"It isn't hard to get Orphan Drug status. Most of the normal...

"It isn't hard to get Orphan Drug status. Most of the normal approval steps are skipped."
I cannot be sure if you are deliberately attempting to mislead here or are just poorly informed. You, as an expert, would be aware that Clinuvel gained ODD for EPP with FDA in 2008! It probably in reality is not that hard, but it is pretty meaningless.

Marketing approval with FDA was granted with the FDA in 2019. Some 11 long years later and I certainly disagree that most of the normal approval steps are skipped. You would also be aware that Clinuvel is the only Australian Drug Development Biotech to have obtained FDA marketing approval with no out licensing arrangement or other deal. Clinuvel is now solely distributing the drug in the US, another Australian first. I have attached a PhD thesis on Australian drug development over the last decade for your reading.

If all you are suggesting is that it is harder to get marketing approval for a non orphan indication then I think even your greatest critic would be in agreement. A non blinded trial of 6 AIS patients is most unlikely to reveal any meaningful conclusions and it is not intended to. It is however a pretty exciting beginning, especially as afamelanotide is already approved for marketing authorisation and could be prescribed "off label" in the US tomorrow.

I leave you with a question. If Clinuvel was to lower the price and position Scenesse on the US market for sun protection/tanning it would be worth a fortune ( the new botox). I would be first in line to take it. Tens of thousands of people have used internet sourced MT-1 over the last several decades. There is clearly demand. Clinuvel management do not do this. They will not even entertain the discussion. Distribution is very very tightly controlled. Either they are fools or there is a greater play at foot. Given past success I am happy to let the story play out.