Uniqure announces orphan drug designation granted to amt-191 for the treatment of fabry disease

Lexington, mass. and amsterdam, sept. 23, 2024 (globe newswire) -- uniqure n.v. (nasdaq: qure), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the u.s. food and drug administration (fda) has granted orphan drug designation to amt-191, uniqure's investigational gene therapy for the treatment of fabry disease, a rare, inherited genetic disease. in august 2024, uniqure announced the dosing of the first patient in its u.s., multi-center, open-label phase i/iia trial of amt-191.
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