Exploring Regulatory Frameworks for Exosome Therapy: Insights and Perspectives.

Area of Science:

• Biotechnology
• Drug Development
• Regulatory Science

Background:

• Extracellular vesicles (EVs) are increasingly recognized for their diagnostic and therapeutic potential in clinical settings.
• The clinical translation of EVs is currently impeded by significant technological and regulatory complexities.
• Addressing these challenges is vital for realizing the full potential of EV-based therapies.

Purpose of the Study:

• To review critical considerations in the manufacturing, quality management, and nonclinical evaluation of extracellular vesicles for drug development.
• To compare international technical regulatory requirements for EV products and clarify governing principles.
• To provide insights into the ongoing international debate on EV regulatory review.

Main Methods:

• Literature review and synthesis of key considerations in EV manufacturing and quality control.
• Comparative analysis of regulatory requirements for EV products across major global regulatory agencies.
• Analysis of current international discussions and proposed frameworks for EV regulation.

Main Results:

• Manufacturing, quality management, and nonclinical evaluation present key challenges for EV drug development.
• Significant international variation and debate exist regarding the regulatory review pathways for EVs.
• A risk-based classification framework, treating EVs as advanced therapeutic drugs, is proposed as a rational approach.

Conclusions:

• Standardized production protocols and a deeper understanding of therapeutic mechanisms are essential for EV advancement.
• Resolving complex regulatory issues is paramount for the successful clinical translation of EV-based therapies.
• A harmonized, risk-based regulatory approach is needed to facilitate the development and approval of EV products.