December 21, 2018

FDA Issues Warning Letter Regarding Unapproved Stem Cell Products

Holland & Knight Healthcare Blog
Sara M. Klock | Michael J. Werner | Jennifer M. Nowak

The U.S. Food and Drug Administration (FDA) issued a statement on Dec. 20, 2018, once again putting stem cell companies on notice about its intention to crack down on the marketing of unapproved stem cell products, and reminding manufacturers about the parameters of its current compliance and enforcement policy for certain human cell, tissue, or cellular or tissue based products (HCT/Ps). This statement comes on the heels of a Nov. 29, 2018 Warning Letter to Genetech for marketing the stem cell products it manufactures without FDA approval and for significant current good tissue practices (CGTPs) and current good manufacturing practices (CGMPs) deviations.

The FDA inspected Genetech in June 2018 and found the "company was processing cellular products from human umbilical cord blood for administration by intra-articular (joint) injection, intravenous injection or application directly to the affected tissue to treat a variety of orthopedic conditions." Based on the intended use expressed in these claims, the FDA considers Genetech's products to be drugs/biologics without proper investigational or premarket approvals. Further, while the products qualify as HCT/Ps, they do not meet the regulatory criteria to be regulated solely under section 361 of the Public Health Safety Act and therefore, are not exempt from premarket review.

In addition to the products' unapproved status, the FDA found nineteen CGTPs and CGMPs violations including:

  • Failure to determine and document donor eligibility based on donor screening and testing;
  • Failure to test donor specimens using appropriate FDA-licensed, approved or cleared donor screening tests, in accordance with the manufacturer's instructions, to adequately and appropriately reduce the risk of transmission of relevant communicable disease agents or diseases;
  • Failure to establish and follow appropriate written procedures designed to prevent microbiological contamination of drug products purporting to be sterile; and
  • Failure to establish and follow a written testing program designed to assess the stability characteristics of its manufactured umbilical cord blood products and to use results of the stability testing to determine the appropriate storage conditions and expiration dates.

According to the FDA, Genetech's violations created a significant risk of contamination and/or other serious quality defects. In a report issued on Dec. 21, 2018, the Centers for Disease Control (CDC) confirmed the FDA's concern, finding twelve individuals in three states developed infections after receiving Genetech's stem cell products.

The FDA's action against Genetech reflects the agency's stated intention not to exercise enforcement discretion for products that pose a potential significant safety risk. In conjunction with its statement, the FDA will be sending this letter to stem cell product manufacturers, encouraging them to contact the FDA to discuss how their products are regulated well in advance of the November 2020 expiration of the agency's enforcement discretion. The letter also reminds manufacturers that the FDA's current enforcement discretion is not unlimited; rather, the FDA takes a risk-based approach focused on the risk associated with method of administration and intended use for treatment of certain diseases and conditions. In line with other recent strong statements issued by the agency, as discussed in a previous blog post, we expect to see the FDA continue to initiate regulatory enforcement action against stem cell companies that shirk their safety and quality responsibilities and put patients at risk.

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