CurrentCentenePolicy CP.PHAR.394

Migalastat (Galafold) clinical coverage

Defines medical necessity criteria, prior authorization expectations, dosing limits, and coverage exclusions for migalastat (Galafold) for adults with Fabry disease across Centene lines of business (Commercial, HIM/ICHRA, Medicaid).

Policy Summary

PayerCentene

PolicyMigalastat (Galafold) clinical coverage

Policy CodePolicy CP.PHAR.394

Change TypeClarified / Added

Effective DateSep 11, 2018

Next Review Date

Key ActionProvider must submit documentation demonstrating diagnostic confirmation and amenable GLA variant confirmation and ensure prescriptions are by or in consultation with a qualified specialist.

SourceLink

POLICY UPDATE CHANGES

Initial approval duration updated from 6 months to 12 months.

Exclusion for concomitant use with Elfabrio added (previously excluded only with Fabrazyme).

18Minimum age for coverage

123 mg eodMaximum dose/frequency covered

12Approval duration (initial/continued)

Commercial/HIM/MedicaidLines of business

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Medical Necessity Criteria for Migalastat (Galafold)

Initial Approval — Fabry Disease

Galafold is medically necessary when ALL of the following are met:

ALL of the following

Diagnosis of Fabry disease confirmed by either enzyme assay demonstrating a deficiency of alpha-galactosidase activity OR DNA testing
Either (a) or (b) must be present
Prescribed by or in consultation with a clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist
Age is greater than or equal to 18 years>= 18 years
Amenable GLA variant confirmation (one required)
- Amenable GLA variant confirmed in Galafold Prescribing Information (package insert; Section 12, Table 2)
- Amenable GLA variant confirmed using the Amicus Fabry GLA Gene Variant Search Tool (galafoldamenabilitytable.com)
- Confirmation via Amicus Medical Information (1-877-4AMICUS or medinfousa@amicusrx.com)
Galafold is not prescribed concurrently with Fabrazyme or Elfabrio
Concomitant use excluded
Dose limits
- Dose does not exceed 123 mg every other day<= 123 mg every other day
- Dose does not exceed 1 capsule every other day<= 1 capsule every other day

Initial Approval — Other Diagnoses/Indications

If medication use falls outside the above and does not reflect a recent label change, follow one of the following:

ANY of the following

If recent label change (within 6 months) not reflected in this policy, refer to no coverage or non-formulary policy for relevant line of business (see related policies)
If requested use not listed under section III and no recent label change applies, refer to the off-label use policy for the relevant line of business (see related policies)

Continued Therapy — Fabry Disease

Continued therapy is medically necessary when ALL of the following are met:

ALL of the following

Member current status
- Currently receiving medication via Centene benefit or previously met initial approval criteria
- Member currently receiving medication and enrolled in a state/product with continuity of care regulations (refer to state addendums CC.PHARM.03A and CC.PHARM.03B)
Member is responding positively to therapy as evidenced by improvement in the individual's Fabry disease manifestation profile
Examples include improvement in pain in extremities, hypohidrosis/anhidrosis, angiokeratomas, GI symptoms, renal function, neuropathic symptoms, fatigue, or cornea verticillata
Galafold is not prescribed concurrently with Fabrazyme or Elfabrio

Continued Therapy — Other Diagnoses/Indications

If continued use falls outside the Fabry Disease criteria:

ANY of the following

If recent label change (within 6 months) not reflected, refer to no coverage/non-formulary policy for relevant line of business
If requested use not listed under section III and no recent label change applies, refer to the off-label use policy for the relevant line of business

Diagnoses/Indications Not Authorized

Coverage is not authorized for the following:

ALL of the following

Non-FDA approved indications not addressed in this policy, unless sufficient documentation per off-label use policies or evidence of coverage documents is provided
See related off-label policies
Users with amenable GLA variants associated with benign phenotypes (i.e., not causing Fabry disease), specifically the GLA mutation c.937G>T (p.(D313Y))
c.937G>T (p.(D313Y)) considered benign and excluded

Product / NDC Information

NDC / ProductNDC|product

Galafold 123 mg capsule

Product availability: Capsule 123 mg

Prior Authorization and Documentation Requirements

Prior Authorization

Specialist prescribing or consultation requirement

Prescriptions must be written by—or in consultation with—an appropriate specialist (clinical geneticist, cardiologist, nephrologist, neurologist, lysosomal disease specialist, or Fabry disease specialist). Prior authorization will require evidence the prescriber meets this requirement.

Documentation Required

Support initial approval with documentation

Submit supporting clinical documentation with initial requests, such as office/chart notes, laboratory results, or other clinical information, to demonstrate diagnostic confirmation and fulfillment of all initial approval criteria.

Prior Authorization

Amenable variant confirmation

Age and Dosing Limits

Coverage thresholds

Age>= 18 years

Maximum dose123 mg every other day (1 capsule every other day)

Approval duration (initial/continued)12 months

Clinical Background

Migalastat (Galafold) is an oral pharmacological chaperone indicated for the treatment of adults with a confirmed diagnosis of Fabry disease who have an amenable GLA variant based on in vitro assay data. The FDA approval for this indication was granted under accelerated approval on the basis of reduction in KIC GL-3 substrate, and continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials (see prescribing information).

Approval of Galafold through the health plan requires documentation confirming the diagnosis and amenable GLA variant (approval is contingent on demonstrated amenability in vitro per the referenced resources) and evidence of clinical benefit during continued therapy. Amenability may be verified using the Galafold Prescribing Information (Table 2), the Amicus Fabry GLA Gene Variant Search Tool (galafoldamenabilitytable.com), or Amicus Medical Information (1-877-4AMICUS or medinfousa@amicusrx.com). The proprietary Amicus HEK-293 in vitro assay defines an amenable variant as one with a relative increase of ≥20% from baseline and an absolute increase of ≥3% of wild-type activity.

Coverage is limited to adults aged ≥ 18 years, and dosing is limited to a maximum of 123 mg every other day (1 capsule every other day). Galafold is not authorized for concurrent use with enzyme replacement therapy or other pharmacologic therapies for Fabry disease; specifically, Galafold must not be prescribed concurrently with Fabrazyme or Elfabrio.

For continued therapy, the member must demonstrate a positive clinical response to treatment (examples include improvement in pain, hypohidrosis/anhidrosis, angiokeratomas, gastrointestinal symptoms, renal function, neuropathic symptoms, fatigue, or cornea verticillata). Initial and continued approvals follow the plan’s prior authorization and documentation requirements and reflect the policy updates including an initial approval duration of 12 months (policy administrative updates effective 2Q 2026).

Defined Terms

Definitions

Amenable variant (Amicus HEK-293 assay)A GLA variant is considered amenable if in vitro mutant alpha-Gal A activity shows >=20% relative increase compared to pre-treatment and an absolute increase of >=3% of wild-type activity.

alpha-Gal AAlpha-galactosidase A enzyme (deficient in Fabry disease).

Enzyme replacement therapyERT (enzyme replacement therapy).

Policy Changes

2026-05-26clarifiedLatest

Initial approval duration updated from 6 months to 12 months; Continued Therapy examples of positive treatment response added and ICHRA line of business added.

4Q 2023added

Exclusion for concomitant use with Elfabrio added (previously only excluded concomitant use with Fabrazyme); exclusion appears in Initial and Continued Therapy sections.

Migalastat (Galafold) clinical coverage

Medical Necessity Criteria for Migalastat (Galafold)

Product / NDC Information

Prior Authorization and Documentation Requirements

Age and Dosing Limits

Clinical Background

Defined Terms

Policies to Read With This Document

Policy Changes