One more benefit to have an orphan designation for a rare...

One more benefit to have an orphan designation for a rare disease is the possibility for early approval. Some drugs have been approved right out of phase2. I don't see that happening here because the numbers of patients treated is probably too low for the FDA to consider it. That said, MSA is a fatal condition, and it looks likes early treatment will have a better result. Assuming the 201 results are good, I think there is a good case for special access to early stage patients. Waiting a few years for a P3 trial to complete could result in their disease being advanced stage by the time they get to treatment.