CurrentmhnPolicy CP.PHAR.394

Migalastat (Galafold) - Coverage Criteria

Defines medical necessity, authorization, and continuation criteria for migalastat (Galafold) for adults with Fabry disease and an amenable GLA variant for members under Centene-affiliated health plans.

Policy Summary

Payermhn

PolicyMigalastat (Galafold) - Coverage Criteria

Policy CodePolicy CP.PHAR.394

Change TypeNo material changes

Effective Date09.11.18

Next Review DateN/A

Key ActionSubmit prior authorization with documentation confirming Fabry disease, age ≥18, specialist involvement, and an amenable GLA variant.

SourceLink

POLICY UPDATE CHANGES

No material clinical or coverage changes in this revision.

123 mgdose every other day (maximum/standard)

≥18minimum age for coverage

12 moapproval duration for continued therapy

Capsule 123 mgproduct availability

Medicaid notedMedicaid precedence

Coverage Criteria for Migalastat (Galafold)

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inv-01: Initial Therapy

Covered when ALL of the following are met

Initial Fabry Disease Criteria: 1) Diagnosis of Fabry disease confirmed by either: (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) Presence of at least one amenable GLA variant confirmed by one of: (a) Galafold Prescribing Information (Section 12, Table 2); (b) Amicus Fabry GLA Gene Variant Search Tool (https://www.galafoldamenabilitytable.com); or (c) Amicus Medical Information (1-877-4AMICUS or medinfousa@amicusrx.com); 5) Galafold is not prescribed concurrently with Fabrazyme or Elfabrio; 6) Dose does not exceed: (a) 123 mg every other day, and (b) 1 capsule every other day.

inv-02: Continuation Therapy

Continued therapy is authorized when ALL of the following are met

Continued Therapy Fabry Disease: 1) Member meets one of the following: (a) currently receiving medication via Centene benefit or previously met initial approval criteria; or (b) currently receiving medication and 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 (see Appendix F examples); 3) Galafold is not prescribed concurrently with Fabrazyme or Elfabrio; 4) If request is for a dose increase, the new dose does not exceed: (a) 123 mg every other day, and (b) 1 capsule every other day.

Approval duration: 12 months

This policy covers migalastat (Galafold) only for adults with a confirmed diagnosis of Fabry disease who have an amenable GLA variant as defined by the proprietary Amicus in vitro HEK-293 assay or listed in the Galafold Prescribing Information. The Amicus HEK-293 assay classifies a variant as amenable when there is at least a 20% relative increase in mutant alpha‑Gal A activity and an ≥3% absolute increase of wild‑type activity; absence from Table 2 of the Galafold PI indicates the variant is non‑amenable or untested and should be evaluated via Amicus resources.

Certain GLA variants associated with benign phenotypes are explicitly excluded from consideration as disease‑causing amenable variants (for example, c.937G>T, p.(D313Y)), and clinicians are advised to consult with clinical genetics as needed to determine whether an identified amenable variant is truly disease‑causing before initiating therapy.

Coverage determinations for migalastat are made in the context of the member's evidence of coverage and benefit documents. This clinical policy provides guidance on medical necessity to inform coverage decisions and benefit administration but does not guarantee payment. In cases of discrepancy, the terms, conditions, exclusions, and limitations of the member's coverage documents, as well as applicable state and federal requirements and Health Plan procedures, take precedence.

Use of migalastat for non‑FDA approved indications that are not addressed in this policy is not authorized unless there is sufficient documentation of efficacy and safety consistent with the Health Plan off‑label use policies (CP.CPA.09 for commercial, HIM.PA.154 for marketplace, CP.PMN.53 for Medicaid) or the applicable evidence of coverage documents. Requests involving variants or indications outside this policy should be evaluated against those off‑label policies and supporting clinical evidence.

Provider Actions, Documentation & Authorization

Prior Authorization

Prior Authorization Required

Prior Authorization Required: Initial requests must include documentation confirming the diagnosis of Fabry disease and that the member meets all initial approval criteria.

Diagnosis confirmed by enzyme assay demonstrating deficiency of alpha‑galactosidase activity or by DNA testing
Prescribed by or in consultation with an appropriate specialist (clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist)
Age ≥ 18 years
Documentation of at least one amenable GLA variant (see Galafold Prescribing Information, Amicus Fabry GLA Gene Variant Search Tool, or Amicus Medical Information)
Statement that Galafold will not be used concurrently with Fabrazyme® or Elfabrio®
Dose does not exceed 123 mg every other day (1 capsule every other day)

Documentation Required

Required Documentation

Provider must submit supporting clinical documentation (e.g., office notes, lab results, genetic testing reports) that demonstrates the member meets all required criteria for approval. Lack of adequate documentation will delay or preclude approval.

Include enzyme assay or DNA testing results that confirm Fabry disease
Include specialist consultation or treating specialist’s documentation
Include evidence of amenable GLA variant (printout or report from listed resources)

Denial Risk

Triggers for Denial

Denial Triggers: Requests missing required diagnostic confirmation, evidence of an amenable GLA variant, documentation of specialist involvement, or age < 18 will be subject to denial.

No enzyme assay or DNA test confirming Fabry disease
No documentation of an amenable GLA variant or documentation of a benign GLA variant (e.g., c.937G>T (p.(D313Y)))
Prescriber not a listed specialist and no documented specialist consultation
Member age under 18

Note

Professional Judgment Disclaimer

Professional Judgment Required: This policy is a guide to medical necessity and coverage determinations; it does not replace clinical judgment. Providers are expected to exercise professional medical judgment in the care and management of members.

Policy assists coverage decisions but does not guarantee payment
Providers retain responsibility for diagnosis and treatment decisions

Note

Policy Referral for Other Indications

Policy Referral for Other Indications: For uses not covered by these criteria (including recent label changes or off‑label uses), refer to the applicable no‑coverage, non‑formulary, or off‑label policies for the member’s line of business.

Formulary / PDL issues and no coverage policies: CP.CPA.190 (commercial), HIM.PA.33 (marketplace), CP.PMN.255 (Medicaid)
Non‑formulary policies: CP.CPA.190 (commercial), HIM.PA.103 (marketplace), CP.PMN.16 (Medicaid)
Off‑label use policies: CP.CPA.09 (commercial), HIM.PA.154 (marketplace), CP.PMN.53 (Medicaid)

Key Definitions

Amenable GLA variant — Amicus HEK-293 assay criteria

DefinitionA GLA gene variant classified as 'amenable' by the proprietary Amicus in vitro HEK-293 assay that shows both: (1) a relative increase ≥20% compared to pre-treatment alpha-Gal A activity and (2) an absolute increase ≥3% of wild-type alpha-Gal A activity.

Source guidanceIf a variant is not listed in Table 2 of the Galafold Prescribing Information it is either non-amenable or not tested in vitro; contact Amicus Medical Information for status questions.

Clinical caveatThe in vitro assay does not determine whether a GLA variant causes Fabry disease; pathogenicity should be assessed by the prescribing physician, with clinical genetics consultation as needed.

Known benign variantGLA variant c.937G>T (p.(D313Y)) is considered benign based on published data; consult a clinical genetics professional for additional evaluation when present.

Background

Migalastat is an oral pharmacological chaperone indicated for the treatment of adults with Fabry disease who have an amenable GLA variant. Approval decisions rely on in vitro amenability testing using the proprietary Amicus HEK‑293 assay, which defines amenability by a ≥20% relative increase and a ≥3% absolute increase in alpha‑Gal A activity. The assay identifies variants listed in Table 2 of the Galafold Prescribing Information; variants not listed are considered non‑amenable or not tested and should be clarified with Amicus Medical Information as needed.

The in vitro assay does not determine whether a variant causes Fabry disease; therefore, the prescribing clinician must confirm that an identified amenable variant is disease‑causing (with consultation from a genetics professional if indicated). Notably, some variants (for example, c.937G>T, p.(D313Y)) are associated with benign phenotypes and are not considered disease‑causing for treatment decisions.

This clinical policy is intended as a guide to medical necessity and is based on current standards and available evidence. Coverage and administration of benefits remain subject to the member's coverage documents and applicable law and regulation.

OpenPayer

Migalastat (Galafold) - Coverage Criteria

Coverage Criteria for Migalastat (Galafold)

Initial Therapy Requirements

Continuation / Reauthorization Requirements

Provider Actions, Documentation & Authorization

Key Definitions

Background

Quantity Limits and Product Details

Revision History