CurrentMass General Brigham Health PlanPolicy N/A

Galafold (migalastat)

Pharmacy benefit prior authorization policy for Galafold (migalastat) for adults with confirmed Fabry disease and amenable GLA variants; includes specialty fill requirement, prescriber qualifications, diagnostic testing, duration limits, and combination therapy exclusion.

Policy Summary

PayerMass General Brigham Health Plan

PolicyGalafold (migalastat)

Policy CodePolicy N/A

Change TypeAdministrative update (no clinical changes)

Effective DateJan 1, 2025

Next Review Date

Key ActionObtain prior authorization under the pharmacy benefit and ensure specialty pharmacy fill; contact the pharmacy benefit phone/fax for authorization.

SourceLink

POLICY UPDATE CHANGES

Reviewed at December P&T. No changes. Effective 1/1/2025.

Effective date 01/01/2025 and designation of Commercial/Exchange plan applicability and pharmacy benefit prior authorization program.

12Approval Duration

>=18Age Requirement (years)

SpecialtyPharmacy Fill

ERTTherapy Exclusion

This is a pharmacy benefit prior authorization policy for Galafold (migalastat) for adults with a confirmed diagnosis of Fabry disease and GLA variants amenable to treatment. The medication is designated specialty and must be filled at a contracted specialty pharmacy. Prescriber requirements include a clinical genetics specialist or nephrologist, or documentation of a consult with one of these specialists. Diagnostic documentation must include enzyme assay results demonstrating reduced or absent α-galactosidase A (α-GAL) activity and evidence the member’s GLA mutation is amenable per in vitro assay data. Approvals are granted for 12 months. Galafold is not authorized in combination with enzyme replacement therapy (ERT).

Quick Facts

Age threshold>= 18 years

Approval duration12 months

Pharmacy fill requirementSpecialty (must be filled at contracted specialty pharmacy)

Therapy exclusionCombination with enzyme replacement therapy (ERT) excluded

Initial Authorization Criteria

Authorization Criteria (new to plan exceptions and standard criteria)

Authorization may be granted when ALL of the following are met:

Exception for members new to plan

Member new to the plan within the past 90 days AND currently receiving treatment with the requested medication (excluding when the product is obtained as samples or via manufacturer's patient assistance programs).
Allows continuation for existing therapy when member newly enrolled

ALL of the following

Age requirement: Member is 18 years of age or older>= 18 years
Diagnosis of Fabry disease
Prescriber or consult: Prescriber is a clinical genetics specialist or nephrologist OR a consult with either specialist is provided
Documentation of specialist prescriber or consult required
Enzyme assay: Submission of results from enzyme assay test showing reduced or absent α-galactosidase A (α-GAL) enzyme activity in plasma, leukocytes, tears, or biopsied tissue
Amenable GLA mutation: The member has GLA mutations which are amenable to treatment with Galafold
Amenability based on in vitro assay data per product labeling

Continuation and Limitations

Limitations / Duration

Limitations

Approval duration: Approvals will be granted for 12 months12 months
Combination therapy exclusion: Galafold will not be authorized in combination with enzyme replacement therapy (ERT)
Combination therapy with ERT is exclusionary

Denial Risk

Combination Therapy Exclusion

Claims will not be authorized if Galafold is prescribed in combination with enzyme replacement therapy (ERT).

Provider Actions & Requirements

Prior Authorization

Prior Authorization Required

Prior authorization required under the pharmacy benefit; specialty pharmacy fill mandated. Contact pharmacy benefit phone/fax for authorization.

Documentation Required

Diagnostic and Genetic Documentation

Submit enzyme assay results showing reduced/absent α-GAL activity and documentation that the member's GLA mutation is amenable to migalastat per in vitro assay data; include specialist consult or that prescriber is a clinical genetics specialist or nephrologist.

Billing Rule

Specialty Pharmacy Dispensing

Medication must be filled at a contracted specialty pharmacy as designated by the plan.

Prior Authorization

Continuation for New-to-Plan Members

Members new to the plan within 90 days who are currently receiving Galafold may be authorized upon evidence of current treatment (excluding samples or manufacturer assistance program supply).

Clinical Evidence & References

Key references include the product labeling (Galafold prescribing information, October 2024) and clinical studies such as Germain et al., N Engl J Med 2016 and Benjamin et al., Genet Med 2017. The product received accelerated approval based on reduction in kidney interstitial capillary cell GL-3, and continued approval may depend on verification of clinical benefit in confirmatory trials.

Background & Definitions

Galafold (migalastat) is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable GLA variant based on in vitro assay data. Approval was granted under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate; continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

Term	Definition
{"text":"α-GAL","status":""},{"text":"Alpha-galactosidase A enzyme (α-galactosidase A) whose reduced or absent activity supports diagnosis of Fabry disease.","status":""}
{"text":"Amenable GLA mutation","status":""},{"text":"GLA gene variants shown to be responsive to treatment with migalastat based on in vitro assay data per product labeling.","status":""}
{"text":"ERT","status":""},{"text":"Enzyme replacement therapy; concurrent use with Galafold is not authorized.","status":""}

Revision History

2024-12-11review