My understanding is that you've already listed the benefits. Tax...

My understanding is that you've already listed the benefits. Tax credits, user fee reductions and 7-10 years of market exclusivity. From the FDA website.....

Sponsors seeking orphan drug designation for a drug must submit a request for designation to the agency. Sponsors requesting designation of the same drug for the same rare disease or condition as a previously designated product must submit their own data and information to support their designation request. Orphan drug designation is a separate process from seeking approval or licensing. Drugs for rare diseases go through the same rigorous scientific review process as any other drug for approval or licensing.

So, assuming phase 2 is successful for the -201 trial (as defined by the FDA), then expect phase 3 trials. Years of patient waiting to come with the share price languishing in the doldrums, with the obvious exception of the spike if the data readout in Jan '25 is impressive.