voretigene neparvovec
Trade name: Luxturna
Gene therapyapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Dec 19, 2017
Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. — Wikipedia
Clinical trial activity
5 trials · 5 clinical orgs · 1 marketing orgs
Phase 1
3
Phase 2
3
Phase 3
2
Phase 4
0
Earliest trial started Sep 1, 2007 (NCT00516477)
Timeline
2000s
- Jan 1, 2007
Spark Therapeutics — Earliest Phase 1 Sponsor(trial)
2010s
- Jan 1, 2012
Spark Therapeutics — Earliest Phase 3 Sponsor(trial)
- Mar 18, 2015
Orphan Drug Designation
- Dec 18, 2017
Spark Therapeutics — First NDA Organization
- Dec 18, 2017
Spark Therapeutics — NDA Organization
- Dec 19, 2017
Earliest FDA Approval
Indications
Approved for
Mechanism of action
- Retinoid isomerohydrolaseEXOGENOUS GENE
Retinoid isomerohydrolase exogenous gene
Virus vector-based gene therapy that provides a correct copy of the human RPE65 gene.
- Retinoid isomerohydrolaseSupplementation
RPE65 supplementation
Approval history
- approvedBreakthroughDec 19, 2017
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
First in class
Sources
- ChEMBLCHEMBL3707275 ↗
- WikipediaVoretigene neparvovec ↗
- NCATS2SPI046IKD ↗
Also known as
- aav2-hrpe65v2
- raav2-hrpe65
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04516369 | Phase 3 | Oct 26, 2020 | Novartis |
| NCT00999609 | Phase 3 | Oct 1, 2012 | Spark Therapeutics, University of Iowa, University of Pennsylvania |
| NCT01208389 | Phase 1/Phase 2 (Phase 2) | Nov 1, 2010 | Spark Therapeutics, University of Pennsylvania |
| NCT00821340 | Phase 1 | Jan 1, 2010 | Hebrew University |
| NCT00516477 | Phase 1 | Sep 1, 2007 | Spark Therapeutics, University of Pennsylvania |
Showing 5 of 5 trials
Page 1 / 1
Organizations
Research & Development (5)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Spark Therapeutics | For profit | 3 | 3 | 3 | 2007 |
| University of Pennsylvania | Academic/Hospital | 3 | 0 | 3 | 2007 |
| Hebrew University | Academic/Hospital | 1 | 1 | 1 | 2010 |
| Novartis | For profit | 1 | 1 | 1 | 2020 |
| University of Iowa | Academic/Hospital | 1 | 0 | 1 | 2012 |
5 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Spark Therapeutics | For profit | NDA | Dec 18, 2017 |