Thank you for your response. Whilst I don't agree with the conclusions drawn, at least it provides a few points to discuss further.
You have quoted my reasoning for the approval of Ryoncil in children with SR-aGvHD. That is however not the same as label extension of the same therapy to also include adult patients:
1. There was no approved therapy in children with SR-aGvHD prior to Ryoncil.
2. Approval was based after the FDA acknowledged (aka doing-a-180) that MSB's 54 patient P3 trial (also in children only) was "well-controlled" - at least now under the new "regime" this is considered well controlled, despite the hold-up for years with the FDA arguing it wasn't.
But let's just say the FDA ignores the issues with the previous trials and uses the same data to grant label extension - what is the timeframe you think this is going to happen? A week? A month? 6 months? Any longer than that and based on current estimated provided by CYP our trial would be fully enrolled already.
However, a very important issue I have been repeating over and over again:
SR-aGvHD vs HR-aGvHD (ignoring the children vs adults aspect).
Do you think the FDA would want to see some sort of clinical evidence forst before they let MSB charge money to adult patients?
What (old) data does MSB have in adults with HR-aGvHD?
I let the FDA tell you:
https://web.archive.org/web/20210310192904/https://www.fda.gov/media/141131/download
Adult patients with HR-aGvHD in addition to SOC - a 37% ORR rate. MSB didn't challenge that statement.
What patient group is CYP targeting again?
a) adults
b) HR-aGvHD
c) combined with SOC
37%
Now get me a doctor that will get an adult patient with HR-aGvHD to wait and addition 3 days before they become eligible to enrol in MSBs trial for SR-aGvHD or charge them for that therapy via label extension if granted within the next 6 months or prescribed it off-label prior to SR-aGvHD onset (also knowing that insurance may not cover it), when that same patient could be enrolled in our trial with a similar type of therapy, possibly 3 days earlier (higher chances of survival of treatment starts immediately after onset) at no cost.
May I also give some food for thought here:
MSB has been granted Orphan Drug Designation in aGvHD. I don't believe such designation can be limited to children only.
Shouldn't the MSB lawyers be calling CYP now due to market exclusivity now that Ryoncil has been approved?
Same, same - but different therapy?
Again, I see no issue with MSB's approval and our ongoing P2 trial. My view.
(20min delay)