Atidarsagene autotemcel
Trade name: Libmeldy
Cell therapyapprovedOrphan Drug FDA
Approved
Mar 18, 2024
Clinical trial activity
2 trials · 3 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
2
Phase 3
2
Phase 4
0
Earliest trial started Dec 15, 2017 (NCT03392987)
Timeline
2010s
- Jan 1, 2017
Earliest Phase 3 Sponsor(trial)
- Mar 8, 2018
Orphan Drug Designation
2020s
- Mar 18, 2024
Kyowa Kirin — Earliest FDA Approval
- Mar 18, 2024
Kyowa Kirin — NDA Organization
Indications
Approved for
Mechanism of action
- ARSAEXOGENOUS GENE
ARSA exogenous gene
Gene therapy.
- Cerebroside-sulfataseSupplementation
ARSA supplementation
Approval history
- approvedMar 18, 2024
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4802262 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04283227 | Phase 3 | Mar 31, 2020 | Orchard Therapeutics Limited, The Foundation Institute San Raffaele G. Giglio of Cefalù |
| NCT03392987 | Phase 3 | Dec 15, 2017 | GlaxoSmithKline, The Foundation Institute San Raffaele G. Giglio of Cefalù |
Showing 2 of 2 trials
Page 1 / 1
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| The Foundation Institute San Raffaele G. Giglio of Cefalù | Academic/Hospital | 2 | 0 | 1 | 2017 |
| GlaxoSmithKline | For profit | 1 | 1 | 1 | 2017 |
| Orchard Therapeutics Limited | For profit | 1 | 1 | 1 | 2020 |
3 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Kyowa Kirin | For profit | NDA | Mar 18, 2024 |