The U.S. Court of Appeals for the Ninth Circuit has affirmed that certain stem cell products are subject to premarket approval by the Food and Drug Administration (FDA) under the Federal Food, Drug, and Cosmetic Act (FDCA). The ruling, issued on September 27, 2024, in USA v. California Stem Cell Treatment Center, Inc., supports the FDA's stance that stem cell products intended to affect the structure or function of the body, or to treat diseases, are regulated as drugs.
This decision reverses a lower court ruling and aligns the Ninth Circuit with the DC and 11th Circuits, reinforcing the FDA's oversight of stem cell therapies. The case has been closely watched by the regenerative medicine industry, as it clarifies the regulatory pathway for stem cell products and could have significant implications for companies developing these treatments.
Background on HCT/P Regulation
Human cells, tissues, and cellular- and tissue-based products (HCT/Ps) intended for implantation, transplantation, infusion, or transfer into a human recipient are regulated as drugs under the FDCA if they are intended to affect the structure or any function of the body, or to diagnose, cure, mitigate, prevent, or treat a disease. Such drugs typically require an approved New Drug Application (NDA) or Biologics License Application (BLA) before they can be legally marketed.
An exception exists for HCT/Ps regulated solely under section 361 of the Public Health Service Act, provided they meet specific criteria, including being minimally manipulated, intended for homologous use, and not combined with other cells or tissues (with limited exceptions). Another exception is the same surgical procedure (SSP) exception, which applies when HCT/Ps are removed from and implanted back into the same individual during the same surgical procedure.
Details of the Case
The case originated in 2017 when the FDA inspected the California Stem Cell Treatment Center and the Cell Surgical Network Corporation, concluding that their stem cell products were unapproved drugs manufactured in violation of the FDCA. The FDA subsequently sought a permanent injunction to prevent the marketing of these unapproved drugs.
The District Court initially ruled that the defendants were engaged in the practice of medicine, not the manufacture of pharmaceuticals, and that their use of stromal vascular fraction (SVF) fell within the SSP exception. However, the Ninth Circuit reversed this decision, asserting that the stem cell product was indeed a drug under the FDCA and that the SSP exception did not apply.
Key Points of the Ninth Circuit's Decision
The Ninth Circuit emphasized the broad definition of "drugs" under the FDCA, based on their intended use. The court noted the defendants' marketing intent to treat a range of diseases and affect body structures, such as regenerating cartilage. This aligned with the DC Circuit's interpretation in Regenerative Sciences.
The court dismissed the argument that classifying stem cell mixtures as drugs would interfere with the practice of medicine, citing a previous Ninth Circuit case, United States v. Kaplan. The court also rejected the defendants' invocation of the major questions doctrine, finding that the FDA's regulation of human cell and tissue products was not a sudden or transformative expansion of authority.
Crucially, the Ninth Circuit determined that the SSP exception did not apply because the "same" HCT/Ps were not removed and implanted back into the patient. The court highlighted that the stem cell procedure involved significant processing, including enzymatic digestion and centrifugation, which introduced a level of risk that the SSP exception was not intended to accommodate.
Implications for the Stem Cell Industry
With three circuit courts now in agreement on the FDA's authority over certain stem cell procedures, this decision has significant implications for companies developing or offering stem cell treatments, particularly those involving SVF. These entities must ensure compliance with the FDCA and its regulations for premarket drug approval, including demonstrating the safety and efficacy of their drugs for specific intended uses. Failure to comply could result in enforcement actions by the FDA and the Department of Justice.
