FDA Issues New Draft Guidance on Allogeneic Cell-Based Medical Products

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FDA has announced a new draft guidance entitled Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products. The document was issued in April 2024 by the agency’s Center for Biologics Evaluation and Research (CBER) (1).

According to FDA in an April 30, 2024 notice in the Federal Register (2), “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.” Thus, the draft guidance provides recommendations to help sponsors of allogeneic cell-based medical products determine what cell safety testing is appropriate to support an investigational new drug application (IND) or a biologics license application (BLA).

The agency notes that cell safety testing should be based on a risk analysis that considers the expansion potential of the cells in addition to the reagents that are used to expand these cells in culture as well as the number of individuals capable of treatment with the cell-based medical product.

Allogeneic cell-based products that are regulated by CBER’s Office of Therapeutic Products are subject to this new guidance. It applies to cultured allogeneic cells—including cell banks—that serve as sources of intended constituents for the final drug product. The guidance also applies to combination products that contain an allogeneic cell or cell-based biologic component in combination with a drug and/or device.

“The recommendations in this guidance also apply to genetically modified allogeneic cells that have been transduced with viral and/or plasmid vectors, and cells that have undergone genome editing. This guidance does not apply to cell substrates that are used during manufacturing of non-cell-based products such as viruses, gene therapy vectors, or recombinant proteins,” the draft guidance document states (1). However, the document emphasizes that recommendations for feeder or other cells used as reagents during manufacturing are beyond the scope of this particular guidance.

FDA is accepting comments on the draft guidance either electronically or in writing until the deadline of July 29, 2024, after which time it will begin work on the final version of the guidance (2).

FDA has stated that this new draft guidance is intended to supplement two other final industry guidance documents, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (3) and Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs) (4).

References

1. FDA. Draft Guidance for Industry, Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products (CBER, April 2024).
2. FDA. Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products; Draft Guidance for Industry; Availability. Notice, Federal Register, 89 FR 34251, 34251–34253.
3. FDA. Guidance for Industry, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (CBER, January 2020).
4. FDA. 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) (CBER, April 2008).