Cell, tissue and gene products with marketing authorization in 2018 worldwide

Abstract

Cell and gene therapies (CGTs) are progressively entering into clinical practice in different parts of the world. The International Society for Cell & Gene Therapy (ISCT), a global scientific society, has been committed since 1992 to supporting and developing knowledge on clinical applications of CGTs. Considering the number of products that have been progressively approved and, in some cases, withdrawn in recent years, the ISCT would like to present a brief annual report on CGTs with marketing authorization (MA) in different regions. This article reflects the dynamic momentum around authorized CGTs coinciding with the parallel increase of unproven approaches where cells are delivered without appropriate and rigorous scientific and regulatory assessment and authorization. This is intended to be a living document with a yearly update linked to a dedicated section of the ISCT website for faster adjustments. The aim is to ultimately inform, by periodic snapshots, the scientific community, healthcare stakeholders and patient associations on authorized CGT products as a way to increase communication around the approved therapeutic approaches charged with heightened expectations.

Challenges in Clinical Development of Mesenchymal Stromal/Stem Cells: Concise Review

“…In January 2019, a search on the public website http://clinicaltrial.gov, using as keywords “MSC,” “mesenchymal cells,” “stromal cells,” resulted in more than 900 registered studies worldwide. A more careful look revealed that most of these MSC‐based trials are still in early phases I or II, while only a small portion of them (less than 50) are in phase III , and currently, only nine products (approximately 1%) have been approved worldwide for market authorization . This should be seen as unexpected for a 50 year‐old cell type, particularly considering that both local and intravascular MSC infusions have been considered relatively safe procedures .…”

“…Despite the development and the interest in MSC‐based therapy and its clinical potentials, few human MSC‐based products have been authorized for market and used in clinics worldwide . Many challenges remain, including the need for more harmonized regulatory frames among countries to facilitate from early developments to large multinational trials.…”

Shifting Paradigms Revisited: Biotechnology and the Pharmaceutical Sciences

“…The approval of the first gene therapy product (Gendicine) in China in 2003 represents an important turning point in the area of these innovative biologics. Currently, there are more than 15 ATMPs approved in the USA and Europe and the list is growing ( Table 2 and Cuende et al 24 ).…”

The Regenerative Horizon: Opportunities for Nursing Research and Practice

“…Regenerative technologies are emerging on a global scale aimed at achieving therapeutic repair in the context of worldwide aging and the pandemic of chronic degenerative diseases (Cuende, Rasko, Koh, Dominici, & Ikonomou, 2018 ;Zacchigna & Giacca, 2018 ). In the United States, the 21st Century Cures Act was designed to help accelerate the next generation of medical innovations to address patient needs (U.S. Congress, 2016 ).…”