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GT-GLA-S03 holds FDA Orphan Drug Designation, which recognizes Fabry as a rare disease and provides development incentives, including potential market exclusivity if the product is approved. RMAT (Regenerative Medicine Advanced Therapy) designation is separate: it requires preliminary clinical evidence that a regenerative therapy may address an unmet need in a serious condition. Our current plan targets RMAT in 2029, after U.S. trial data are available.
Interpretation: ODD is in hand; RMAT would come later and depend on clinical data from the U.S. trial.
Uncertainty: Neither designation means approval or guarantees safety or efficacy. RMAT is a future goal, not a certainty, and depends on data we don't have yet.
Questions about the regulatory path? Post them below.
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@Brian Christie @Neva West @Chris Hopkins @Tetsu Yung
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