viltolarsen
Trade name: Viltepso
Nucleic acidapprovedOrphan Drug FDAFast Track FDA
Approved
Aug 12, 2020
Clinical trial activity
8 trials · 5 clinical orgs · 1 marketing orgs
Phase 1
1
Phase 2
5
Phase 3
2
Phase 4
1
Earliest trial started Jun 1, 2013 (NCT02081625)
Timeline
2010s
- Jan 1, 2013
Earliest Phase 1 Sponsor(trial)
- Jan 1, 2016
Nippon Shinyaku Co Ltd — Earliest Phase 2 Sponsor(trial)
- Jan 12, 2017
Orphan Drug Designation
- Jan 1, 2019
Nippon Shinyaku Co Ltd — Earliest Phase 3 Sponsor(trial)
2020s
- Aug 12, 2020
Nippon Shinyaku Co Ltd — Earliest FDA Approval
- Aug 12, 2020
Nippon Shinyaku Co Ltd — Marketing Organization
- Aug 12, 2020
Nippon Shinyaku Co Ltd — NDA Secondary Org
- Aug 12, 2020
Nippon Shinyaku Co Ltd — NDA Organization
Indications
Studied for
Mechanism of action
- Dystrophin pre-mRNAANTISENSE INHIBITOR
Dystrophin pre-mRNA antisense inhibitor
- Dystrophin pre-mRNAPOSITIVE MODULATOR
Dystrophin pre-mRNA positive modulator
Approval history
- approvedPriority reviewAcceleratedFast trackAug 12, 2020
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
- Multi-specific
- No
Oral
No
Parenteral
Yes
Topical
No
Sources
- ChEMBLCHEMBL4298062 ↗
Also known as
- ns-065
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04956289 | Phase 2 | Jul 1, 2021 | Nippon Shinyaku Co Ltd |
| NCT04768062 | Phase 3 | Apr 13, 2021 | Nippon Shinyaku Co Ltd |
| NCT04687020 | Phase 4 | Feb 28, 2021 | Nippon Shinyaku Co Ltd |
| NCT04060199 | Phase 3 | Sep 30, 2019 | Nippon Shinyaku Co Ltd |
| NCT03167255 | Phase 2 | Jul 1, 2017 | Cooperative International Neuromuscular Research Group, Nippon Shinyaku Co Ltd, Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), University of Pittsburgh |
| NCT02740972 | Phase 2 | Dec 1, 2016 | Cooperative International Neuromuscular Research Group, Nippon Shinyaku Co Ltd, Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS), University of Pittsburgh |
| NCT02081625 | Phase 1 | Jun 1, 2013 | National Center of Neurology and Psychiatry, Japan, Nippon Shinyaku Co Ltd |
| NCT04337112 | N/A | — | Nippon Shinyaku Co Ltd |
Showing 8 of 8 trials
Page 1 / 1
Organizations
Research & Development (5)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Nippon Shinyaku Co Ltd | For profit | 8 | 7 | 5 | 2013 |
| Cooperative International Neuromuscular Research Group | Academic/Hospital | 2 | 0 | 1 | 2016 |
| Therapeutic Research in Neuromuscular Disorders Solutions (TRiNDS) | For profit | 2 | 0 | 1 | 2016 |
| University of Pittsburgh | Academic/Hospital | 2 | 0 | 1 | 2016 |
| National Center of Neurology and Psychiatry, Japan | Government | 1 | 1 | 1 | 2013 |
5 organizations
Page 1 / 1
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Nippon Shinyaku Co Ltd | For profit | MKTG | Aug 12, 2020 |
| Nippon Shinyaku Co Ltd | For profit | NDA2 | Aug 12, 2020 |
| Nippon Shinyaku Co Ltd | For profit | NDA | Aug 12, 2020 |