tofersen
Trade name: Qalsody
Nucleic acidapprovedOrphan Drug FDAFast Track FDA
Approved
Apr 25, 2023
Clinical trial activity
8 trials · 5 clinical orgs · 2 marketing orgs
Phase 1
3
Phase 2
2
Phase 3
1
Phase 4
1
Earliest trial started Jan 31, 2016 (NCT02623699)
Timeline
2020s
- Jan 1, 2021
- Apr 25, 2023
Biogen-IDEC — Earliest FDA Approval
- Apr 25, 2023
Biogen — Marketing Organization
- Apr 25, 2023
Biogen-IDEC — NDA Secondary Org
- Apr 25, 2023
Biogen-IDEC — NDA Organization
- Jan 1, 2025
Earliest Phase 2 Sponsor(trial)
Indications
Approved for
Mechanism of action
- Superoxide dismutaseANTISENSE INHIBITOR
SOD1 antisense inhibitor
- SOD1 mRNAANTISENSE INHIBITOR
SOD1 mRNA inhibitor
Thought to reduce the production of SOD1 protein
- SOD1 mRNAINHIBITOR
SOD1 mRNA inhibitor
Thought to reduce the production of SOD1 protein
Approval history
- approvedPriority reviewAcceleratedFast trackApr 25, 2023
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
- Multi-specific
- No
Oral
No
Parenteral
Yes
Topical
No
First in class
Sources
- ChEMBLCHEMBL3833346 ↗
Also known as
- biib067
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07294144 | Phase 2 | Dec 31, 2025 | Biogen, Washington University in St. Louis |
| NCT07223723 | Phase 4 | Dec 2, 2025 | Biogen |
| NCT04856982 | Phase 3 | May 17, 2021 | Biogen |
| NCT03764488 | Phase 1 | Dec 20, 2018 | Biogen |
| NCT03070119 | Phase 1 | Mar 8, 2017 | Biogen, Ionis Pharmaceuticals, Inc. |
| NCT02975986 | N/A | Jan 31, 2017 | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), The University of Texas, Dallas |
| NCT02623699 | Phase 1 | Jan 31, 2016 | Biogen, Ionis Pharmaceuticals, Inc. |
| NCT04972487 | N/A | — | Biogen |
Showing 8 of 8 trials
Page 1 / 1
Organizations
Research & Development (5)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Biogen | For profit | 7 | 6 | 5 | 2016 |
| Ionis Pharmaceuticals, Inc. | For profit | 2 | 0 | 1 | 2016 |
| National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Government | 1 | 0 | 1 | 2017 |
| The University of Texas, Dallas | Academic/Hospital | 1 | 1 | 1 | 2017 |
| Washington University in St. Louis | Academic/Hospital | 1 | 1 | 1 | 2025 |
5 organizations
Page 1 / 1
Marketing (3)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Biogen | For profit | MKTG | Apr 25, 2023 |
| Biogen-IDEC | For profit | NDA2 | Apr 25, 2023 |
| Biogen-IDEC | For profit | NDA | Apr 25, 2023 |