Vestronidase Alfa
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Also known as MepseviiUX-003UX003Vestronidasa alfaVestronidase alfa vjbkVestronidase alfa-vjbk
Summary
Vestronidase Alfa (CHEMBL3707382) is an approved enzyme (ATC A16AB18); indicated across 2 conditions including mucopolysaccharidosis type 7.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Enzyme
- ATC class: A16AB18
- Indications: 2 conditions
- Clinical trials: 6
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL3707382 |
| Name | Vestronidase Alfa |
| Type | Enzyme |
| Max phase | 4 |
| ATC | A16AB18 |
Also known as: Mepsevii, UX-003, UX003, Vestronidasa alfa, Vestronidase alfa, Vestronidase alfa vjbk, Vestronidase alfa-vjbk, VESTRONIDASE ALFA
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
2 indications (1 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 |
|---|---|---|---|
| mucopolysaccharidosis type 7 | 4 | MONDO:0009662 | MONDO:0009662 |
| mucopolysaccharidosis | 3 | MONDO:0019249 | MONDO:0019249 |
Clinical trials
Total trials: 6.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE3 | 2 |
| Not specified | 2 |
| PHASE1/PHASE2 | 1 |
| PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT02230566 | PHASE3 | COMPLETED | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) |
| NCT02432144 | PHASE3 | COMPLETED | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) |
| NCT01856218 | PHASE1/PHASE2 | COMPLETED | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) |
| NCT02418455 | PHASE2 | COMPLETED | Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age |
| NCT03775174 | Not specified | AVAILABLE | Expanded Access to Mepsevii |
| NCT02097251 | Not specified | NO_LONGER_AVAILABLE | An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient |
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).