Post by
RetailRube on Jul 29, 2014 2:36pm
Orphan Drug Status vs Fast Track Designation
Today, as you know, Bioniche announced they submitted their application to the FDA for Orphan Drug Status for MCNA. A decision by the FDA is expected within 90 days. That means end-of-October this year. So we will know by the time we get together in Belleville for the AGM in early November.
What does Orphan Drug Status mean? Here is a summary of what I read in Wikipedia:
Several decades ago, the US governement increased the amount of testing required before a drug could be approved. This was to avoid a repeat of the Thalydomide scandal. However, the increased clinical trial cost meant it was no longer economic for pharma companies to develop treatments for diseases with relatively rare incidence (lower annual revenue). These rare diseases became "orphaned" as pharma companies abandoned their trials for them. Thefore, the US created the Orphan Drug category which received financial incentives to offset the poor business case. These sweeteners included longer patent protection and lower FDA application fees.
But Orphan Drug Status does not mean faster approval, as far as I can determine.
Faster approval comes from "FDA Fast Track Designation".
From a press release dated May 04, 2006: "Bioniche today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company's proprietary Mycobacterial Cell Wall-DNA Complex (MCC - trademarked Urocidin) for the treatment of non-muscle invasive bladder cancer in patients who are refractory (unresponsive) to Bacillus Calmette-Guerin (BCG), the current standard therapy." "This FDA Fast Track designation means that, when data from the Phase III program becomes available, the Company could expect an expedited review of its Biologics Licensing Application for Urocidin."
I do not know if there is an expiry date on Fast Track Status. I also do not know if Endo used up that status during their discussions of Phase 3a results with the FDA, which led to the ill-fated Phase 3b trial, subsequently abandoned. However, since it refers to faster review of the BLA (which was never submitted) then just maybe this Fast Track Status is still alive.
If we have both of these designations, then it doesn't get much better than this. Right?