Drugs / RIVUNATPAGENE MIZIPARVOVEC
RIVUNATPAGENE MIZIPARVOVEC
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | Wilson disease | — | “Ultragenyx submitted an Investigational New Drug (IND) application in December as planned for UX701, an AAV9 gene therapy for the treatment of Wilson Disease.” ultragenyx.com ↗ “Ultragenyx has submitted an Investigational New Drug (IND) application for UX701, an AAV9 gene therapy for the treatment of Wilson Disease.” ultragenyx.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT04884815 | Sep 2021 → Mar 2029 expected | Wilson disease | Ultragenyx Pharmaceutical Inc | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2024-10-03 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease ultragenyx.com ↗
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus 2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. |
| 2023-10-16 | Ultragenyx Pharmaceutical Inc | Results Ultragenyx Announces Program and Pipeline Updates at Analyst Day Including Interim Data from Ongoing Studies in Osteogenesis Imperfecta (OI), Angelman Syndrome (AS) and Wilson Disease ultragenyx.com ↗
Four out of 5 patients in the low-dose Cohort 1 have had reductions in urinary copper and are tapering off of chelators and/or zinc therapy, including 2 of 3 earlier treated patients in the Cohort that are now completely off standard therapy. |
| 2021-01-21 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX701, a New Gene Therapy for the Treatment of Wilson Disease 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 UX701, an investigational AAV9 gene therapy being evaluated for the treatment of Wilson Disease. |
| 2021-01-08 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies ultragenyx.com ↗
IND for UX701 for Wilson Disease Submitted; Expect to Enter Clinic in First Half 2021 using AAV Drug Product Made by HeLa PCL Platform |
| 2020-12-09 | Ultragenyx Pharmaceutical Inc | Regulatory Ultragenyx Announces Orphan Drug Designation for UX701 for the Treatment of Wilson Disease ultragenyx.com ↗ |
All press releases naming this drug 8 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 2 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RIVUNATPAGENE MIZIPARVOVEC | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314707 ↗ |
| Known as | DTX-701 | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314707 ↗ |
| Known as | Rivunatpagen miziparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL5314707 ↗ |
| Known as | UX701 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04884815 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “AAV Gene Therapy” NCT04884815 ↗ |
| Route | Intravenous | “peripheral intravenous (IV) infusion” NCT04884815 ↗ |