REGULATORY UPDATE: FDA Issues Draft Guidances Related to Cell and Gene Therapy

FDA recently released two draft guidances, “Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products” and “Considerations for the Use of Human- and Animal-Derived Materials in the Manufacture of Cellular and Gene Therapy and Tissue-Engineered Medical Products” intended to advance safety for biotherapy products.

The first draft guidance is intended to assist sponsors in designing appropriate cell safety testing as part of their regulatory submission for an investigational new drug application or blood license application to ensure the quality and safety of these innovative therapies. Allogeneic cells of human origin may be expanded in culture to manufacture medical products consisting of live cells, inactivated cells, cell lysates or other cell-based materials, including cell-derived particles. The guidance emphasizes the importance of rigorous cell safety testing that accounts for the expansion potential of the cells, the reagents that are used to expand the cells and the number of individuals the cell-based medical product may be capable of treating.

The second draft guidance includes recommendations to take into account when developing cellular and gene therapy (CGT) products and tissue-engineered medical products (TEMPs) that are manufactured using human- and animal-derived materials. These considerations include donor screening and testing, adventitious agent testing and screening, risk assessment, and materials management. The guidance also includes points to consider for manufacturers of human- and animal-derived materials used in the manufacture of CGT products or TEMPs.

Both draft guidance documents supplement two final guidances, “Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),” dated January 2020; and “Guidance for FDA Reviewers and Sponsors:  Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs),” dated April 2008. 

Both newly released draft guidances include a 90-day comment period. AABB will be reviewing the guidances to determine the need for comments. Members with questions may contact regulatory@aabb.org.