In the recent years the EMA and the FDA have intensified their collaboration and they now have agents posted in both camps to strengthen that work. However, there is no guarantee that a drug approved by the EMA will be approved by the FDA. In clinical trials the CROs, sponsors and regulators also take into account the geographical, racial and cultural differences there might be. Even though Europe and the US seem very much alike, there might be differences in behavior, culture, food habits or climate (just to name a few) that can have a direct effect in the scientific evaluation of a drug. To us it seems hysteric but it all serves one purpose: To deliver safe drugs that work in the targeted population.
In Clinuvel's case, I admit that it seems that the FDA are dragging their feet. They could indeed have expedited the process and let Clinuvel submit an NDA, but then again, with the PASS protocol not yet in place in Europe, they can piggyback and just wait for that to be finished and perhaps use it directly in the US.
It's worst for the patients. I can't imagine how tough it must be to know there is a safe drug out there but you can't get it.
I believe the FDA will eventually approve Scenesse. But I don't think it will be this year.
My assumption is that Clinuvel will be allowed to submit an NDA later this year. They will get accelerated approval which means that they will rely on surrogate endpoints and thus perform a confirmatory phase 4 after the marketing of Scenesse in the US, which I believe will take off in March or April, 2017 at the earliest.
I also think they might want to distribute Scenesse for EPP through a BP partner who will also be on board for the further advancement of the Vitiligo program.
All speculation but we will know the first steps soon enough I think.
In the meantime, we should pray for compassionate use in the US for all the EPP patients there.
