Introduction:
Advanced Therapy Medicinal Products (ATMPs) represent innovative treatments for various diseases and injuries based on cells, genes or tissues. Despite their potential, Europe lags behind in the early development of ATMPs. The EU-funded project JOIN4ATMP aims to identify challenges and possible solutions to drive the European ATMP development. One key objective is to map the European trial landscape by creating a database of preclinical and clinical ATMP trials.
Methods:
To develop a database of clinical ATMP trials conducted in Europe data from the European Union Clinical Trial Register (EU CTR) was primarily used. All trials that self-classified as ATMPs were extracted by an automated method. These were further categorized by phase (trials on EMA-approved ATMPs, Phase I/II, Phase II) and mode of action (gene therapy, tissued-engineered medicine, somatic cell therapy, combinational ATMP, others). Additional online research aimed to identify ATMPs preclinically developed in Europe. Furthermore, the target disease and disease category were analyzed. The initial focus was on early-phase gene therapy trials. However, the same analysis on the other categories will follow.
Results:
Of 43,989 registered clinical trials in the EU CTR (as of 08/2024), 727 early ATMP-related studies were identified in Europe, including 49 involving EMA-approved ATMPs. Phase I and I/II studies (n=342) were categorized by mode of action: gene therapies were most common (n=169), followed by somatic cell therapies (n=107), tissue-engineered products (n=30) and combinational ATMPs (n=7). 29 studies could not be clearly categorized via EU CTR entries and required manual classification. Of the gene therapies, 110 had preclinical development in Europe and were further analyzed by target disease category: common/rare cancers and common/rare disease (Fig. 1). The classification by mode of action of Phase II trials (n=336) are shown in Fig. 2.
Conclusion:
Although ATMP-related studies are increasing in Europe, a comprehensive overview of the landscape is still lacking. Many ATMPs developed outside Europe reach European patients only in later stages (≥Phase II), while ATMPs developed and entering Phase I in Europe rarely reach market authorization. The JOIN4ATMP subproject aims to build a comprehensive database to analyse these studies regarding possible barriers and share successful strategies for preclinical development.
References:
Acknowledgement:
Funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Health and Digital Executive Agency (HADEA). Neither the European Union nor the granting authority can be held responsible for them.