etranacogene dezaparvovec
Trade name: Hemgenix
Gene therapyapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Nov 22, 2022
Clinical trial activity
3 trials · 3 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
3
Phase 3
2
Phase 4
0
Earliest trial started Jul 24, 2018 (NCT03489291)
Timeline
2010s
- Jan 1, 2018
Earliest Phase 2 Sponsor(trial)
- Apr 17, 2019
Orphan Drug Designation
2020s
- Nov 22, 2022
CSL Behring — Earliest FDA Approval
- Nov 22, 2022
CSL Behring — NDA Secondary Org
- Nov 22, 2022
CSL Behring — NDA Organization
- Jan 1, 2023
CSL Behring — Earliest Phase 3 Sponsor(trial)
Indications
Approved for
Studied for
Mechanism of action
F9 other
- Coagulation factor IXEXOGENOUS PROTEIN
Coagulation factor IX exogenous protein
AAV5 based vector containing a codon-optimised cDNA of the human coagulation Factor IX variant R338L (FIX-Padua) gene under the control of a liver-specific promoter (LP1)
Approval history
- approvedBreakthroughNov 22, 2022
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL5095451 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07080905 | Phase 3 | Jul 28, 2025 | CSL Behring |
| NCT06003387 | Phase 3 | Oct 2, 2023 | CSL Behring |
| NCT03489291 | Phase 2 | Jul 24, 2018 | UniQure N.V., University of Michigan |
Showing 3 of 3 trials
Page 1 / 1
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| CSL Behring | For profit | 2 | 2 | 1 | 2023 |
| UniQure N.V. | For profit | 1 | 1 | 1 | 2018 |
| University of Michigan | Academic/Hospital | 1 | 0 | 1 | 2018 |
3 organizations
Page 1 / 1
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| CSL Behring | For profit | NDA2 | Nov 22, 2022 |
| CSL Behring | For profit | NDA | Nov 22, 2022 |