onasemnogene abeparvovec
Trade name: Zolgensma
Approved
May 24, 2019
Onasemnogene abeparvovec, sold under the brand name Zolgensma, is a gene therapy used to treat spinal muscular atrophy (SMA), a disease causing muscle function loss in children. It involves a one-time infusion of the medication into a vein. It works by providing a new copy of the SMN gene that produces the SMN protein. — Wikipedia
Clinical trial activity
13 trials · 6 clinical orgs · 2 marketing orgs
Earliest trial started Apr 1, 2014 (NCT02122952)
Timeline
2010s
- Aug 8, 2013
AveXis, Inc. — IND Organization
- Aug 8, 2013
AveXis, Inc. — IND Organization
- Jan 1, 2014
AveXis, Inc. — Earliest Phase 1 Sponsor(trial)
- Sep 30, 2014
Orphan Drug Designation
- Jan 1, 2017
AveXis, Inc. — Earliest Phase 3 Sponsor(trial)
- May 24, 2019
AveXis, Inc. — Earliest FDA Approval
- May 24, 2019
AveXis, Inc. — NDA Organization
- May 24, 2019
Novartis — NDA Secondary Org
Indications
Mechanism of action
Survival motor neuron protein agonist
- Survival motor neuron proteinEXOGENOUS GENE
Survival motor neuron protein exogenous gene
Virus vector-based gene therapy that provides a correct copy of the human SMN1 gene.
- Survival motor neuron proteinSupplementation
SMN1
Approval history
- approvedPriority reviewFast trackBreakthroughMay 24, 2019
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- WikipediaOnasemnogene abeparvovec ↗
- NCATSMLU3LU3EVV ↗
- ChEMBLCHEMBL4297240 ↗
Also known as
- avxs-101
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT05335876 | Phase 3 | Dec 31, 2022 | Novartis |
| NCT05386680 | Phase 3 | Sep 30, 2022 | Novartis |
| NCT05089656 | Phase 3 | Dec 21, 2021 | Novartis |
| NCT05073133 | Phase 4 | Oct 20, 2021 | Novartis |
| NCT04851873 | Phase 3 | Sep 8, 2021 | Novartis |
| NCT04042025 | Phase 4 | Sep 1, 2019 | AveXis, Inc. |
| NCT03837184 | Phase 3 | May 15, 2019 | AveXis, Inc., Pharmaceutical Research Associates, Inc. (PRA) |
| NCT03461289 | Phase 3 | Aug 29, 2018 | AveXis, Inc., Sponsor GmbH |
| NCT03505099 | Phase 3 | Apr 10, 2018 | AveXis, Inc. |
| NCT03381729 | Phase 1 | Dec 14, 2017 | AveXis, Inc. |
| NCT03306277 | Phase 3 | Sep 29, 2017 | AveXis, Inc. |
| NCT02122952 | Phase 1 | Apr 1, 2014 | AveXis, Inc., Ohio State University |
| NCT03955679 | N/A | — | AveXis, Inc., United BioSource Corporation |
Organizations
Research & Development (6)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| AveXis, Inc. | For profit | 8 | 7 | 4 | 2014 |
| Novartis | For profit | 5 | 5 | 2 | 2021 |
| Ohio State University | Academic/Hospital | 1 | 0 | 1 | 2014 |
| Pharmaceutical Research Associates, Inc. (PRA) | For profit | 1 | 0 | 1 | 2019 |
| Sponsor GmbH | Academic/Hospital | 1 | 0 | 1 | 2018 |
| United BioSource Corporation | For profit | 1 | 1 | 1 | — |
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| AveXis, Inc. | For profit | NDA | May 24, 2019 |
| AveXis, Inc. | For profit | IND | Aug 8, 2013 |
| Novartis | For profit | NDA2 | May 24, 2019 |