FDA and European Commission Grants Orphan Drug Designation to UX053

Release Date: 14-Sep-2021

Ultragenyx Pharmaceutical Inc. receives US FDA and European Commission for UX053 for the treatment of Glycogen Storage Disease Type III (GSDIII). UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debranching enzyme encapsulated in a lipid nanoparticle (LNP) designed to provide the deficient protein in GSDIII.


Glycogen Storage Disease Type III (GSDIII) affects more than 10,000 patients in the developed world. Enrollment in a Phase 1/2 clinical trial evaluating the tolerability, safety, and efficacy of UX053 in adults with GSDIII is expected to begin in the second half of 2021.


Ultragenyx Pharmaceutical is creating UX053 and a number of other mRNA therapies in the preclinical stage for undisclosed indications pursuant to its collaboration with Arcturus Therapeutics which is a clinical-stage messenger RNA medicines company.

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