Drugs / voretigene neparvovec
voretigene neparvovec
also known as
voretigene neparvovec-rzyl · AAV-2-HRPE65V2 · Luxturna · SPK-RPE65 · Voretigen neparvovec
targets RPE65
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Luxturna | — | 2018-11-22 | europa.eu ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT00516477 | Sep 2007 → Mar 2018 | Leber congenital amaurosis | Spark Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01208389 | Nov 2010 → Oct 2027 expected | Leber congenital amaurosis | Spark Therapeutics, Inc. | Active not recruiting | No outcome recorded |
| Phase 32 trials · 1 met primary | ||||||
| Phase 3 | NCT04516369 | Nov 2020 → Apr 2022 | RPE65-related recessive retinopathy | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT00999609 | Oct 2012 → Jul 2015 overdue | Leber congenital amaurosis 2 | Spark Therapeutics, Inc. | Active not recruiting | Met primary |
News releases announcing trial results or a regulatory action · 2
| Date | Issuer | Release |
|---|---|---|
| 2018-11-23 | Novartis Pharmaceuticals | Regulatory Novartis announces landmark EU approval for one-time gene therapy Luxturna® to restore vision in people with rare inherited retinal disease novartis.com ↗ |
| 2018-09-21 | Novartis Pharmaceuticals | Regulatory Novartis announces positive CHMP opinion for one-time gene therapy Luxturna® to treat children and adults with rare inherited retinal disease novartis.com ↗ |
All press releases naming this drug 2 releases
| Date | Issuer | Release |
|---|---|---|
| 2018-11-23 | Novartis Pharmaceuticals | Novartis announces landmark EU approval for one-time gene therapy Luxturna® to restore vision in people with rare inherited retinal disease novartis.com ↗ |
| 2018-09-21 | Novartis Pharmaceuticals | Novartis announces positive CHMP opinion for one-time gene therapy Luxturna® to treat children and adults with rare inherited retinal disease novartis.com ↗ |
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Voretigene neparvovec | “Voretigene neparvovec (VN) is the first available gene therapy for patients with biallelic RPE65-mediated inherited retinal dystrophy who have sufficient viable retinal cells.” PMID 38254722 ↗ Jan 2024 |
| Known as | AAV-2-HRPE65V2 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707275 ↗ |
| Known as | Luxturna | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707275 ↗ |
| Known as | SPK-RPE65 | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707275 ↗ |
| Known as | Voretigen neparvovec | ChEMBL registry synonym — accepted as the source's own label CHEMBL3707275 ↗ |
| Known as | voretigene neparvovec-rzyl | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00516477 ↗ |
| Action | Restore | “subretinal injection of an adeno-associated virus (AAV) carrying the normal cDNA of that gene (AAV2-hRPE65v2)” PMID 21606598 ↗ May 2011 |
| Modality | Gene therapy (AAV / viral vector) | “Using Adeno-Associated Viral Vector to Deliver the Gene for Human RPE65 Into the Retinal Pigment Epithelium (RPE)” NCT00516477 ↗ |
| Route | Other | “Subretinal administration of gene therapy vector AAV2-hRPE65v2” NCT00999609 ↗ |
| Target | RPE65 | “providing the normal human RPE65 gene to the cells of the retina.” NCT00999609 ↗ |