4. Special cases in marketing authorisations: Advanced therapy medicines (ATMPs)


1. Advanced therapy medicines (ATMPs)

Advances in science have yielded a new class of innovative and complex biological medicinal products based on manipulation[1] of genes, somatic cells or tissue engineering, so-called advanced therapy medicinal products (ATMP). They are very different from medicines based on chemical entities or of biological / biotechnological origin due to their complexity.  While these  novel health technologies  have the potential to address unmet medical needs in the treatment of disease as well as the repair of tissue or organ defects, they also involve specific risks (e.g., immunogenicity, viral safety, tumorigenicity etc.) that need to be accounted for in an appropriate regulatory framework.

Moreover, the regulatory context for cell or gene-based therapies was heterogeneous across EU Member States until the early 2000s and resulted in divergent national procedures for classification and authorisation. In order to harmonise, and as per the EC “to ensure the free movement of ATMPs within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field while guaranteeing the health protection for patients”, the European Council adopted a lex specialis[2] in the form of the regulation on advanced therapy medicinal products (Regulation (EC) No 1394/2007), (in force 30 December 2008) to be read in conjunction with Directive 2001/83/EC and Regulation (EC) No 726/2004 as amended.

The ‘ATMP Regulation’ lays down specific rules for the authorisation, supervision and pharmacovigilance of ATMPs and establishes the Committee for Advanced Therapies (CAT) at the EMA.

[1] More than “minimally manipulated”: For structural tissue: processing that alters the original relevant characteristics of the tissue relating to the tissue’s utility for reconstruction, repair, or replacement. For cells or non-structural tissues: processing that alters the relevant biological characteristics of cells or tissues.

[2] The Latin `lex specialis' notion comes from the legal maxim `lex specialis derogate legi generali'. A `lex specialis' is a `law' which governs a specific subject matter. The legal maxim means that a law governing a specific subject matter overrides a law that only governs general matters. For our subject, Regulation (EC) No. 1394/2007 overrides the general EU pharmaceutical legislation, which applies unless otherwise specified in the ATMP regulation.