uniQure N.V. announced on September 23, 2024, that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to AMT-191, its investigational gene therapy for the treatment of Fabry disease. This designation is a crucial step for the AAV5-based gene therapy, which aims to address the α-galactosidase A (aGAL-A) enzyme deficiency in patients with this rare, inherited genetic disease.
The Orphan Drug Designation provides uniQure with several incentives, including tax credits, grants, and waivers of certain administrative fees for clinical trials. Furthermore, it offers seven years of market exclusivity in the United States following potential drug approval, which is a significant commercial advantage for a therapy targeting a rare condition affecting an estimated one in 40,000 to one in 117,000 individuals.
This regulatory recognition follows the dosing of the first patient in the U.S. Phase I/IIa trial of AMT-191 in August 2024. Management stated that this designation highlights the need for new gene therapies for Fabry disease, given the suboptimal effectiveness of current chronic treatments, and supports the rapid generation of clinical proof-of-concept data, with initial data anticipated in 2025.
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