In my view, SI clearly signalled the immediate future direction of the company in one sentence of the Bioworld interview that @Xenox posted a few days ago.
https://www.bioworld.com/articles/4...-of-fda-crl-for-ryoncil-bla-in-pediatric-gvhd“We’re going to have a meeting with the FDA in the next 30 days to discuss an accelerated approval, which is a conditional pathway, and
we will agree to do another trial but after the product gets approved.”
So to me, it’s clear that a negative result at the Type A meeting will be a major roadblock for both pediatric and adult aGVHD. MSB will be forced to shift focus to Covid ARDs, to give the FDA what they want, compelling data from a randomised placebo controlled trial. I hope this prospect inspires aGVHD interest groups to launch a concerted lobbying and media campaign. It’s definitely worked in the past.
But there’s another factor that seems to have been lost in all recent washup.
Since July 2nd, Remestemcel-l has been available through an FDA sanctioned Expanded Access Protocol for Multisystem Inflammatory Syndrome (MIS-C). This allows for up to 50 children aged 2 months to 17 years to be treated across the U.S.
This condition is so debilitating that I suspect most parents would have to at least seriously consider the EAP for their sick child, should they qualify.
Now the U.S. Centre for Disease Control and Prevention (CDC) has just announced that, “
As of October 1st, the number of cases of multisystem inflammatory syndrome (MIS-C) in the U.S. surpassed 1000.”
So if just 5% of those cases were successfully enrolled, the EAP would be just about complete.
https://www.cdc.gov/mis-c/cases/index.html(An interesting short read if you’ve got the time)
Of course, the EAP is not a randomised controlled trial, but regardless, since SR-aGVHD and MIS-C are both inflammatory conditions affecting children, imo it would be extremely fortuitous if MSB had available pediatric observational and biomarker data from this EAP in time for the Type A meeting.
As an aside, last Friday the CDC released another report identifying the MIS condition in adults, (MIS-A). Most patients either tested positive to Covid-19 or carried antibodies for it, indicating recent infection.
https://www.livescience.com/multisystem-inflammatory-syndrome-adults-covid-19.htmlMIS-A just adds to the growing list of conditions that MSB could potentially target, both on and off label, in the future.
My personal view is that there appears to be considerable scepticism about stem cell technology in general, at senior levels of the FDA. It clearly will require unequivocal evidence to gain the first, even conditional approval, but once the dam wall breaks, anything is possible.
Usual bias. Keep the faith.
Herro
(20min delay)