uniQure Gains FDA Orphan Drug Status for Fabry Disease Therapy
Company Announcements

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.

See more data about QURE stock on TipRanks’ Stock Analysis page.

Related Articles
TipRanks Auto-Generated NewsdeskuniQure N.V. Reports Q3 2024 Progress and Financials
TheFlyuniQure reports Q3 EPS (91c), consensus ($1.01)
Jason CarruniQure N.V. (QURE) Q3 Earnings Cheat Sheet
Looking for investment ideas? Subscribe to our Smart Investor newsletter for weekly expert stock picks!
Get real-time notifications on news & analysis, curated for your stock watchlist. Download the TipRanks app today! Get the App