beremagene geperpavec
Trade name: VYJUVEK
Gene therapyapprovedOrphan Drug FDA
Approved
May 19, 2023
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 Aug 31, 2020 (NCT04491604)
Timeline
2010s
- Nov 2, 2017
Orphan Drug Designation
2020s
- Jan 1, 2020
Krystal Biotech, Inc. — Earliest Phase 3 Sponsor(trial)
- May 19, 2023
Krystal Biotech, Inc. — Earliest FDA Approval
- May 19, 2023
Krystal Biotech, Inc. — NDA Secondary Org
- May 19, 2023
Krystal Biotech, Inc. — NDA Organization
- Jan 1, 2025
Earliest Phase 2 Sponsor(trial)
Indications
Approved for
Mechanism of action
- Collagen alpha-1(VII) chainEXOGENOUS GENE
Collagen alpha-1(VII) chain exogenous gene
- Collagen alpha-1(VII) chainSupplementation
supplementation - COL7A1
Approval history
- approvedMay 19, 2023
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Availability
- Prescription Only
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4594328 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06731933 | Phase 2 | Feb 28, 2025 | Epidermolysis Bullosa Research Partnership, Stanford University |
| NCT04917874 | Phase 3 | May 25, 2021 | Krystal Biotech, Inc. |
| NCT04491604 | Phase 3 | Aug 31, 2020 | Krystal Biotech, Inc. |
Showing 3 of 3 trials
Page 1 / 1
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Krystal Biotech, Inc. | For profit | 2 | 2 | 1 | 2020 |
| Epidermolysis Bullosa Research Partnership | Academic/Hospital | 1 | 0 | 1 | 2025 |
| Stanford University | Academic/Hospital | 1 | 1 | 1 | 2025 |
3 organizations
Page 1 / 1
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Krystal Biotech, Inc. | For profit | NDA2 | May 19, 2023 |
| Krystal Biotech, Inc. | For profit | NDA | May 19, 2023 |