Account Info
Log Out
English
Back
Log in to access Online Inquiry
Back to the Top

NEWS

uniQure Announces Orphan Drug Designation Granted to AMT-191 for the Treatment of Fabry Disease
uniQure N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce GLA protein.
The Phase I/IIa trial will include two cohorts of up to six adult male patients each, with low and high doses. Patients will be followed for 24 months to assess safety, tolerability, and early efficacy signs. The Orphan Drug Designation provides incentives including tax credits, grants, fee waivers, and seven years of market exclusivity upon approval.
Disclaimer: Community is offered by Moomoo Technologies Inc. and is for educational purposes only. Read more
Translate
Report
4508 Views
Comment
Sign in to post a comment
    Spread kindness and love. Life is short. Don’t let greed eat you.
    2606Followers
    107Following
    28KVisitors
    Follow