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Orphan Drug Designation and RMAT: What Each Does, and Doesn't, Mean

Glafabra
Posted at Oct 26

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 t...more

Categories: Advanced Therapeutics | Autoimmune Disease | Cell & Gene Therapy | Clinical Research | Drug Development | Fabry Disease | Rare Disease

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