Abstract
Advanced therapy medicinal products (ATMP) in the European Union (EU) are regulated by Regulation 1394/2007 and comprise gene and cell therapy and tissue-engineered products. Under this framework, ATMP are authorised by the centralised procedure, coordinated by the European Medicines Agency (EMA), whereas clinical trial authorisations remain at the remit of each National Competent Authority. The Committee for Advanced Therapies is responsible for the scientific evaluation of the marketing authorisation applications and for generating a draft opinion that goes to the Committee for Human Medicinal Products for a final opinion. For every application, data and information relating to manufacturing processes and quality control of the active substance and final product have to be submitted for assessment together with data from non-clinical and clinical safety and efficacy studies. Technical requirements for ATMP are defined in the legislation, and guidance for different products is available through several EMA/CAT guidelines.
Due to the diverse and complex nature of ATMP, a need for some regulatory flexibility was recognised. Thus, a risk-based approach was introduced in Regulation 1394/2007 allowing adapted regulatory requirements. This has led, for instance, to the development of good manufacturing practice (GMP) guidelines specific for ATMP. This, together with enhanced regulatory support, has allowed an increasing number of successful marketing authorisation applications resulting in 25 licensed ATMP in the EU, mainly gene therapy medicinal products. The promise of messenger RNA and genome editing technologies as therapeutic tools make the future for these innovative medicinal products look even brighter.
This chapter reviews the regulatory landscape together with some of the support initiatives developed for ATMP in the EU.
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Abbreviations
- AAV:
-
Adeno-associated Virus
- ADME:
-
Absorption, Distribution, Metabolism and Excretion
- ATIMP:
-
Advanced Therapy Investigational Medicinal Product
- ATMP:
-
Advanced Therapy Medicinal Product
- CAR:
-
Chimeric Antigen Receptor
- CAT:
-
Committee for Advanced Therapies
- CBMP:
-
Cell-based Medicinal Product
- CE:
-
Conformité Européenne
- CHMP:
-
Committee for Human Medicinal Products
- CRISPR:
-
Clustered Regularly Interspaced Short Palindromic Repeats
- CRS:
-
Cytokine Release Syndrome
- EC:
-
European Commission
- EDQM:
-
European Directorate for the Quality of Medicines
- EMA:
-
European Medicines Agency
- ERA:
-
Environmental Risk Assessment
- EU:
-
European Union
- FDA:
-
Food and Drug Administration
- FIH:
-
First in Human
- GCP:
-
Good Clinical Practice
- GLP:
-
Good Laboratory Practice
- GMO:
-
Genetically Modified Organism
- GMP:
-
Good Manufacturing Practice
- GTIMP:
-
Gene Therapy Investigational Medicinal Product
- GTMP:
-
Gene Therapy Medicinal Product
- ICANS:
-
Immune Effector Cell-Associated Neurotoxicity Syndrome
- ICH:
-
International Council for Harmonisation
- iPSC:
-
Induced Pluripotent Stem Cell
- IVDR:
-
In Vitro Diagnostic Regulation
- MAA:
-
Marketing Authorisation Application
- MD:
-
Medical Device
- MDR:
-
Medical Devices Regulation
- MSC:
-
Mesenchymal Stromal Cell
- PD:
-
Pharmacodynamic
- PRIME:
-
Priority Medicines
- RCV:
-
Replication Competent Virus
- RNA:
-
Ribonucleic Acid
- SME:
-
Small- and Medium-sized Enterprises
- SmPC:
-
Summary of Product Characteristics
- SoHO:
-
Substances of human origin
- TALEN:
-
Transcription Activator-like Effector Nuclease
- TEP:
-
Tissue-engineered Product
- TIL:
-
Tumour-Infiltrating Lymphocyte
- ZFN:
-
Zinc Finger Nuclease
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Schuessler-Lenz, M. et al. (2023). Marketing Regulatory Oversight of Advanced Therapy Medicinal Products in Europe. In: Galli, M.C. (eds) Regulatory Aspects of Gene Therapy and Cell Therapy Products. Advances in Experimental Medicine and Biology, vol 1430. Springer, Cham. https://doi.org/10.1007/978-3-031-34567-8_1
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