Cacharreando por Pubmed haciendo búsquedas con ATMP, Advanced therapies, Advanced therapies medicinal products etc. he encontrado tres artículos que me han parecido muy interesantes.
- EU Regulatory Pathways for ATMPs: Standard, Accelerated and Adaptive Pathways to Marketing Authorisation. Mol Ther Methods Clin Dev. 2019 Jun 14; 13: 205–232. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6378853/
Un compendio de la normativa que fija los procedimientos de autorización de los ATMP´s
- Technology forecast: advanced therapies in late clinical research, EMA approval or clinical application via hospital exemption. J Mark Access Health Policy. 2019 Apr 19;7(1) https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6493298/
Un trabajo importante para ver los ATMP´s que vienen a través de los CT solicitados, entre otras cosas. Les copiamos la tabla con los que tienen una MAA solicitada y van a venir en 2019 y después. He quitado de su tabla a Luxturna y Zynteglo porque ya tienen la MA.
ATMPs in the process of marketing authorisation or with a planned marketing launch.
| Product | Developer | Indication | Regulatory/ Clinical status |
|---|---|---|---|
| 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 |
- Recent Progress in European Advanced Therapy Medicinal Products and Beyond (Published online 2018 Sep 21). Front Bioeng Biotechnol. 2018; 6: 130. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6161540/
Una gráfica parecida a la nuestra
No veo de qué modo los autores arriba mencionados saben de los MAA, que supongo es a través de la web de la EMA, aunque no sé como porque con haciendo búsquedas con Habeo ó Neocart, que parece ser van a tener una próxima MA no da ningún resultado. Entiendo puede deberse al acceso a ámbitos profesionales ó con registro.
Continuando con las búsquedas. En la web de la EMA he hecho la siguiente búsqueda: KEYWORD "advanced therapies medicinal products" CATEGORIES Human MEDICINE European public assessment reports (EPAR). Da 13 resultados y se cuelan Heparesc y Prevenar13, que no son ATMP´s y faltan Zynteglo, Luxturna e Imlygic.
un 5!!!
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