Friday and Saturday showed a burst in FDA activity related to unproven stem cells and regenerative products including a warning letter to the unproven perinatal “stem cell” provider Liveyon.
This was an unusual 48-hour period and a very good one for those who believe in science-based regenerative medicine but in my view a bad day for perinatal stem cell clinic and exosome suppliers. For general background on exosomes see these past The Niche posts.
I’m sure there are a lot of hardworking folks at the FDA who deserve hat tips for this effort and also former Commissioner Scott Gottlieb, but I give a particularly big hat tip to Dr. Peter Marks, head of the FDA’s CBER branch (pictured at left).
Here’s a summary of the actions on Friday and Saturday:
- Warning Letter to Liveyon Labs/Liveyon LLC (and accompanying press release: FDA sends warning to companies for offering unapproved umbilical cord blood products that may put patients at risk.) The PR also mentions two recent untitled letters, “In addition to the warning letter issued to Liveyon Labs and Liveyon LLC earlier this month, the FDA sent untitled letters to RichSource Stem Cells, Inc., and Chara Biologics, Inc., for offering unapproved stem cell products to patients.”
- So-called 483 inspection reports now publicly available on Liveyon LLC and Liveyon Labs. These were sent to Erin M. Sairafe, Chief Compliance Officer for both firms.
- On Saturday, the FDA CBER branch issued a safety communication on exosomes that includes reports of adverse events in Nebraska due to an unproven exosome drug product.
Starting with Liveyon, it has been back in the news the last month or so because of the compelling podcast Bad Batch. The warning letter to the firm was addressed to Liveyon CEO/President Roya Panah and founder John Kosolcharoen. Who is Roya Panah? It turns out that Panah (pictured at right from LinkedIn) also goes or went by the name Roya Kosolcharoen and may be John’s ex-wife.
The letter lists many problematic issues for the company based on an inspection in May of the company’s own production facilities. Note that this is an important point on the location because since the product from independent supplier Genetech led to a dozen or more people going to the hospital, Liveyon has been making its own biologics.
The new warning letter focuses in part on insufficient testing of the umbilical cord product and concerns about sterility. Notably, the letter also indicates that Liveyon’s products are unapproved drugs. It also recommends testing this kind of product for Zika virus.
From the accompanying PR from the FDA:
“The FDA’s mission includes protecting public health by helping to ensure the safety and efficacy of medical products that patients rely on. The agency is aware that there are establishments who prey upon vulnerable populations by commercially marketing stem cell products with false and misleading claims about their effectiveness for treating serious diseases,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “The FDA is taking this action today because Liveyon Labs and Liveyon LLC failed to take appropriate measures to protect patient safety. As evidenced by the number of actions that the agency has taken this month alone, there are still many companies that have failed to come into compliance with the Federal Food, Drug, and Cosmetic Act and FDA’s regulations during the period in which the agency intends to exercise enforcement discretion for certain products with respect to FDA’s investigational new drug and premarket approval requirements, when the use of the product does not raise reported safety concerns or potential significant safety concerns. This period, which ends in November 2020, has allowed product manufacturers time to engage with the FDA to determine if they need to submit a marketing authorization application and, if so, seek guidance on how to submit their application to the FDA for approval. The agency continues to urge these manufacturers to engage with the agency about their regulatory requirements in the coming months.”
Shifting gears, what about the exosome notice by the FDA? Overpriced and “underscienced” exosomes are gaining more traction in the unproven stem cell clinic sphere, perhaps in part because of the actions against adipose stem cell clinics. Here’s a key part of the FDA communication on exosomes, noting adverse events:
“The Food and Drug Administration (FDA) is informing the public, especially patients, health care practitioners, and clinics, of multiple recent reports of serious adverse events experienced by patients in Nebraska who were treated with unapproved products marketed as containing exosomes. These reports were brought to the agency’s attention by the Centers for Disease Control and Prevention, among others, and the agencies worked with the Nebraska Department of Health and Human Services. FDA is carefully assessing this situation along with our federal and state partners.
There are currently no FDA-approved exosome products. Certain clinics across the country, including some that manufacture or market violative “stem cell” products, are now also offering exosome products to patients. They deceive patients with unsubstantiated claims about the potential for these products to prevent, treat or cure various diseases or conditions. They may claim that they these products do not fall under the regulatory provisions for drugs and biological products – that is simply untrue. As a general matter, exosomes used to treat diseases and conditions in humans are regulated as drugs and biological products under the Public Health Service Act and the Federal Food Drug and Cosmetic Act and are subject to premarket review and approval requirements.”
I’m hoping to learn more about what happened with the exosomes in Nebraska such as who administered them and who was the supplier.
Overall, the FDA continues to generally increase its activity in the unproven stem cell and regenerative medicine arena. The last few days have been particularly encouraging.