Benzinga | Jul 27, 2021 11:13AM EDT

Ultragenyx Receives Orphan Drug Designation from FDA and European Commission for UX053, an Investigational mRNA-therapy for the Treatment of Glycogen Storage Disease Type III

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) and the European Commission have granted Orphan Drug Designation for UX053 for the treatment of Glycogen Storage Disease Type III (GSDIII), a metabolic disease that affects more than 10,000 patients in the developed world. Enrollment in a Phase 1/2 clinical trial evaluating the safety, tolerability, and efficacy of UX053 in adults with GSDIII is expected to begin in the second half of 2021.