uniQure receives FDA Orphan Drug Designation for AMT-191 gene therapy for Fabry disease

uniQure N.V.

FDA granted Orphan Drug Designation to AMT-191 for Fabry disease on Sep 23, 2024.
AMT-191 is a one-time IV AAV5-based gene therapy delivering a GLA transgene.
First patient dosed in Aug 2024 in a U.S. multi-center open-label Phase I/IIa trial.
Designation provides tax credits, fee waivers, and 7-year US market exclusivity if approved.
Initial Phase I/IIa clinical data expected in 2025.

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