Current Status of Nucleic Acid Drugs and the Challenges in Regulatory Science Relating to Quality Control

Abstract

Nucleic acid drugs have recently attracted considerable attention as a novel category of pharmaceutical agents following the development of small molecular therapeutics and antibody-based drugs. While to date no more than five nucleic acid drugs have been approved for use around the world, the number of clinical trials of nucleic acid drugs being carried out currently stands at between 100 and 200, and this vigor is unlikely to abate in the near future. However, there are currently no guidelines that specifically address nucleic acid drugs. Consequently, examining the regulation of product quality, non-clinical safety, and clinical features is important for the promotion of nucleic acid drug research and development. This paper outlines the current status of research involving nucleic acid drugs and discusses a number of points related to the challenges in regulatory science pertaining to this drug class from the particular standpoint of quality control, including issues associated with synthesis, purification, analysis, chemical modification, and the management of higher-order structures.