FDA approval process

I thought it would be timely to start a thread so we can discuss something other than how the share price is moving up and down each day...

Ryoncil has fast track designation (granted in Mar17) just submitted it's BLA on 31 Jan 2020 and will hear back from the FDA some time in March 2020 on whether or not priority review will be granted.

FDA website - fast track designation

FDA website - priority review

In my books, the FDA will give MSB priority review ... it is clear that Ryoncil achieves significant improvement for aGVHD sufferers... it would be very hard to justify why they wouldn't.

Now to what could happen later this year when the FDA completes its review... there are three broad possible outcomes and they are:

a) Approval granted
b) Complete response letter (CRL)
c) BLA rejected

Broadly speaking, a CRL is actually very common. This basically means the FDA requires further information or details before it will approve the product. The FDA will clearly articulate the deficiencies to the company, but in Australia that is not usually shared with shareholders.. in the US it is not required, but is commonly shared (probably because the large shareholders know how the FDA works and demand this information). These can range from simple things like tweaking the manufacturing process ... to the other end of the scale where further clinical data is required.

I am hoping we get approval straight off the bat (and I am quietly confident it will), but I am also prepared that a CRL may be issued .. in which case MSB will need to schedule a post CRL meeting to discuss how to amend the BLA and re-submit, which will obviously delay product launch.

Food for thought... usually the food that doesn't taste good or go down well is best for you.