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FDA Warns American CryoStem Over Unapproved Stem-Cell Product
The FDA has issued a warning letter to American CryoStem Corporation for marketing an adipose-derived stem cell product without the agency’s approval and for significant deviations from current good manufacturing practice requirements, including some that raise potentially significant safety concerns.
The FDA recently inspected American CryoStem and found that the company was receiving and processing adipose tissue into a product called Atcell and marketing it without the required FDA approval. Though the product is intended for autologous use (in the individual from whom it was recovered), the manner in which it is being processed involves more than minimal manipulation of the adipose tissue, the FDA says. These manufacturing steps alter the original relevant characteristics of the tissue and have the potential to introduce contamination of the product, creating risks of harm to patients. As a result, under existing law, the product is required to undergo FDA review to ensure the treatment is safe and effective, the agency says.
Under the FDA’s risk-based enforcement strategy, the product also creates concerns as a result of the way it is being administered into patients’ bodies and how it is promoted for the treatment of serious or life-threatening diseases or conditions. Atcell is being distributed directly to physicians to treat patients for a variety of serious or life-threatening diseases or conditions, including anoxic brain injury, Parkinson’s disease, amyotrophic lateral sclerosis, stroke, and multiple sclerosis. Physicians are directed by the company to administer Atcell by various routes of administration, including intravenously, intrathecally, and by aerosol inhalation.
Compounding these risks, the FDA’s inspection also uncovered evidence of significant deviations from current good manufacturing practice requirements in the manufacture of Atcell that pose significant additional risks, such as potentially being contaminated with microorganisms or having other serious product quality defects. Specific deviations included unvalidated processes, an inadequately controlled environment, lack of control of components used in production, and a lack of sufficient and validated product testing.
“The use of Atcell raises potential significant safety concerns, due in part to the fact that there is little basis on which to predict how the product will perform in a patient,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research. “In addition, this product may also cause harm to patients who may put their trust in an unproven therapy and make the decision to delay or discontinue medical treatments proven to be safe and effective.”
American CryoStem was issued a list of inspectional observations. The firm has responded to those observations; however, the FDA has found that the response inadequately addresses the observations and fails to recognize that to lawfully market Atcell, a valid biologics license must be in effect. The FDA has requested a response from American CryoStem, within 15 working days, that details how the violations noted in the warning letter will be corrected. Companies that do not correct violations may be subject to enforcement action such as seizure, injunction, and/or prosecution.
American CryoStem CEO John Arnone said the company has initiated changes in response to the FDA's initial observations. "With regard to quality and cGMP compliance matters, we stand by our commitment to fully comply with the cGMP quality standards at our facility. We will further respond with an updated comprehensive plan to address FDA's updated observations contained in the letter received January 3, 2018, within the stipulated time-frame of 15 business days. We will actively engage with the agency to resolve these issues and continue to improve our quality systems and processes, as an organization-wide priority."