Drugs / RGX-111
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | mucopolysaccharidosis, type 10 | 2026-01-28 | “the U.S. Food and Drug Administration (FDA) placed a clinical hold on its investigational gene therapy, RGX-111, for the treatment of MPS I” regenxbio.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03580083 | Apr 2019 → Jul 2027 | Hurler syndrome, Hurler-Scheie syndrome | REGENXBIO Inc. | Suspended | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2026-01-28 | REGENXBIO Inc. | Regulatory REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs regenxbio.com ↗
The FDA also placed a clinical hold on RGX-121, for the treatment of MPS II, also known as Hunter Syndrome, citing the similarities in products, study populations, and shared risk between the clinical studies. |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2026-01-28 | REGENXBIO Inc. | REGENXBIO Announces Regulatory Update on Ultra Rare MPS Programs regenxbio.com ↗ |
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RGX-111 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03580083 ↗ |
| Action | Restore | “RGX-111 is designed to deliver a functioning gene enabling the production of IDUA in the brain.” NCT03580083 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.” NCT03580083 ↗ |
| Route | Other | “receive a single dose of RGX-111 administered using an investigational device by IC or IVR injection.” NCT03580083 ↗ |
| Target | IDUA | “functional copy of the α-L-iduronidase (IDUA) gene” NCT03580083 ↗ |