Galafold - NC Standard

Restricted Product(s)

• Galafold® (migalastat) 123mg capsules

FDA Approved Use

• For the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data

Criteria for Approval of Restricted Product(s)

Initial Coverage for Approval

1. The patient is 16 years of age or older; AND 
2. The patient has been diagnosed with Fabry disease; AND 
3. The patient has an amenable galactosidase alpha gene (GLA) variant that has been FDA approved for Galafold (medical documentation required); AND
   1. The GLA mutation has NOT been determined to be benign, of uncertain clinical significance, or variant of uncertain significance (VUS) (medical documentation required); AND 
   2. The amenable GLA variant has been determined to be pathogenic and clinically significant for the patient by a specialist in genetics (medical documentation required); AND 
4. The prescriber has assessed current levels of ALL the following (medical documentation required):
   1. Kidney function (e.g., proteinuria, GFR) 
   2. Cardiac function (e.g., left ventricular hypertrophy, conduction, or rhythm, mitral or aortic insufficiency) 
   3. Optic neuropathy 
   4. Neuropathic pain 
   5. Gastrointestinal symptoms; AND 
5. The patient will NOT be using the requested agent in combination with any enzyme replacement therapy (e.g., Fabrazyme, Elfabrio) for the requested indication; AND 
6. The provider is aware that if approved, any previous authorizations through Blue Cross NC for enzyme replacement therapy agents used for the same indication as the requested agent will be terminated; AND 
7. The patient is being managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., geneticist); AND
8. Indications outside of FDA labeling will be subject to medical necessity review in accordance with specific strong endorsement or support by nationally recognized compendia, when such recommendation is based on strong/high levels of evidence, and/or uniform consensus of clinical appropriateness has been reached. Medical records and references / evidence must be provided; AND 
9. For formularies that exclude (non-formulary) the requested medication, Non-formulary Exception Criteria applies.
   
   Duration of Approval: 180 days

Continuation Criteria for Approval

1. The patient has been previously approved with Blue Cross and Blue Shield of North Carolina (Blue Cross NC) or would have met initial criteria for approval upon the start of therapy; AND 
2. The patient has experienced clinical benefit since initiation of therapy as indicated by improvement in at least ONE of the following (medical record documentation required):
   1. Kidney function (e.g., proteinuria, GFR) 
   2. Cardiac function (e.g., left ventricular hypertrophy, conduction, or rhythm, mitral or aortic insufficiency), 
   3. Optic neuropathy 
   4. Neuropathic pain 
   5. Gastrointestinal symptoms; AND 
3. The patient continues to NOT use the requested agent in combination with any enzyme replacement therapy (e.g., Fabrazyme, Elfabrio) for the requested indication; AND 
4. The provider is aware that if approved, any previous authorizations through Blue Cross NC for enzyme replacement therapy agents used for the same indication as the requested agent will be terminated; AND 
5. The patient continues to be managed by or in consultation with a specialist in the area of the patient’s diagnosis (e.g., geneticist); AND 
6. The prescribed dose and quantity are appropriate based on intended use and FDA labeling.
   
   Duration of Approval: 365 days (1 year)

Quantity Limit Exception Criteria

1. The quantity (dose) requested is for documented titration purposes at the initiation of therapy (authorization for a 90-day titration period); AND 
2. The prescribed dose cannot be achieved using a lesser quantity of a higher strength; AND 
3. The quantity (dose) requested does not exceed the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert; OR
4. If the quantity (dose) requested exceeds the maximum FDA labeled dose, when specified, or to the safest studied dose per the manufacturer’s product insert, then the prescriber must submit documentation in support of therapy with a higher dose for the intended diagnosis (submitted documentation may include medical records OR fax form which reflects medical record documentation that shows the length of time the requested dose has been used, and what other medications and doses have been tried and failed).
   
   Duration of Approval: 365 days (1 year)

References

All information referenced is from FDA package insert unless otherwise noted below.

Policy Implementation/Update Information

Criteria and treatment protocols are reviewed annually by the Blue Cross NC P&T Committee, regardless of change. This policy is reviewed in Q1 annually.

July 2023: Criteria update: Added Elfabrio to list of medications patient will NOT be using in combination with the requested agent.
April 2023: Criteria change: Formatting changes. Updated age indication to include 16 years of age or older. Lengthened duration of approval for continuation criteria and quantity limit exception criteria.
October 2021: Criteria change: Addition of medical records requirement for specialist and assessment of kidney function criteria points.
February 2020: Criteria change: Removed requirement for average number of GL-3 inclusions per KIC. Added assessment of clinical symptoms to initial and continuation criteria. Removed requirement that no ERT has been used within the last 6 months. Removed requirement of a trial and failure of Fabrazyme.
July 2018: Policy origination.