"The FDA says it will rule on all of the applications it received while in Minneapolis within 60 days. Typically, between 55 percent and 70 percent of orphan drug status applications are approved."
Rare conditions are often described as orphan diseases when there are very few treatment options, largely due to a lack of money for drug research. There are about 7,000 known orphan diseases in the U.S.
The designation comes with a 50 percent tax credit on any clinical trials that need to be conducted. Drug fees, which currently cost around $1.6 million for one drug application, are eliminated. And a company that succeeds in getting final FDA approval will be allowed exclusive drug marketing rights for seven years.
"When you get orphan status designation, you go up on our website saying that the FDA has given a nod that this is promising for this rare disease," said Cote. "And then the venture capital rains down from the heavens. That is how it has tended to work for a lot of these companies."