donidalorsen
Nucleic acidapprovedOrphan Drug FDA
Approved
Aug 21, 2025
Clinical trial activity
4 trials · 1 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
3
Phase 3
3
Phase 4
0
Earliest trial started Dec 31, 2021 (NCT05139810)
Timeline
2020s
- Jan 1, 2021
Ionis Pharmaceuticals, Inc. — Earliest Phase 3 Sponsor(trial)
- Sep 19, 2023
Orphan Drug Designation
- Aug 21, 2025
Earliest FDA Approval
- Aug 21, 2025
Ionis Pharmaceuticals, Inc. — Marketing Organization
Indications
Approved for
Mechanism of action
- Plasma kallikreinANTISENSE INHIBITOR
KLKB1 antisense inhibitor
Approval history
- approvedAug 21, 2025
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4650236 ↗
- ChEMBLCHEMBL4650235 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07298447 | Phase 3 | Jan 31, 2026 | Ionis Pharmaceuticals, Inc. |
| NCT05392114 | Phase 3 | Jul 13, 2022 | Ionis Pharmaceuticals, Inc. |
| NCT05139810 | Phase 3 | Dec 31, 2021 | Ionis Pharmaceuticals, Inc. |
| NCT06415448 | N/A | — | Ionis Pharmaceuticals, Inc. |
Showing 4 of 4 trials
Page 1 / 1
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | For profit | 4 | 4 | 2 | 2021 |
1 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Ionis Pharmaceuticals, Inc. | For profit | MKTG | Aug 21, 2025 |