GTC Biotherapeutics (fka GTCB)

[DewDiligence]

FDA decisions regarding an orphan-drug designation, i.e. whether two drugs ought to be considered the same or different, are notoriously hard to predict. However, I would be surprised if an orphan designation for an inhaled AAT would block the marketing of an injected AAT for the same indication (hereditary deficiency).

A separate question—and a harder one to answer—is whether an orphan designation for an injected plasma-derived AAT would block marketing of an injected recombinant AAT for the same indication. This is presumably one of the matters GTC will address in deciding how strongly to pursue an AAT program.