US Stem Cell bid for FDA RMAT rejected?

Can a stem cell clinic business get FDA RMAT designation? At least one announced it was trying, but now seems to have given up.

Stem cell clinic business US Stem Cell, Inc. has reportedly announced that it is at least temporarily abandoning its efforts at getting Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. The company’s penny stock $USRM has been gyrating for months and I had earlier wondered if could be some fake news about it. The USRM news now seems real and not good on the RMAT front. Shares plummeted earlier this week (see earlier stock graph above).

As to plans, here’s something from an apparent company PR:

“Until then, U.S. Stem Cell will focus on opening new clinics around the country to better serve patients in need. In addition to the original Sunrise clinic (that has successfully treated hundreds of patients and generated over $2m in revenues in the past 12 months alone), recent clinic openings include Miami and Palm Beach, Florida. Upcoming openings include Dallas, Texas (thanks in part to the early adoption of patient rights by the State of Texas), Chicago, Atlanta, and Denver – as well as other clinics in the northeast and the west coast.”

Opening more clinics…selling non-FDA approved offerings?

This may be what the company sees as a good business move, but in my opinion it puts more patients at potential risk and takes their money for “stem cell treatments” that are not conclusively proven to be safe or effective. I have not seen RCT data from USRM to support their commercial stem cell efforts.

What happened with the US Stem Cell, Inc. RMAT application? We may never know for sure, but rejection by the FDA is one possibility. Over at the RAPS site, a new piece on CBER has this to say (“Marks” refers to CBER Director Peter Marks):

“Thanks to the 21st Century Cures Act, FDA now has a new designation for regenerative medicines, known as the regenerative medicine advanced therapy (RMAT) designation. As of last week, Marks said there have been 19 requests for RMAT designations, 18 of which CBER has acted on, and four of which have been granted…’

Unless US Stem Cell, Inc. is the 1 out of the 19 applications on which the FDA has not acted, then it’s not looking promising for their RMAT just based on the odds. Another possibility is the FDA did not reject it, but asked US Stem Cell for a lot more data and that constitutes “acted on”.

It’s worth a reminder that this business was linked to the blinding of three patients in a presentation at a 2016 FDA meeting and in a NEJM publication. The company has had patient lawsuits too, which seem to have been settled out of court.

More broadly in the oversight arena, to my knowledge the FDA and CBER specifically have not issued any warning letters to or taken other actions (at least in the public domain) on stem cell clinics in ages despite hundreds of such businesses marketing unproven stem cell offerings without FDA approval. And whatever happened to those four key FDA draft guidances? Does CBER have enough funding and staff to tackle the burgeoning stem cell business arena? It remains unclear how the FDA and CBER will handle key challenges under the new Trump administration and with new FDA Commissioner Scott Gottlieb.