Stem cell therapy may violate the good manufacturing and labeling provisions of Federal Food, Drug and Cosmetic Act ("FDCA"), if the biological characteristics of the cells employed are altered prior to administration to a subject. That seems to be the red line drawn by Circuit Court of Appeals for the District of Columbia in the case U.S. v.  Regenerative Sciences, LLC decided earlier this month.

There is no doubt that FDA has ramped up its enforcement activities in recent years. Continued with this theme, physician practice groups that offer certain types of cell therapy can be the new target for the FDA scrutiny.   In  its letter  to Regenerative Sciences,  FDA cited the medical clinic for utilizing mesenchymal stem cells in an advertised Regenexx" procedure for treatment of certain orthopedics conditions.  As such FDA highlighted that the cell cocktail used in the Regenexx procedure is neither under an active investigational new drug application, nor an approved biologics license application. FDA thus declared the use of such stem cells cocktails as violating the Public Health Service Act (PHSA) and the FDCA.

The District Court and the Court of Appeals agreed with the FDA. They reasoned that the cell cocktail used in Regnexx procedure was indeed an article "intended to treat orthopedic diseases and to affect musculoskeletal function," and thus necessary to comply with good manufacturing and labeling provisions of the FDCA and PHSA.  The Court of Appeals rejected Appellants' arguments that the cocktail was exempt from the FDA jurisdiction, even though it used the patient's own stem cells. The Court did not categorize the cocktail as a product falling under compounding pharmacy practice, nor did it characterize the cocktail as an exempted "minimally manipulated" biological product.  The Court rejected Appellants interpretation  that doctors who manufacture and administer drugs in the course of their professional practice are considered exempt from FDCA requirments, as the process  is a State regulated practice of medicine. Denying Appellant's construction of the FDCA, the Court of Appeals reasoned that "while the FDCA was not intended to regulate the practice of medicine, it was obviously intended to control the availability of drugs for prescribing by physicians."

Cell therapy procedures are under the FDA's radar and the cell product used in such procedures may require premarket approval. Early assessment for a regulatory strategy is advisable to minimize the risk of violating FDCA and PHSA.

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