uniQure Gains FDA Orphan Drug Status for Fabry Disease Therapy

An announcement from uniQure ( (QURE) ) is now available.

uniQure N.V. has announced the U.S. FDA’s Orphan Drug Designation for their investigational gene therapy AMT-191, targeting Fabry disease, a rare genetic disorder. This status can lead to benefits such as tax credits and seven years of market exclusivity upon approval. AMT-191, designed to produce the deficient enzyme in Fabry disease via a one-time infusion, is currently undergoing a Phase I/IIa clinical trial in the United States. This major step emphasizes the company’s commitment to advancing gene therapies and providing new treatment options for patients with severe medical needs.

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