Voretigene Neparvovec
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Also known as LuxturnaSPK-RPE65Voretigen neparvovecVoretigene neparvovec rzylVoretigene neparvovec-rzyl
Summary
Voretigene Neparvovec (CHEMBL3707275) is an approved gene (ATC S01XA27); indicated across 3 conditions including eye disorder and leber congenital amaurosis.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Gene
- ATC class: S01XA27
- Indications: 3 conditions
- Clinical trials: 6
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL3707275 |
| Name | Voretigene Neparvovec |
| Type | Gene |
| Max phase | 4 |
| ATC | S01XA27 |
Also known as: Luxturna, SPK-RPE65, Voretigen neparvovec, Voretigene neparvovec, Voretigene neparvovec rzyl, Voretigene neparvovec-rzyl, VORETIGENE NEPARVOVEC
Targets
Targets
No target linkage available.
Bioactivity
No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
3 indications (3 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| eye disorder | 4 | MONDO:0005328 | EFO:0003966 |
| Leber congenital amaurosis | 4 | MONDO:0018998 | MONDO:0018998 |
| retinitis pigmentosa | 4 | MONDO:0019200 | HP:0000580 |
Clinical trials
Total trials: 6.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 2 |
| Not specified | 2 |
| PHASE1/PHASE2 | 1 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00999609 | PHASE3 | ACTIVE_NOT_RECRUITING | Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis |
| NCT04516369 | PHASE3 | ACTIVE_NOT_RECRUITING | Study of Efficacy and Safety of Voretigene Neparvovec in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy |
| NCT01208389 | PHASE1/PHASE2 | ACTIVE_NOT_RECRUITING | Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 |
| NCT00516477 | PHASE1 | COMPLETED | Safety Study in Subjects With Leber Congenital Amaurosis |
| NCT03602820 | Not specified | ACTIVE_NOT_RECRUITING | Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2) |
| NCT03597399 | Not specified | COMPLETED | A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).