Ultragenyx Gets FDA, EC Orphan Designation for UX053 in Metabolic Disease

By Colin Kellaher

Ultragenyx Pharmaceutical Inc. on Tuesday said the U.S. Food and Drug Administration and the European Commission granted orphan-drug designation to UX053 for the treatment of glycogen storage disease type III.

The Novato, Calif., biopharmaceutical company said there are currently no approved treatment options for the rare metabolic disease that results in glycogen accumulation in the liver and muscle and affects more than 10,000 patients in the developed world.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

Ultragenyx said it expects to begin enrollment in a Phase 1/2 clinical trial evaluating the safety, tolerability, and efficacy of UX053 in adults in the second half of 2021.

Write to Colin Kellaher at colin.kellaher@wsj.com

(END) Dow Jones Newswires

July 27, 2021 08:34 ET (12:34 GMT)

Ultragenyx Pharmaceutical (NASDAQ:RARE)
Historical Stock Chart
From Mar 2024 to Apr 2024