Hi...

Hi 07Gerard,

https://www.fda.gov/regulatory-information/search-fda-guidance-documents/potency-assurance-cellular-and-gene-therapy-products

The regulatory stance was changing even as the article you kindly provided was written. It is dated 23 Dec 2023. On 28 Dec 2023, this draft guidance above was issued by the FDA. The FDA followed with a zoom presentation by Dr. Klinker, the CMC reviewer for the company’s SR aGVHD product, clearly explaining how such a strategy is even more important than potency testing.

I think the article is well written, and it is factually correct in pointing out the dodginess of the stem cell industry in the IS (if there’s such a word) that the FDA is wary of. However, what is less talked about is a warning a few years ago to the industry to clean up its act. I think it was to prepare the industry and the agency for a more serious look at the possibility of approval.

Gamida is an example cited in the article as habving received approval in the last 12 months. It may also illustrate the regulator’s new readiness to approve such therapies.

I agree with the sentiment or doubt expressed in the article about the company not being American, which is potentially an issue. I covered this in jest in another thread, the one on the issuance of new shares, to be approved st the next AGM, to the CMO.

All in all, a good article and thank you for drawing our attention. However, I do believe that changes in the FDA’s attitude are happening right as we speak. I
did work for a government regulator in another industry, so I know how they work - typically cautiously and slow to embrace change, but they do use their avenues to communicate change when it is afoot - eg. public speeches like the one by the commissioner in Jan/Feb on rare diseases and the difficulty of recalling drugs because of a lack of authority, townhall series to explain published guidances which in turn explain how an agency exercises its legislative authority.

I will post the link with the FDA historic warning to the stem cell industry separately if I manage to locate it.