Something seems to have dramatically changed with the FDA at its Center for Biologics Evaluation and Research or CBER.
Did they start handing out free Red Bull by the case?
So many recent FDA warnings on cell and tissue biologics
CBER is the part of the FDA that regulates regenerative medicine products like stem cells, other cellular materials, and cell products like exosomes.
Compared to past years, in just the first 10 months of 2024, the FDA biologics branch has issued at least 12 warning letters in this space. I recently wrote about the big spike in CBER warnings to cell therapy and other biologics firms. The number then was nine. Now we have to add three more to the list.
New FDA warning letters from CBER on cells and related biologics
Here are the new warnings:
The first two warning letters relate to unapproved amniotic drugs, while the third may relate to a bone marrow study for ALS, although it is a bit vague.
Let’s go through them one by one. The first two letters in part mention the same product, which is interesting.
In some ways, the last one to Dr. Goyal is the most surprising though.
Amnio Technology, LLC
The FDA warning here went to Merrill B. Stromer, Chairman of this Arizona firm. What is Amnio Technology? It is an amniotic membrane-focused tissue processor and manufacturer.
CBER notes that some of the firm’s products, like PalinGen Flow, are unapproved drugs.
Something called ProMatrX ACF is either a drug or a device, says the agency. The letter mentions that there is no IND in place.
CGMP issues are also listed.
It’s a pretty standard warning letter in the perinatal cell therapy space.
Pinnacle Transplant Technologies, LLC
Now we have another Arizona firm, this one led by Roger W. Rose. What is Pinnacle Transplant Technologies? It is an allogeneic tissue bank and processor.
Here the FDA mentions in part the same product as for Amnio Technology: PalinGen.
Are these firms linked together? The letters list different addresses for the firms. MedPage Today got a quote that Pinnacle Transplant Technologies is a contract manufacturer for Amnio Technology. Stromer is also a founder of Pinnacle.
These letters continue the FDA’s trend over the past few years of focusing particular attention on birth-related or perinatal cell products.
FDA has issued a warning letter to Dr. Namita A. Goyal for violations that occurred while conducting clinical research in Orange, CA. Goyal is a researcher at UCI, where I work. The research was overseen by UCI's IRB & Human Research Protections Program. https://t.co/uj5K7HMr8T
— Leigh Turner (@LeighGTurner) October 23, 2024
Namita A. Goyal, M.D.
This letter begins, “This Warning Letter informs you of objectionable conditions observed during a Food and Drug Administration (FDA) inspection conducted at your clinical study site between June 12, 2023 and June 23, 2023.”
The inspection was conducted via the agency’s Bioresearch Monitoring (BIMO) Program. The letter has a long list of issues with the clinical trial work. These include problems related to the IRB component. It seems from the letter that one or more IRB members who reviewed the protocol were also involved in the clinical study.
The letter isn’t entirely clear as to the nature of the studies and biologics involved. It does mention ALS and bone marrow aspiration (BMA). To try to figure this out, I turned to Clinicaltrials.gov, which listed 5 studies by Dr. Namita Goyal.
One of the listed studies is on NurOwn for ALS, which uses autologous bone marrow. Is that at all tied into the FDA warning or unconnected? Note that BrainStorm Cell Therapeutics makes Nurown.
Leigh Turner pointed out in a tweet (above) on this warning that Dr. Namita Goyal works at UC Irvine, where Leigh is a professor. It’s rare to see the FDA warn someone at a top-tier university.
Why is CBER so active in 2024?
Why is the FDA so unusually active on cell and tissue biologics this year?
By my count CBER has now issued 12 warning letters in the cellular and regenerative therapy arena in just about 10 months. In the past, there may have been nearly an entire decade when CBER may only issue 12 warning letters total.
Why the dramatic change? The surge in warnings began before the agency succeeded in its appeal in the key Cell Surgical Network case, but could the agency have had a sense prior to that that it was going to win?
Did Commissioner Califf decide, regardless, that they were going to ramp way up on biologics enforcement? Whatever the reason, I see this as a major step in the right direction.
For more background, see this explainer post: What is an FDA warning letter & how should firms respond?