uniQure Biopharma Granted Orphan Drug Designation For Its 'adeno-associated Viral Vector Serotype 5 Encoding Human Alpha-galactosidase A' For The Treatment Of Fabry Disease
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uniQure Biopharma has been granted Orphan Drug Designation by the FDA for its gene therapy targeting Fabry disease. This designation is for their adeno-associated viral vector serotype 5 encoding human alpha-galactosidase A.

September 19, 2024 | 3:54 pm
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uniQure Biopharma's gene therapy for Fabry disease has received Orphan Drug Designation from the FDA, potentially boosting its market position and future revenues.
The Orphan Drug Designation can provide uniQure with benefits like market exclusivity, tax credits, and reduced regulatory fees, which can enhance its market position and future revenue potential.
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