FDA Drafts Guidance Document for Materials Used in the Manufacture of CGTs and Tissue-Engineered Therapeutics

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FDA has made available a new draft guidance entitled Considerations for the Use of Human- and Animal-Derived Materials and Components in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products for public comment (1). The draft document was issued by FDA’s Center for Biologics Evaluation and Research in April 2024.

In the draft guidance, manufacturers of cellular and gene therapy (CGT) and tissue-engineered medical products (TEMPs) are provided recommendations for assuring the safety, quality, and identity of materials—both human and animal origin—that are used to manufacture CGTs and TEMPs. Recommendations are given in regard to the chemistry, manufacturing, and control (CMC) information related to the use of human- and animal-derived materials. This type of information is submitted for an investigational new drug application.

Materials covered by this draft guidance include the reagents, feeder cells, excipients, and other inactive ingredients in the drug product that are in direct contact with the starting material, intermediates, and final products as well as any materials used to manufacture reagents, feeder cells, and excipients, and materials incorporated in TEMPs, according to FDA (1). Materials excluded from the guidance include human cells used as starting material to manufacture human cells, tissues, and cellular and tissue-based products, including TEMPs.

The agency states in the document that “Use of human- and animal-derived materials during product manufacturing may increase risks of infectious disease transmission, and raises potential safety concerns, such as the possible introduction of adventitious agents or other impurities into CGT products and TEMPs. Thus, human- and animal-derived materials should be thoroughly characterized and described in your regulatory submission” (1).

Comments can be submitted electronically or in writing by the deadline date on July 29, 2024, after which time, the agency will begin working on a final version of the guidance (2).

According to the agency, this guidance is intended to supplement the two final industry guidance documents, Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (3) 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) (4).

References

1. FDA. Draft Guidance for Industry, Considerations for the Use of Human-and Animal-Derived Materials in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products (CBER, April 2024).
2. FDA. Considerations for the Use of Human-and Animal-Derived Materials and Components in the Manufacture of Cell and Gene Therapy and Tissue-Engineered Medical Products; Draft Guidance for Industry; Availability. Notice, Federal Register, 89 FR 34253, 34253-34254.
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).

Source: FDA