Ann: FDA Grants Rare Pediatric Disease Designation for Revascor, page-150

I think the overhang is more likely to be removed if they give us positive news on the potency assay situation for Ryoncil's aGvHD inventory. Approval of Ryoncil for aGvHD in kids could only be a few months away if the potency assays succeed, whereas an approval of Revascor in HLHS could still take a long time, even with an accelerated approval. A BLA will still need to be lodged for Revascor (MPC technology) which will have some overlap with the Ryoncil BLA (MSC technology) and which may not need a new full plant inspection as much of it may have all been covered in the last inspection (but I don't know), but BLAs require a truckload of information (literally).The fireside chat video I referenced a few days ago shows the FDA attitude right at the top for AA in these rare indications - as long as safety and efficacy can be shown, they are happy to take the risk of early approval to try to make these drugs available quickly and hopefully encourage drug companies to service rare indications by making the process less expensive. If subsequent trials and RWE don't back up the initial results, then FDA would revoke the AA. FDA might not even require the new trial to have started to grant AA. However, I would guess that an AA will still take several months, probably into the second half of this year.It would probably also generate demand from doctors to use Revascor off label in heart and back pain patients, and help in partnering talks. So this is an important announcement.

Aloha E