; improvement or stabilization in renal function; improvement or stabilization in clinical symptoms, including pain).

Medical records or laboratory data required to demonstrate benefit.

Continuation: Patient is not receiving Galafold in combination with enzyme replacement therapy (e.g., Fabrazyme [agalsidase beta]).

Concurrent ERT is excluded for ongoing therapy.

Prior Authorization, Documentation, and Denials

Prior Authorization

Prior Authorization Required

Prior authorization is required for Galafold (migalastat). Prescriptions must be initiated by or in consultation with an appropriate specialist and meet the age, diagnostic, and genetic criteria below for initial approval.

Prior authorization required before coverage
Prescriber: specialist (geneticist, nephrologist, or physician specializing in Fabry disease) or in consultation with one
Age: ≥16 years for initial approval
Diagnostic confirmation: enzyme assay demonstrating deficient alpha-galactosidase activity or genetic testing (or symptomatic obligate carrier)
Genetic requirement: GLA variant shown amenable by validated in vitro assay
Continuation requirement: documented positive clinical response (e.g., reduction in plasma GL-3/Gb3 or inclusions; improvement/stabilization of symptoms or renal function)
Therapy exclusion: not to be used in combination with enzyme replacement therapy (ERT) such as Fabrazyme or Elfabrio
Members receiving samples or patient assistance must meet the same prior authorization criteria

Step Therapy

No explicit step therapy sequence is described for Galafold; there is no requirement to trial or fail another specific medication prior to authorization.

Documentation Required

Required Documentation

Required documentation must be submitted with the prior authorization request to support medical necessity and to confirm diagnosis and response.

Medical records and chart notes documenting clinical history and specialist consultation
Laboratory results (e.g., enzyme assay demonstrating alpha-galactosidase deficiency; plasma GL-3/Gb3 levels) showing baseline and, for continuation, evidence of response
Genetic testing results confirming diagnosis and identifying the GLA variant
Documentation that the identified GLA variant is amenable based on validated in vitro assay data
Progress notes documenting improvement or stabilization in clinical signs/symptoms and renal function for continuation requests
For members previously receiving samples or patient assistance, documentation equivalent to initial approval criteria

Denial Risk

Denial Triggers

Requests will be denied when PRIOR AUTH criteria are not met or when contraindicated conditions are present. Common denial triggers are listed below.

Member is under 16 years of age for initial therapy requests
Lack of confirmatory diagnostic testing (no enzyme assay or genetic testing documenting Fabry disease or symptomatic obligate carrier status)
No documentation of an amenable GLA variant based on in vitro assay data
Absence of documentation demonstrating a positive clinical response for continuation (no reduction in plasma GL-3/Gb3 or clinical improvement/stabilization)
Concurrent use of Galafold with enzyme replacement therapy (ERT) such as Fabrazyme or Elfabrio
Incomplete medical records or missing required laboratory/genetic reports
Members who received samples or assistance but whose records do not meet initial approval criteria

Policy Summary

PayerBlue Cross Blue Shield - Iowa

PolicyGalafold (migalastat) — Coverage and Prior Authorization Criteria

Policy CodePolicy 05.02.53

Change TypeRevised (Jan 2025)

Effective DateMar 1, 2025

Next Review Date

SourceLink

On This Page

OpenPayer

Galafold (migalastat) — Coverage and Prior Authorization Criteria

Coverage Criteria for Galafold (migalastat)

Initial Therapy Requirements

Continuation / Renewal Requirements

Procedures, Billing Codes, and Quantity Limits

Prior Authorization, Documentation, and Denials

Key Definitions

Background and Clinical Context

Site of Care and Dosing Limits

Policy Revision History