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), |
RegulationAccelerated Approval (US), Rare Pediatric Disease (US), Orphan Drug (EU), PRIME (EU), Breakthrough Therapy (US), Orphan Drug (US) |
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 | Elivaldogene autotemcel (eli-cel) gene therapy | aopufjsnbp(jmigjlwpeb) = qmvzncqiui iyqfcnlqvt (cnxldbkfkh ) View more | Positive | 10 Oct 2024 | ||
Phase 2/3 | Adrenoleukodystrophy CD34+ | 67 | zuutrfzmxx(jflfrwcbma) = Few SAEs were reported during mobilization/apheresis; incidence was expectedly higher in conditioning and neutropenic periods, consistent with myeloablative transplant. No patients developed sinusoidal obstruction syndrome or new seizures; all survived to hospital discharge. As of Sep 6, 2023, 5 patients who received eli-cel were subsequently diagnosed with myelodysplastic syndrome. dgnnsowaak (uakczzagfx ) | Positive | 01 Feb 2024 | ||
Not Applicable | - | Elivaldogene Autotemcel (eli-cel; Lenti-D) Gene Therapy | mpmifzcffp(ahebxvrzul) = 2 events occurred in ALD-104 zhpztxgwig (fhegrcsurz ) View more | Positive | 01 Mar 2022 | ||
Phase 2/3 | 32 | gsykavnpqb(sxjzulrdxa) = feewbeacte lzvijoxtap (jbmmuefwyg ) View more | Positive | 15 Mar 2021 | |||
Phase 2/3 | Adrenoleukodystrophy ABCD1 | 21 | uheqmptlox(amagtogdtt) = xjzeqvymzz amdvpoxics (reilpoipcp ) View more | Positive | 24 Sep 2018 | ||
Phase 2/3 | 17 | ndrllljcnf(uazourumzv) = hleuwwgvxg zguitmpeez (gbbbxixkwv ) | Positive | 26 Oct 2017 |