Navigating the European regulatory framework for ATMPs: GTMPs, CTMPs and TEPs
With an emerging and rapidly growing segment of biological medicinal products and advanced therapy medicinal products (ATMPs), it is helpful to understand the three types of ATMPs that are based on genes, cells or tissues.
- Gene therapy medicinal products (GTMPs) contain genes that elicit a therapeutic prophylactic or diagnostic effect and work by inserting recombinant genes into cells, usually to treat a variety of genetic disorders, e.g., cancer or chronic diseases. These represent the majority of ATMPs approved in the European Union (EU).
- Somatic cell therapy medicinal products (CTMPs) contain cells or tissues that have been manipulated to change their biological characteristics and can be used to cure, diagnose or prevent disease, e.g., the use of a patient’s manipulated immune cells (e.g., chimeric antigen receptor T-cells [CAR-T]) to fight the remaining cancer cells in their body. Historically, three CTMPs have been approved in the EU, but two (Provenge® and Zalmoxis) have had their marketing authorization (MA) withdrawn.
- Tissue-engineered products (TEPs) contain cells or tissues that have been modified so they can be used to repair, regenerate or replace tissue, e.g., artificial skin used to treat patients with burns. Four TEPs have been approved in the EU, but only two, Holoclar® and Spherox®, have retained their MA.
If an ATMP contains one or more medical devices as an integral part of the product, it could be considered a “combined ATMP” or “CATMP,” which is a product classification regulated under the ATMP Regulation (EC) No. 1394/2007 (unless the cell/tissue component is non-viable, and its action is ancillary to that of the device components. ATMPs offer promising therapies for the treatment of diseases with unmet medical needs.
To support sponsors developing these state-of-the-art products, Sanjay Jain, PhD, PGMP, Senior Director, Global Regulatory Affairs at Fortrea, provided an overview of the European Medicines Agency regulatory requirements, pathways and process options for CATMPs. Find out how the EU is trying to create an open market for CATMPs by adopting the ATMP regulations.
Reference
- European Medicines Agency. Reflection paper on classification of advanced therapy medicinal products, EMA/CAT/600280/2010 Rev.1, 21 May 2015. https://www.ema.europa.eu/en/documents/scientific-guideline/reflection-paper-classification-advanced-therapy-medicinal-products_en.pdf-0 Accessed July 8, 2024
