Abstract
Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while in EU, there are four major groups, i.e., gene therapy, somatic cell therapy, tissue-engineered therapies, and combined advanced therapies; in US, the sub-classification covers two major groups of products, i.e., gene therapy and cellular therapy. The inclusion criteria that define a gene therapy are equivalent in both regions, and the exclusion criteria are directly related to the indications of the product. In the EU, there is a clear differentiation between cell- and tissue-based products regarding their classification as advanced therapies or coverage by other legal frameworks, whereas in US, there is a broader classification about whether or not these products can be categorized as biologic products. Both in EU and in US, in order to classify a cell- or a tissue-based product as an advanced therapy, it must be ensured that the processing of the cells implies a manipulation that alters their biological characteristics, although the term of manipulation in US differentiates between structural and non-structural cells and tissues. The regulatory terminology used to define ATMPs and their sub-classification reveals some differences between EU and US.
Highlights
Advanced therapy medicinal products (ATMPs) comprise a category of innovative and complex biological products, which in most cases require extensive and complicated preclinical and clinical developments
A modified vaccinia virus ankara (MVA) into which two genes have been placed for the treatment of non-small cell lung cancer is classified as a gene therapy medicinal products (GTMP), but if these genes lead to foreign protein expression for the treatment of human immunodeficiency virus (HIV) disease, the product will not be considered an advanced therapy, but a vaccine (European Medicines Agency, 2016b; Draper and Heeney, 2010)
Our analysis reveals a difference between European Union (EU) and United States (US) in the sub-categorization of advanced therapies and the regulatory terminology defining them
Summary
Advanced therapy medicinal products (ATMPs) comprise a category of innovative and complex biological products, which in most cases require extensive and complicated preclinical and clinical developments. This complexity has been observed since the idea of transferring genetic material to cure a genetic disease was foreseen decades ago. The first ATMP product approved in the European Union (EU) came in 2009 with the authorization of ChondroCelect®, a tissue-engineered product indicated for the treatment of cartilage defects (European Medicines Agency, 2017a). In United States (US), the first approved ATMP came out 1 year later with PROVENGE®, a somatic cell therapy for the treatment of some prostate cancers (U.S Food and Drug Administration, 2019a). The first authorized gene therapy was launched in 2012, when Glybera® achieved marketing authorization in EU (European Medicines Agency, 2012)
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