You had me at iterative
@dolcevita.
Great description.
Agree with your sentiment. The FDA has shown itself to be inconsistent in its process and procedures. Ignoring the positive ODAC, if anyone cares to review the stats, was an entirely new precedent by the FDA. It had not even gone against negative advisory review at such proportions in granting approval in the past (when palms don't get greased unless approval granted) in its history, for all the self congratulatory Harry Hindsights on these forums.
Marks is diplomatically trying to explain past inconsistency here, and again, objectively, you'd be asking, so why didn't you approve MSB using your explained logic, by which approval should have been granted? He would probably reply something like "on balance, we did not find the evidence compelling enough and would refer you to my previous comments on the need for an exacting form of evidence in AA, as I did say we are working on, both generally and specifically with MSB".
MSB, and its patients in light of Marks comments, past approvals, etc, were unlucky to get another CRL, just like Serepta was very lucky to gain approval. For all the formal process and procedures, companies are still hanging it in the breeze on approvals to a degree. Some have clearly improved that breeze in the past by subtle bribes, the most common practice "pay-later agreements" of which there are literally thousands of examples at the FDA:
https://www.science.org/content/article/hidden-conflicts-pharma-payments-fda-advisers-after-drug-approvals-spark-ethicalThis is just one example of one form of questionable practice at the FDA. There are so many examples of impropriety and inconsistency at the FDA.
People, if we feel robbed, can you imagine how SI and co. must feel? Whilst some investors have criticized SI and co. for being complacent with investors money (and that likely warranted in the past), SI, Krause, Rose, Fachinna etc must be furious with the FDA. They will have read the Endpoints interview just like all of us and be dying to say to the FDA, you are literally testing the bounds of your credibility now with your treatment of us, given these comments and other approvals. But instead
SI and co. hopefully will intelligently use Marks comments to argue them into a corner on a Remestemcel-L approval on commencement of an RCT in adults, which they have course already set the foundations for in the US. I actually suspect Marks has MSB in mind to a degree with a number of his comments. I absolutely messed up on my research and underestimated the importance of the Feb 2023 FDORA act, which I bet was at least informally thrown back at any MSB protests:
Drug Reforms
Accelerated Approval– FDA requested various updates to the language in the FDCA authorizing the agency to grant accelerated marketing approval to certain products for serious or life-threatening disease or conditions. FDORA amends FDA’s accelerated approval process for drugs and biologics by:
Allowing FDA to require the sponsor to have confirmatory clinical trials underway before accelerated approval is awarded.
Once MSB has a confirmatory trial underway, the FDA will literally be pushing the bounds of credibility on its own procedures, processes and policies if it does not grant MSB an accelerated approval on a third BLA attempt with a confirmatory trial underway, that was done so in alignment with the FDA at the 45 day and other meetings/consults with the FDA.
(20min delay)