Ann: Positive Safety Data from Phase 1 Clinical Trial, page-108

Typically I would say that the FDA would need another pivotal trial…
They have their rules and guidelines in place, so i would imagine that they could provide this treatment Fast Track and Breakthrough Designation; which would then lead to a expedited pivotal trial with a small number of patients. So they have their measures to significantly expedite and support the „special cases“ Having said that…

Based on the urgency and drastic need for this treatment it is not out of this world that they do simply allow RCE to start treating patients, while in parallel they request RCE to update them on the newly treated patients for a certain duration. The FDA would then keep assessing them until let’s say 100 patients have been successfully treated; to then reach to a final conclusion: Approved.
I would consider this really to depend on how management „sell“ it to the FDA and how good their current data is. They really have to prepare a „convincing package“. Possibly a partnership with a well established pharma company in the US would strongly support this notion; strengthening the position of having all the facilities, logistics and medical network already in place to smoothly start treating patients with no disturbances and „ramp up“ complications…