Drug Type AAV based gene therapy |
Synonyms Lixmabegagene Relduparvovec |
Target |
Mechanism β-galactosidase modulators(Beta-galactosidase modulators) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhaseDiscontinuedPhase 1/2 |
First Approval Date- |
RegulationRare Pediatric Disease (US), Fast Track (US), Orphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Gangliosidosis, GM1 | NDA/BLA | US | 11 May 2021 | |
Gangliosidosis, GM1 | NDA/BLA | FR | 11 May 2021 | |
Gangliosidosis, GM1 | NDA/BLA | GB | 11 May 2021 |