Here’s another warning from the FDA in 2020 showing its mindset in cleaning the deck so that it can set up a robust regulatory regime going forward:
https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/consumer-alert-regenerative-medicine-products-including-stem-cells-and-exosomes
It still stands today, it seems. What is clearly changing is that after substantial additional work and much greater understanding of what the FDA wants to see in these types of products, in terms of not just clinical efficacy but also manufacturing safeguards (which are more complex those for these biologics), since 2020, there is a BLA from a minnow Australian company sitting on the FDA’s desk that is being looked at and waiting for approval. It has clear support of real world, long term evidence, clarity about the potency of the product ready for commercial release compared to an unimproved product that was used in an earlier, failed trial by a different company, and potency assays that have been developed to give support to particular critical quality attributes pointing to the mechanism of action.
It is in this sense that any approval of Ryoncil for kids’ aGVHD will be pivotal, and landscape changing.
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Here’s another warning from the FDA in 2020 showing its mindset...
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