FDA Issues Warning to New Jersey Developer for Marketing Unapproved Stem Cell Product

Last night, the U.S. FDA posted a warning letter addressed to American CryoStem Corporation for marketing an adipose derived stem cell product without FDA approval and for significant deviations from current good manufacturing practice requirements.

Last night, the U.S. Food and Drug Administration (FDA) posted a warning letter addressed to American CryoStem Corporation for marketing an adipose derived stem cell product without FDA approval and for significant deviations from current good manufacturing practice requirements.

John S. Arnone, Chairman/Chief Executive Officer of the Monmouth Junction, New Jersey company, was issued the warning after it was discovered that the product raised potential significant safety concerns, putting patients at risk.

The product, Atcell, was found being distributed directly to physicians to treat patients for an array of serious or life-threatening conditions, including anoxic brain injury, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), stroke, and multiple sclerosis (MS).

“As part of our comprehensive policy framework for the efficient development and regulation of cell-based regenerative medicine, we’re going to be stepping up enforcement activities against those who manufacture and market products in ways that put patients at risk,” said FDA Commissioner Scott Gottlieb, M.D. in the official FDA press release.

Atcell was found by the FDA to have included adipose tissue and been marketed sans FDA approval. While the product is designed for autologous tissue, the way in which it was being processed requires more than minimal manipulation of the adipose tissue. Because the manufacturing steps alter the original relevant characteristics of the tissue and have the potential to introduce contamination of the product, it can put patients at the risk of harm and, under existing law, this is enough to require the product to undergo FDA review.

“We see great promise from the field of cell based regenerative medicine, but there are also novel risks,” continued Gottlieb. “Our goal is to implement our new policy framework in a way that helps this promising field advance, while making sure that we address issues that can put patients at risk. It also means taking an efficient, risk-based approach to making sure that products undergoing more than minimal manipulation, which makes them subject to FDA’s review and approval, and particularly products intended to treat serious and/or life-threatening disease or conditions, must prove that they deliver the benefits they’re promising to patients.”

The product creates concerns under the FDA’s risk-based enforcement strategy because 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. Physicians were being directed by the company to deliver the drug to patients in several different ways, including via intravenous injection, intrathecal injection or infusion into the central nervous system (CNS) and by aerosol inhalation.

Additionally, the inspection by the FDA discovered evidence of significant deviations from current good manufacturing practice requirements in the manufacture of Atcell that pose risks, including possibly being contaminated with microorganisms or having other severe product quality defects. Specific deviations included invalidated processes, an ineffectively controlled environment, lack of control components used in production, and a lack of adequate and proven product testing.

American CryoStem was issued a list of inspectional observations at the conclusion of the inspection, and the FDA has concluded that the response unsatisfactorily tackles the observations and fails to recognize that to lawfully market the drug, a valid biologics license must be in effect. While being developed, an investigational new drug application (IND) must be in effect for the drug to be legally distributed for clinical use.

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.

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