In August, the U.S. Food and Drug Administration (FDA) issued a stern
to a California-based company for manufacturing and distributing unapproved
products derived from umbilical cord blood and for failure to comply with
current good tissue practice (CGTP) and current good manufacturing practice
(CGMP) requirements. Because these products were derived from human cells,
tissues, or cellular or tissue-based products (HCT/Ps), they are subject to
specific FDA regulations, which the company purportedly failed to follow.
Products such as these are part of a larger, growing market for
regenerative medicine products, which includes products involving HCT/Ps
and other genetic material used to treat and manage diseases. The global
regenerative medicine market is estimated to reach $48.97 billion by 2025.
With the rise in popularity of regenerative medicine products, this recent
warning letter serves as a reminder to the industry of the importance of
understanding FDA’s requirements in this area.
FDA issued the warning letter after an inspection resulted in a Form 483
noting significant deviations from CGMPs and CGTPs. Although the company
responded to these concerns, the FDA issued the warning letter noting the
company’s failure to have an investigational new drug (IND) application in
effect to study the products or an approved biologics license application
(BLA) to lawfully market such products. The warning letter noted that the
company described itself as "specializing in the harvesting and isolation
of stem cells to manufacture and commercialize innovative products," and
distributed the products directly to physicians throughout the United
Specifically, the FDA’s inspection revealed that the company’s umbilical
cord blood-derived products were being manufactured for use in recipients
unrelated to the donors of the products. Accordingly, because these
products were not intended for homologous use only (i.e., to perform the
same basic function or functions in the recipient as in the donor), FDA
considered these products as both drugs and biological products requiring
both an IND and a BLA.
The FDA’s still-evolving framework for regulating regenerative medicine
products currently includes
four significant guidance documents
that discuss FDA’s risk-based approach to regulating such products. Such an
approach includes considerations about how products are being administered
as well as what diseases and conditions they are intended to treat.
Regulatory Considerations for Human Cells, Tissues, and Cellular and
Tissue-Based Products: Minimal Manipulation and Homologous Use, issued in 2017, outlines the criteria for HCT/P products to avoid
regulation as a drug, device or biological product under the Food, Drug,
and Cosmetic Act (FDCA).
In a press release following the warning letter, the director of FDA’s
Center for Biologics Evaluation and Research
that “[t]he FDA remains highly committed to facilitating the development
and availability of safe and effective cellular therapy products” but
warned that the FDA “will not hesitate to take appropriate action to
protect people from being harmed by products with potential significant
safety concerns.” In addition to the warning letter highlighted here, the
FDA sent another 20 letters to manufacturers and providers that FDA
suspects are offering unapproved stem cell products.
Manufacturers of these products, and providers using such products, should
take careful note of FDA’s approach to regulating HCT/Ps and other
regenerative medicine products. For questions related to regenerative
medicine products, HCT/Ps or stem cell products specifically, please
or another member of the
McGuireWoods life sciences team.