Approval of First CAR-Ts: Have we Solved all Hurdles for ATMPs?
Open accessAbstract
T cells are known as the most potent killer cells of the immune system, designed by nature to prevent unwanted challenges. The first class of therapeutic products harnessing the power of T cells for target-specific treatment of oncological diseases was bispecific antibodies. The first T-cell engaging bispecific antibodies that obtained approval were catumaxomab and blinatumomab 1 , 2 . Eight years later, the first chimeric antigen receptor (CAR)-T cells received regulatory approval 3 . CAR-T cells are the cellular interpretation of T-cell engaging therapies and have shown remarkable clinical results. CAR-T cells belong to the regulatory group of advanced therapy medicinal products (ATMPs). Due to the cell-/gene-based complex nature, ATMPs are far more challenging to develop than other, more defined, medicinal products. Despite very encouraging clinical results, there have been many set-backs in the development of ATMPs during the past 20 years. Therefore, the approval of the first two CAR-Ts KYMRIAH and YESCARTA is highly encouraging for the field. In this article we review the current landscape of CAR-Ts as a special class of ATMPs. This comprises the pathway to approval including the use of dedicated regulatory tools and challenges that were faced during the procedure. Furthermore, we highlight important future trends in the field.
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Kymriah is a lentiviral vector-based product in clinical trials for ex vivo gene therapy to treat acute lymphoblastic leukemia.
“Moreover, Kymriah is another lentiviral vector-based product in clinical trials for ex vivo gene therapy to treat acute lymphoblastic leukemia [53].”
From The Ocular Gene Delivery Landscape (2021) · cites this paper as [53]
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References 24 · Cited by 90
Research works printed but not linked: 1
- [11]Seimetz D. ATMPs: How to successfully master challenges and foster the regulatory success rate? Pharmazeutische Medizin. 2016; 18(3): 132– 39. https://static1.squarespace.com/static/57d16156414fb5307d454a1d/t/59b107ef03596ea2cfbaa206/1504774134784/ATMP_Seimetz.pdf (accessed 1 October 2018).
- Authors
- Seimetz, D., Heller, K., & Richter, J.
- Affiliations
- Biopharma Excellence GmbH, Munich, Germany
- Shire (Switzerland) (CH)
- Corresponding author
- Seimetz, Diane
- Venue
- Cell Medicine
- Publisher
- SAGE Publications
- Published
- 2019-01-01
- Volume
- 11
- Pages
- 2155179018822781-2155179018822781
- ISSN
- 2155-1790 (ISSN-L)
- PMC
- PMC6343443
- OpenAlex
- W2912531885
- Cited by
- 90
- References
- 24
- Citations by year
- 2026: 3 · 2025: 5 · 2024: 13 · 2023: 11 · 2022: 8 · 2021: 25 · 2020: 20 · 2019: 5
- FWCI
- 1.38
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Locations
- Cell Medicine (publishedVersion, cc-by-nc)
- Europe PMC (PubMed Central) (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Oncology › CAR-T cell therapy research
- Health Sciences › Medicine › Physiology › Biomedical Ethics and Regulation
- Life Sciences › Immunology and Microbiology › Immunology › Immunotherapy and Immune Responses
- Keywords
- chimeric antigen receptor T cells, advanced therapy medicinal products, regulatory approval, tisagenlecleucel, axicabtagene ciloleucel, bispecific antibodies, cell therapy, regulatory instruments, hematologic malignancies
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