Media Thread, page-18124

Totally agree @Taureanbull

IMU holds an orphan drug designation for CF33-hNIS in cholangiocarcinoma (bile duct cancer), an extraordinarily hard to treat condition.

So why is Orphan Drug Status important?

FDA orphan drug status is granted to encourage the development of treatments for rare diseases affecting fewer than 200,000 individuals in the US.

With this designation, IMU is offered many incentives:

•tax credits;

•possible grant opportunities;

•exemption from specific administrative fees; and

•7 years of market exclusivity following FDA approval.

Now possible Accelerated Approval (AA).

Dr Vinay Prasad, head of CBER - the FDA’s Centre for Biologics Evaluation and Research has stated that “the FDA plans to “rapidly make available” rare disease drugs and make use of surrogate endpoints to get promising medicines to patients before they clear the traditional efficacy bar for authorization”. This certainly may include VAXINIA in Bile Duct Cancer.

As recently as April, the FDA Commissioner, Marty Makary stated that the FDA was planning a ‘new pathway’ for rare disease drugs. This has now become a consistent theme with the FDA.

Only time will tell if our Bile Duct Cancer treatment isconsidered more closely, due to this new approach. We can keep our fingers crossed.

I suspect that IMU is, and has been, exploring other potential ‘Orphan’ conditions to target.

Again, just the opinion of one poster.