Table 4.
ATMPs in the process of marketing authorisation or with a planned marketing launch.
| Product | Developer | Indication | Regulatory/ Clinical status |
|---|---|---|---|
| LuxturnaTM | Novartis | Biallelic RPE65-mediated retinal dystrophy | MAA submitted EMA: CHMP pos. opinion 09/2018 Phase III NCT00999609 Open-label, randomized controlled trial At least 24 patients planned Estimated study completion date 2029 |
| LentiGlobinTM | BlueBird Bio | Transfusion dependant ß-thalassemia, sickle cell disease | MAA submitted EMA: accelerated approval granted Phase III NCT02906202 Single arm, multi site, single dose study Approx. 23 patients planned Estimated study completion date 2020 |
| HabeoTM | Cytori Therapeutics | Hand dysfunction due to scleroderma | EMA: Orphan drug designation granted Phase n.a. NCT02396238 prospective, randomized, multi-center device trial 88 patients enrolled Study completion date 2018 |
| Lenti-DTM | BlueBird Bio | Cerebral adrenoleukodystrophy | Phase II/III NCT01896102 Single arm open label 30 patients planned Estimated study completion date 2021 |
| Neocart® | Histogenics corporation | Cartilage repair | Phase III NCT01066702 245 participants enrolled Randomized, open label Estimated study completion date 2020 |
| ATIR101 | Kiadis Pharma | AML, ALL or myelodysplastic syndrome | MAA submitted response to EMA submitted 03/2018 Phase III NCT02999854 Randomized controlled multicenter open-label study 250 participants planned Estimated study completion date 2021 |
| JCAR 017 | Celgene | DLBCL | EMA: PRIME Phase III NCT03575351 Randomized open label study 182 participants planned Estimated study completion date 2023 |
| bb2121 | Celgene | Multiple myeloma | EMA: PRIME eligibility 11/2017 Phase III NCT03651128 Multicenter randomized open label 381 participants planned Estimated study completion date 2025 |
| Tab-celTM | Atara Biotherapeutics | EBV associated post-transplant lymphoproliferative disorder | Phase III NCT03392142 Multi-center, single arm, open label 33 participants planned Estimated study completion date 2020 |
| Lenadogene nolparvovec | GenSight Biologics SA | Vision loss from Leber hereditary optic neuropathy | Phase III NCT02652767 Randomized, double-masked, sham-controlled clinical trial 36 participants planned Estimated study completion date 2019 |
| REX-001 | Rexgenero | Critical limb ischemia | EMA: Certificate for manufacturing and non-clinical data 01/2018 Phase III NCT03174522 Randomized, double-blind, controlled clinical trial 78 participants planned Estimated study completion date 2021 |
| Multistem | Athersys | Ischemic stroke | Phase III NCT03545607 Randomized, quadruple-masked clinical trial 300 participants planned Estimated study completion date 2021 |
| PLX-PAD | Pluristem therapeutics | Critical limb ischemia | Phase III NCT03006770 Multicenter randomized controlled clinical trials 246 participants planned Estimated study completion date 2020 |
EMA … European Medicines Agency
MAA … Marketing Authorisation Application
CHMP … Committee for Medicinal products for Human Use
AML … Acute Myeloid Leukaemia
ALL … Acute Lymphoblastic Leukaemia
DLBCL … Diffuse Large B Cell Lymphoma
EBV …. Epstein Barr Virus
PRIME …PRIority MEdicines scheme