Drugs / UX053
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04990388 | Oct 2021 → Mar 2023 | glycogen storage disease III | Ultragenyx Pharmaceutical Inc | Terminated | No outcome recorded Stop: Business |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2021-07-27 | Ultragenyx Pharmaceutical Inc | Regulatory 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.com ↗
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. |
| 2021-03-08 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III ultragenyx.com ↗
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) has cleared the Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII). |
All press releases naming this drug 3 releases
| Date | Issuer | Release |
|---|---|---|
| 2021-12-01 | Ultragenyx Pharmaceutical Inc | Ultragenyx Announces First Patient Dosed in Phase 1/2 Clinical Study of UX053, an mRNA Therapy for the Treatment of Glycogen Storage Disease Type III ultragenyx.com ↗ |
| 2021-07-27 | Ultragenyx Pharmaceutical Inc | 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.com ↗ |
| 2021-03-08 | Ultragenyx Pharmaceutical Inc | Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III ultragenyx.com ↗ |
Evidence & citations 2 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | UX053 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04990388 ↗ |
| Route | Intravenous | “peripheral intravenous (IV) infusion of UX053” NCT04990388 ↗ |