vestronidase alfa-vjbk
Trade name: mepsevii
Approved
Nov 15, 2017
Vestronidase alfa, sold under brand name Mepsevii, is a medication for the treatment of Sly syndrome. It is a recombinant form of the human enzyme beta-glucuronidase. It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called mucopolysaccharidosis type VII, also known as Sly syndrome. MPS VII is an extremely rare, progressive condition that affects most tissues and organs. — Wikipedia
Clinical trial activity
1 trials · 1 clinical orgs · 1 marketing orgs
Timeline
2010s
- Feb 16, 2012
Orphan Drug Designation
- Nov 15, 2017
Ultragenyx Inc. — Earliest FDA Approval
- Nov 15, 2017
Ultragenyx Inc. — Marketing Organization
- Nov 15, 2017
Ultragenyx Inc. — NDA Organization
Indications
Approved for
Mechanism of action
- GlycosaminoglycansHYDROLYTIC ENZYME
Glycosaminoglycans hydrolytic enzyme
Approval history
- approvedPriority reviewFast trackNov 15, 2017
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
Oral
No
Parenteral
Yes
Topical
No
Sources
- WikipediaVestronidase alfa ↗
- NCATS7XZ4062R17 ↗
- ChEMBLCHEMBL3990048 ↗
- ChEMBLCHEMBL3707382 ↗
- WikipediaVestronidase alfa-vjbk ↗
Also known as
- vestronidase alfa-vjbk
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT03775174 | N/A | — | Ultragenyx Inc. |
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Ultragenyx Inc. | For profit | 1 | 1 | 1 | — |
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Ultragenyx Inc. | For profit | MKTG | Nov 15, 2017 |
| Ultragenyx Inc. | For profit | NDA | Nov 15, 2017 |