RCE Charts, page-946

There are multiple reasons for Orphan drug designation.
The rare disease criteria is one option. As far as I am aware there has always been another path which is for a serious condition that has no other recognised treatment.

Below from tga listing of orphan drug. This is Australian tga but I believe usa fda has similar. The treatment this guy received could fit if it was truly as life threatening as the announcement indicated, although it was probably more about quality of life than life threatening.



2. Serious Condition the indication is the treatment,
prevention or diagnosis of a life-
threatening or seriously
debilitating condition1 in a
particular class of patients (the
relevant patient class)
the indication is the treatment,
prevention or diagnosis of a life-
threatening or seriously
debilitating condition