FDA Guidance Outlines CMC Requirements for Cell-Based Cardiovascular Therapies

April 10, 2009

The US Food and Drug Administration has released a new draft guidance on somatic cell therapies that are designed to advance the treatment of heart disease through the regeneration of cardiac tissues.

The US Food and Drug Administration has released a new draft guidance on somatic cell therapies that are designed to advance the treatment of heart disease through the regeneration of cardiac tissues. The document includes information on preclinical testing, evaluating delivery systems, clinical trial design, and chemistry, manufacturing, and controls (CMC) information required.

As for other types of cellular products, cell therapies for cardiac disease should include detailed descriptions of and data about the cell source, collection methods, processing methods, ancillary materials, formulation, testing methods, product release criteria, storage and shipping conditions, stability, segregation procedures to prevent cross contamination, and container labeling (in process and final product).

The guidance notes that if antibodies are used during processing to select cell populations enriched for a specific cell surface antigen, those antibodies should be demonstrated to be free of adventitious agents and of appropriate quality to function as intended.

The document also says that sufficient testing must be carried out to ensure the product is biocompatible with the delivery system. Such testing should evaluate cell viability and activity, cell adhesion to the device, and device integrity. “Because biologics can aggregate and adhere more easily at low flow rates, the worst-case delivery rate for this test may differ from that used in the other preclinical tests,” the guidance warns, adding that each new delivery system–product combination must be tested, because of the extremely variable sensitivities that cell therapies can have to physical and chemical stimuli.

The guidance document is available at www.fda.gov/OHRMS/DOCKETS/98fr/FDA-2009-D-0132-gdl.pdf