The FDA has announced a new draft guidance entitled Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products. Issued in April 2024 by the agency’s Center for Biologics Evaluation and Research (CBER), this document provides recommendations for sponsors of allogeneic cell-based medical products to determine appropriate cell safety testing to support an investigational new drug application (IND) or a biologics license application (BLA).
According to the FDA, 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 such as cell-derived particles. The draft guidance emphasizes that cell safety testing should be based on a risk analysis considering the expansion potential of the cells, the reagents used for expansion, and the number of individuals capable of treatment with the cell-based medical product.
This new guidance applies to cultured allogeneic cells, including cell banks, that serve as sources of intended constituents for the final drug product, and to combination products containing an allogeneic cell or cell-based biologic component in combination with a drug and/or device. It also covers genetically modified allogeneic cells transduced with viral and/or plasmid vectors and cells that have undergone genome editing. However, it does not apply to cell substrates used during the manufacturing of non-cell-based products such as viruses, gene therapy vectors, or recombinant proteins.
The FDA is accepting comments on the draft guidance either electronically or in writing until July 29, 2024, after which it will begin work on the final version of the guidance. This draft guidance is intended to supplement two other final industry guidance documents related to Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) and Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs).