FDA Grants Orphan Drug Designation, page-15

Gingerclam:

Assuming I'm reading the FDA site correctly,

If the FDA gave Orphan status during Friday business hours in the US, that may well have been after Neuren could have put an ann out to the ASX.

I think what the website is saying that, while they have Orphan Designation, they're not allowed to use the drug to treat Retts in an arbitrary fashion. To me, "FDA approved" means "able to go on the market". A few things I can speculate on:

I have no idea whether Orphan and Breakthrough are discussed at the same meeting. It may be that Neuren gets Orphan first ad Breakthrough (if it gets it) a little later.

Even if 2566 gets Breakthrough, the "not FDA approved" may well still stay the same, as any move to use 2566 for treatment- whether as part of Ph III or more generally- may still be subject to further discussion with the FDA.

I was intruiged that, in the ann from 30 Dec, the comment that "Neuren expects to meet with the FDA in the first half of 2015 to discuss remaining requirements" was distinct from the Orphan/Breakthrough process.[/QUOTE]