We are into the end of September 2020, but it certainly hasn't been the September to remember that I was hoping for!
But, September ain't over just yet... and it is coming down to the wire on the Ryoncil approval.
Nothing is for sure, but everything is pointing to a positive results.
To me we've passed the ODAC exam, and we are just waiting for the ceremony to pick up our Ryoncil certificate
I look at the circumstantial evidence for MSB'ers and feel confident that Ryoncil will be approved.
The FDA had plenty of time to raise their concerns to Mesoblast about GVHD, they could have stopped the the BLA on GVHD, but they didn't - so why would FDA continue on a charade to stop GVHD at the 11th hour?
- GVHD is a niche market
- Manufacturing was brought up in ODAC review, but was never put to a vote
- ODAC had no guidance on manufacturing anyway
- JCR issue with manufacturing actually highlights that there are QA processes in place to stop manufacturing when required
In the first year for Mesoblast, we are likely to treat max of 600 children in GVHD... but, if you look at the list above and add them up, how many patients have been treated by Rem-L already?
So if FDA had issues with manufacturing - well, where is their duty of care to these patients?
e.g. "we don't trust your manufacturing, but use this in your trial as these patients aren't real"????
No way - they would have looked at the same data, the same manufacturing and based on that conclusively gave Rem-L the go ahead for ARDS and MIS-C.
Also, why would NIH run a trial in their premium hospitals if they don't trust the product?
Both the FDA and NIH are not playing games here, lives are at risks.
Recent publicity- Fierce Biotech Innovation Award for Remestemcel-L, Mesoblast was a key participant in the Regenerative Medicine
- Cell Therapies Virtual Event - why were we invited to these events? Because, the scientific community is appreciating Mesoblast work.
So, there's always a risks of Ryoncil not been approved or receive a CRL.
But, based on the above I feel pretty confident that Ryoncil will be approved.
If FDA don't have a good basis for rejecting Ryoncil, then they will stifle innovation - as which other companies will be willing to go through the arduous, long and costly FDA process only to be rejected on the 11th hour. That's just not good business.
The FDA has on record said that they want to cut out all the noises but encourage real science, and that science is via data.
They have looked at the data and ODAC approved 9-1!
Unlike the ODAC event, I feel quite confident and relaxed.
At least we don't have to do an all nighter - but, it was fun
So yeah - we've passed the ODAC exam, and we are just waiting for the ceremony to pick up our Ryoncil certificate
Enjoy the ride MSB'ers - and HODL
Get excited - we hold four P3+ aces (GVHD, C-19 ARDS, CLBP, CHF) and our first big milestone is almost here
Tick tock tick tock
Bring them on
(20min delay)