mhn migalastat (Galafold) Coverage Update

Coverage Criteria for Migalastat (Galafold)

inv-01: Initial Therapy

Covered when ALL of the following are met for Initial Approval:

Initial Approval - Fabry Disease: 1. Diagnosis of Fabry disease confirmed by one of the following: (a) enzyme assay demonstrating a deficiency of alpha-galactosidase A 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 the following resources: (a) Galafold Prescribing Information (Section 12, Table 2) OR (b) Amicus Fabry GLA Gene Variant Search Tool OR (c) Amicus Medical Information; 5. Galafold is not prescribed concurrently with Fabrazyme or Elfabrio; 6. Dose does not exceed 123 mg every other day (1 capsule every other day).

Initial approval requires all listed conditions.

inv-02: Continuation Therapy

Covered when ALL of the following are met for Continued Therapy:

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) 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, or cornea verticillata); 3. Galafold is not prescribed concurrently with Fabrazyme or Elfabrio; 4. If request is for a dose increase, the new dose does not exceed 123 mg every other day (1 capsule every other day).

Approval duration: 12 months.

Non‑FDA approved indications not specifically addressed in this policy are generally not covered unless the request is supported by adequate documentation of safety and efficacy per the applicable off‑label use policies: CP.CPA.09 (commercial), HIM.PA.154 (Health Insurance Marketplace/ICHRA), or CP.PMN.53 (Medicaid). Additionally, certain GLA variants that are associated with benign phenotypes (for example, c.937G>T (p.(D313Y))) are listed as exclusions and should not be treated as disease‑causing without further clinical genetics evaluation.

When assessing amenability, use the Galafold prescribing information (Table 2) or Amicus resources; note that the in‑vitro assay determining amenability does not itself establish whether a variant causes Fabry disease and clinicians should confirm pathogenicity prior to treatment initiation.

For Medicaid members, state Medicaid coverage provisions take precedence if they conflict with the coverage provisions in this clinical policy. Providers and reviewers should follow the applicable state Medicaid manual for any differing coverage requirements.

Use of migalastat for non‑FDA approved indications that are not specifically addressed in this policy is not authorized unless there is sufficient supporting documentation of efficacy and safety per the referenced off‑label use policies (CP.CPA.09, HIM.PA.154, CP.PMN.53) or other evidence of coverage documents.

Coding and Amenability Criteria

No explicit CPT/HCPCS/NDC codes listed in this part of the documentmixed

No codes listed

inv-07: Amenability assay criteria

Amenability assay thresholdsRelative increase ≥20% compared to pre-treatment alpha‑Gal A activity AND absolute increase ≥3% of wild‑type (normal) alpha‑Gal A activity in the proprietary Amicus HEK‑293 in vitro assay.

Source for amenability statusIf a GLA variant is not listed in Table 2 of the Galafold Prescribing Information it is either non‑amenable or has not been tested; contact Amicus Medical Information for variant status.

Assay limitationThe in vitro assay classifies amenability but does NOT determine whether a GLA variant is disease‑causing; clinical interpretation is required.

Provider Actions, Prior Authorization and Documentation

Prior Authorization

Prior Authorization Required

Prior authorization is required. Provider must submit documentation (e.g., office chart notes, laboratory results, genetic testing reports) supporting that the member meets all approval criteria for Galafold prior to coverage. Requests should document confirmed diagnosis of Fabry disease and presence of an amenable GLA variant when applicable.

Submit enzyme assay or DNA testing results to confirm Fabry disease
If applicable, provide evidence of an amenable GLA variant (Galafold prescribing information, Amicus amenability tool, or Amicus Medical Information)
Prescriber must be a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist — or include documented consultation with one

Denial Risk

Triggers for Denial

Coverage will be denied if required documentation or criteria are missing. Common triggers for denial include absence of documented diagnostic confirmation, patient age < 18 years, lack of confirmation of an amenable GLA variant when indicated, missing specialist prescriber or documented consultation, concurrent prescription with excluded therapies (Fabrazyme or Elfabrio), or dosing in excess of policy limits.

Initial Therapy Criteria

inv-20: Initial Therapy

Initial approval requires diagnostic confirmation, specialist involvement, adult age, amenable variant confirmation, no concurrent ERT, and dose limits.

Initial Therapy Criteria: 1. Diagnosis confirmed by enzyme assay demonstrating alpha-galactosidase A deficiency OR 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 via Galafold Prescribing Information Table 2, the Amicus Fabry GLA Gene Variant Search Tool, or Amicus Medical Information; 5. Not prescribed concurrently with Fabrazyme or Elfabrio; 6. Dose ≤ 123 mg every other day (1 capsule every other day).

inv-21: INITIAL THERAPY CRITERIA

Initial authorizations are now expected to be issued for 12 months (updated from 6 months).

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Continuation / Continued Therapy Criteria

inv-22: Continued Therapy

Continuation criteria require documented positive clinical response and no concurrent ERT.

Continued Therapy Criteria: 1. Member currently receiving medication via Centene benefit or previously met initial approval criteria OR continuity of care applies; 2. Documented positive clinical response demonstrated by improvement in the member's Fabry disease manifestation profile; 3. Galafold is not prescribed concurrently with Fabrazyme or Elfabrio; 4. Dose must not exceed 123 mg every other day (1 capsule every other day).

Approval duration 12 months.

inv-23: CONTINUATION CRITERIA

Continued therapy criteria were expanded with examples of positive treatment response referenced from Appendix F.

Examples of Positive Treatment Response: Examples of clinical improvement that may support continuation include, but are not limited to: reduction in pain in the extremities; improvement in hypohidrosis or anhidrosis; reduction in angiokeratomas; improvement in gastrointestinal symptoms (diarrhea, abdominal pain, nausea, vomiting, flank pain); improved or stabilized renal function; reduction in neuropathic pain, improved heat and cold tolerance, reduction in vertigo or diplopia; decreased fatigue; resolution or improvement in cornea verticillata.

Quantity Limits and Dosage

inv-24: Migalastat (Galafold) capsule quantity limit / dosing note

Quantity limit (capsule)1 capsule per dosing day (policy expresses limit as 1 capsule; dosing is every other day).

Capsule strength123 mg per capsule.

Dosing regimen noteRecommended dose: 123 mg orally every other day (1 capsule every other day).

Site of Care

Note

No site‑of‑care restrictions specified

No site‑of‑care restrictions are specified in this portion of the policy.

Setting designation in the policy: home; no restrictions noted.

Definitions and Regulatory Context

inv-17: Amenable GLA variant definition

Definition of amenable GLA variantA GLA variant is classified as 'amenable' when the proprietary Amicus HEK‑293 in vitro assay shows both a ≥20% relative increase in mutant alpha‑Gal A activity and an absolute increase of ≥3% of wild‑type alpha‑Gal A activity.

Where to confirm amenabilityAmenability lists are in the Galafold Prescribing Information (Table 2) or via the Amicus Fabry GLA Gene Variant Search Tool / Amicus Medical Information.

Clinical considerationPresence on the amenability list does not determine pathogenicity; clinicians should determine whether the variant causes Fabry disease prior to treatment initiation.

inv-18: Accelerated approval basis

Basis of accelerated approvalGalafold was granted accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL‑3) substrate.

Background

Fabry disease is caused by deficient alpha‑galactosidase A activity due to pathogenic GLA gene variants. Migalastat (Galafold) is an oral pharmacological chaperone that binds certain mutant forms of alpha‑galactosidase A to stabilize the enzyme and increase its activity in patients with amenable GLA variants. Amenability is determined using the proprietary Amicus HEK‑293 in‑vitro assay, which defines an "amenable" variant as one producing a relative increase ≥20% and an absolute increase ≥3% of wild‑type alpha‑Gal A activity. The assay result informs whether a variant is likely to respond to migalastat, but the assay does not determine whether a variant is disease‑causing; clinicians should confirm that the variant is pathogenic for Fabry disease before initiating therapy.

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Migalastat (Galafold) coverage