I think we need to remember that Clinuvel hasn't formally submitted an NDA in the US. FDA can't approve a drug formally without the submission of a dossier.
They have other options, of course, but full approval is not possible before the company sees the time fit to submit an NDA.
I think the APF is somewhat forgetting this.
If EPP suffered in the US are to benefit from Scenesse this coming summer, which I truly hope will be the case, then compassionate use or some other unconventional opportunity needs to present itself.
As for the delays in Europe I think it's a matter of the EMA being overly cautions and fearing that Scenesse will be used off label.
Also, the company clearly made a lot of rookie mistakes and probably still is. However, I think they will succeed and the sufferers will get the treatment they deserve. I can't imagine how painful and frustrating it must be knowing that a solution is out there but you cannot get it.
As for off label: It is very unwise and extremely counterproductive to dream about off label and the fact is that investors don't need off label to get a good ROI. There are plenty of legit conditions out there that need treatment with Scenesse to catapult the pps to hights that no one cannot really predict at this time. EPP could easily justify a market cap of $500-700 m. Vitiligo is not at all priced in at this time. Fully approved Vitiligo will be worth at least $3-5 m. in market cap.
Then add label extensions for HHD and all the other conditions that the company ran trials on. Then add pediatric EPP treatment which in my view is by far the most important place to focus for the company tight now from an ethical point of view.
And if a topical solution for the children through the Singapore JV will lead to a topical solution for the adult population as well then it is also good for investors.
Bottom line: We don't need off label. We should salute management for preventing off label because it it counterproductive and will only upset regulators. I believe the company will fight off label and that it is the only right thing to do.
GL
