New FDA Draft Guideline regarding Safety Testing of human allogeneic Cells

The draft guidance on "Safety Testing of Human Allogeneic Cells Expanded for Use in Cell-Based Medical Products" is designed to help sponsors of allogeneic cell-based medical products develop a cell safety testing strategy for investigational new drug (IND) applications or biologics license applications (BLAs). It specifically addresses cultured allogeneic cells, including cell banks and combination products, but excludes cell substrates for non-cell-based products like viruses or gene therapy vectors.

The guidance highlights the inherent risks of contamination in cell-based products, especially those extensively cultured during manufacturing. This can include contamination from the original cell source or during the production process, and potential genomic changes leading to tumorigenic cells.

Sponsors are advised to outline their strategies for assessing the risk of contamination with adventitious agents in line with the electronic Common Technical Document (eCTD) section 3.2.A.2. They should also include results of donor screening and testing in their submissions. For INDs they are asked to list all manufacturing materials and their quality grades to help the FDA assess the adequacy of the proposed safety tests.
Specific tests recommended include bacterial and fungal sterility testing according to the US Pharmacopeia's (USP) <71>, and mycoplasma testing as specified in USP<63>, though alternative methods may be used if they are comparably sensitive.

The FDA may impose a clinical hold on INDs lacking sufficient Chemistry, Manufacturing, and Control (CMC) information to evaluate safety risks.

This guidance complements existing FDA guidances

  • from January 2020 - Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs)
  • from April 2008 - 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) related to CMC information for human gene therapy and somatic cell therapy INDs.

The deadline to comment on the guidance is 29th July.

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