Drug Type Gene therapy, Stem cell therapy |
Synonyms Adrenoleucodystrophy gene therapy - bluebird bio, eli-cel, Elivaldogene autotemcel + [8] |
Target |
Mechanism ABCD1 gene stimulants |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseApproved |
First Approval Date EU (16 Jul 2021), |
RegulationBreakthrough Therapy (US), Accelerated Approval (US), Orphan Drug (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Accelerated assessment (EU) |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Adrenoleukodystrophy | EU | 16 Jul 2021 | |
Adrenoleukodystrophy | IS | 16 Jul 2021 | |
Adrenoleukodystrophy | LI | 16 Jul 2021 | |
Adrenoleukodystrophy | NO | 16 Jul 2021 |
Phase 2/3 | 32 | dsnlkgzeij(geqxiqjcxf) = mrzfixvugx btyiaqaenb (oxvrgqvaou ) View more | Positive | 15 Mar 2021 | |||
Phase 2/3 | Adrenoleukodystrophy ABCD1 | 21 | lobyqpvojr(ocdlshctgr) = yuiwuhswiu gatxayqngg (ojndwbygou ) View more | Positive | 24 Sep 2018 | ||
Phase 2/3 | 17 | ijeysrfalo(bmsimvwsli) = livsqerxzc auxtdpvufm (lsjghyvehh ) | Positive | 26 Oct 2017 |