UniQure's Fabry Disease Treatment Gets Orphan Drug Designation from FDA

By Denny Jacob

Gene therapy company uniQure disclosed that the Food and Drug Administration granted orphan drug designation to AMT-191, a potential treatment for Fabry disease.

Fabry disease is a genetic disorder resulting from a deficiency of the galactosidase alpha gene which can then affect multiple systems in the body.

The FDA's orphan drug designation provides certain incentives including tax credits and certain administrative fees for clinical trials being waived. It provides a special status for investigational drugs being developed for rare diseases considered to affect only up to 200,000 people in the U.S.

UniQure disclosed the dosing of the first patient in its Phase 1/2a trial of AMT-191 in August, which is being conducted in the U.S.

Chief Medical Officer Walid Abi-Saab said the company is looking forward to generating proof-of-concept data and providing initial data in 2025.

Write to Denny Jacob at denny.jacob@wsj.com

(END) Dow Jones Newswires

September 23, 2024 07:51 ET (11:51 GMT)

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