FDA disappointed with slow uptake of approvals for stem cell therapies

Regulatory NewsRegulatory News | 09 June 2021 |  By 

FDA's Wilson Bryan

At a recent meeting, a US Food and Drug Administration official said the agency is “concerned” about the slow uptake from companies seeking regulatory approval to market and distribute stem cell therapies and is therefore stepping up their enforcement efforts against companies making unapproved therapies.
Wilson Bryan, director of FDA’s Office of Tissues and Advanced Therapies (OTAT) in FDA’s Center for Biologics Evaluation and Research, told attendees of an 8 June meeting sponsored by the Food and Drug Law Institute that “we have been extremely concerned” about the high number of cell therapies, and particularly stem cell therapies that are being marketed to patients “for a wide variety of indications without having evidence of safety and effectiveness.” Very few firms have taken necessary steps to seek approval of their products before marketing.
He added that because of this foot dragging “we are going to have to increase or continue to increase our enforcement activities.”
Bryan said that enforcement discretion is now over for makers of these products. Except for limited circumstances, those marketing stem cell therapies must seek FDA approval. This point was also made by Peter Marks, director of FDA’s Center for Biologics Evaluation and Research, who announced at a meeting last month that the 3-year clock for enforcement discretion is over and there will be no more extensions; the COVID 19 pandemic prompted a 6-month extension.  (RELATED: Time’s up for questionable cell and tissue products, Regulatory Focus, 22 April 2021)
Bryan also sought to dispel a misperception that enforcement discretion makes it legal to market unapproved products. He said that enforcement discretion “means that we are not going to enforce the law, or we will enforce this with a lighter hand.”
Bryan said that during the period of enforcement discretion, CBER sent 400 letters to manufactures and health care providers who may be offering violative stem cell or related products. There have also been two enforcement actions for injunction against manufacturers of violative products; FDA was successful in obtaining permanent injunctions against the firms.  (RELATED: FDA wins federal court case against stem cell clinics, Regulatory Focus 4 June 2019)
Bryan said that particularly concerning are those products that are administered for orthopedic indications and injected into joints to relieve pain. “These are very popular…. we just feel that there needs to safety and effectiveness data [for these products],” he said. He is also concerned about practitioners administering stem cell products into the spinal cord.
FDA issued two guidances in November 2017 explaining its criteria for determining whether devices, drugs or biological products derived from stem cells should be subject to FDA regulatory approval. Products that are minimally manipulated would not be subject to approval.


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Tags: cells, FDA, stem

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