Many companies get orphan drug status but not NDA fast-track approval. Getting one does not mean you'll get the other.
For instance Corcept Therapeutics just had a new drug approved a few weeks back. They received orphan status before completing their clinical III trials and filing the NDA. At the time of their NDA filing the requested a priority review from the FDA. The FDA determined the disease Cushings Syndrome, for which Corcept's drug was to be used for, did not warrant a FDA priority 6 month review. instead the FDA scheduled Corcept a standard review which is 10 months.
Corcept now has drug approval, and orphan status on their drug which gives them substantial tax credits and exclusive rights for 7 years.
Terminal cancer is a different story than Cushings Syndrome. Given the relative short life expectancy of THLD's cancer patients I have to feel the FDA would fast-track or priority review the drug but orphan status has nothing to do with that in the US.
Not quite......approval as an orphan drug (orphan drug status) occurs when the marketing application is submitted (NDA) by the sponsor. Orphan status has nothing to do with allowing clinical trials. The drug is clearly designated to treat an orphan disease, however formal approval occurs (if qualifies) at the time of NDA, which then all benefits kick in assuming approval.
The way I understand this when I read it, we have been given designation approval, in turn, giving us "orphan status" allowing us to conduct clinical trials. The not FDA designation not approved, simply means we are not approved to market the drug. The clinical trials must substantiate our claims, evidenced by clinical data, to treat a disease with a specific drug for a specific purpose, according to our application. Then, if approved, we will obtain FDA designation approval, meaning we are given exclusive 7 year rights and the benefits of being an orphan. Is this correct?
Thanks for clarifying that. I believe you know more about these things than I do, based on other posts I have seen from you. I'm not a medical/clinical expert, just someone who hopes TH-302 changes the lives of many who suffer from cancer. I love investing in companies that actually do something good for society.
I'm no expert on this stuff, but I believe that they have filed an application for orphan drug status, which is not yet granted. If I look at the link to the FDA site, they have clearly applied, but the application for orphan status is not approved yet. I don't know how long that normally takes, but I would guess it is greater than 5 days. Don't get me wrong, I think it is good news they applied and I believe they will probably get approval. Just doesn't appear to be there yet.