Drugs / Onasemnogene Abeparvovec-brve
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 4 | NCT07448610 | Jul 2026 → May 2032 expected | spinal muscular atrophy | Novartis Pharmaceuticals | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2025-11-24 | Novartis Pharmaceuticals | Regulatory Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) novartis.com ↗
Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma ® (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 ( SMN1 ) gene, making it the first and only gene replacement therapy available for this broad population. |
All press releases naming this drug 1 release
| Date | Issuer | Release |
|---|---|---|
| 2025-11-24 | Novartis Pharmaceuticals | Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) novartis.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 | Onasemnogene Abeparvovec-brve | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07448610 ↗ |
| Route | Intrathecal | “single, intrathecal dose of onasemnogene abeparvovec-brve” NCT07448610 ↗ |