Srt Fabry Galafold denied as not FDA-approved for this use by Aetna?

Aetna's specific coverage criteria for srt fabry galafold are defined in its own published medical/coverage policy and the FDA-approved prescribing label. A successful appeal documents that your medical records satisfy each criterion those sources list — confirmed diagnosis, any required prior treatments (with dates and outcomes), and clinical severity. If the exact criteria weren't included with your denial, request them in writing; your appeal then maps each requirement to the matching fact in your chart.

## Why Aetna Issues a "Not FDA-Approved" Denial for Galafold — and Why It Is Likely Wrong

Galafold (migalastat) received FDA approval for a specific adult population with Fabry disease who carry an amenable GLA mutation. A "not FDA-approved" denial for this drug almost always reflects one of two documentation failures: the submission did not make clear that the patient carries an amenable mutation as defined in the FDA-approved prescribing label, or the reviewer conflated the drug's narrow approved indication (amenable-mutation Fabry disease) with an off-label use.

This type of denial is strongly appealable because the drug IS FDA-approved — the appeal simply needs to demonstrate that the patient falls within the approved indication.

## Your Federal Appeal Rights

ACA §2719 guarantees independent external review for non-grandfathered plans. An external review organization's clinical reviewers are required to evaluate whether the denial was consistent with the evidence and the terms of the plan. When the denial basis is factually incorrect (the drug is FDA-approved), external reviewers overturn these denials at high rates. You have approximately four months from the denial notice to file for external review, and expedited external review is available for urgent situations. ERISA §503 requires any employer-plan to provide a full-and-fair internal review with specific clinical rationale before external review is triggered.

## Correcting the Record

The core of your appeal is a clear, factual correction: Galafold holds FDA approval and the patient's situation falls within the approved labeling. To make this case:

• Attach the FDA-approved prescribing label (available on FDA.gov or DailyMed) and highlight the approved indication language.
• Document amenability: Include the genetic sequencing report identifying the specific GLA mutation and, where available, the amenability assessment used to confirm eligibility under the prescribing label's criteria.
• Prescriber attestation: The treating physician should write a brief letter affirming that the prescription is for the FDA-approved indication and that the patient's mutation status meets the criteria described in the label.

## Documentation Categories

• Genetic laboratory report confirming GLA mutation identity
• Amenability determination (from the assay or the prescribing label's amenability table)
• Diagnosis records confirming Fabry disease
• Prescriber letter tying the patient's case to the FDA-approved indication
• Copy of the FDA-approved prescribing label

Present these materials together in a structured rebuttal that walks the reviewer from the denial reason directly to the documentation that refutes it. Keep the language factual and cite the label directly rather than relying on secondary sources.