Pharmacoepidemiol Risk Manage.  2024 Mar;16(1):11-17. 10.56142/perm.24.0007.

Gene Therapy Products in Advanced Therapy Medicinal Products: Comparative Mechanism and Safety Study by Product

• Park J
• Park D
• Kim S
• Jung SY
• Shin JY

• KMID: 2556388
• DOI: http://doi.org/10.56142/perm.24.0007

Abstract

Globally, advanced therapy medicinal products (ATMP) such as stem cell therapies and gene therapies have become the focal point of the pharmaceutical market. In response to this trend, Korea, like the United States and Europe, has established various regulations and systems pertaining to ATMP. The Ministry of Food and Drug Safety enacted the ‘Act on the Safety and Support of Advanced Regenerative Medicine and Advanced Therapy Medicinal Products,’ restructuring the existing approval system from one centered around synthetic drugs and conventional biopharmaceuticals to one tailored to the characteristics of ATMP. While this regulatory framework allows for consideration of the unique attributes of ATMP, standardized safety measures specific to each product category are currently lacking. To address this gap, we provided foundational data for the safety and effective management of ATMP. This was accomplished by comparing and analyzing the mechanisms of gene therapy products, which are central to ATMP. Additionally, we conducted an analysis of indications and potential adverse events. Based on our research, we anticipate that our study will serve as foundational data for the safety and effective management of gene therapy.

Keyword

Long term follow-up; Advanced therapy medicinal products; Gene therapy