The realm of medicine has now advanced to include Cell and gene therapies (CGT), which hold tremendous potential in extending life expectancy and occasionally providing remedies for illnesses that were previously considered untreatable. However, these groundbreaking therapies come at a high cost and often require unconventional methods of approval.
It is expected that the FDA and EMA have varying terminologies for CGT. Although the process for preparing a marketing application that meets approval requirements is consistent, it is crucial for sponsors to have knowledge of the appropriate language and terminology.
To illustrate, EMA substitutes the term CGT with advanced therapy medicinal products (ATMP). Additionally, EMA classifies ATMPs into gene therapy medicinal product (GTMP), somatic cell therapy medicinal product (SCTMP), tissue-engineered therapies (TET), and combined advanced therapies.
There have been a total of 27 CGTs approved by the FDA, including 14 cell therapies and 13 gene therapies. The EMA has approved 24 CGTs, including 16 GTMPs, 4 SCTMPs, and 4 TETs. 13 products have been approved by both regions, including CAR-T cell therapies for blood cancers. The FDA has approved eight cord blood products to treat hematopoietic system disorders, whereas the EMA has not approved any, but two have received orphan designation. Holoclar is approved as a stem cell therapy for corneal tissue in the EEA, but only received orphan designation in the US. No US-approved products have been withdrawn from the market, while seven out of 24 EEA products have been withdrawn after receiving approval.
For more information on the challenges of demonstrating the safety and efficacy of clinical trials, as well as establishing manufacturing processes and analytical methods that meet regulatory requirements in both continents, refer to the original article in Cell and Gene.com.