Clinuvel Pharmaceuticals has announced that they have been granted orphan-drug status by the FDA to treat the skin condition erythropoietic porphyrias. Orphan drug status is granted to new drugs that treat rare conditions and is used as an instrument to obtain accelerated review by the FDA. The FDA allows tax reductions and market exclusivity for orphan drugs to encourage otherwise un-profitable drugs targeting rare conditions to be developed.
Afamelanotide (formerly Melanotan) is currently being tested by Clinuvel to treat 3 UV related skin disorders and 2 cancer related treatments: Erythropoietic Protoporphyria, Phototoxicity associated with Photodynamic Therapy, Polymorphous Light Eruptions, Actinic Keratosis and Solar Urticaria.
The official answer to whether afamelanotide can be used to treat rosacea is given via Clinuvel’s FAQ:
Could afamelanotide (CUV1647) be used to treat Vitiligo or Rosacea?
Clinuvel is not currently testing for the indications of Vitiligo or Rosacea, and at present no clinical trials are planned for the foreseeable future. By subscribing to our database, you will receive company updates on any clinical trials.
Despite these official designations, there continues to be interest in whether afamelanotide or Melanotan 2 can provide any benefits for rosacea symptoms.
Melanotan is the proprietary name for afamelanotide (CUV1647) which is being development by Clinuvel Pharmaceuticals. Afamelanotide has been designated by the world health organisation as the International Nonproprietary Name for the melanocyte stimulating hormone under development by Clinuvel.
Note that Melanotan 2 is a different peptide to afamelanotide and is not connected with Clinuvel Pharmaceuticals. Afamelanotide is being promoted as a photoprotective product, with any cosmetic tanning effects being downplayed.
[Update:]
When will afamelanotide be available ?
What does this announcement mean for the availability of products based on afamelanotide ? Sadly, it is still difficult to say - even if everything goes well; when you might be able to get hold of it.
Here are the best comments towards an answer that I can find so far; (this is all corporate speak, sorry).
some quotes from Clinuvel CEO Philippe Wolgen:
"While the drug continues to exhibit its pharmacological action, one could say that commercialisation has now become a function of our execution and ability to test the drug in the required amount of patients. However, the worldwide management of an orphan indication is far from simple and still bears risks. Physicians and patients remain our most precious assets for Clinuvel to be successful in our efforts to commercialise afamelanotide."
and
"The current statistics demonstrate that the FDA is looking at novel drugs more critically than ever. According to the FDA, there were only 17 new approvals for new molecular entities [in 2007]"
and
"In afamelanotide, we identified a first-in-class drug which delivers novel pharmaceutical activity. We anticipate and plan to file for our first Investigational New Drug (IND) this year, 2008. Much technical work to generate compelling data on the drug and formulation has been done over the past 10 months to compile a dossier that meets US regulatory standards. Our one and only goal is to build a dossier that will be robust enough to withstand critique by US FDA examiners, all set against background of stricter regulatory review process."
2 comments ↓
This has the potential to make billions in the US.
How long does it take for an orphan-drug to hit your local pharmacy?
Hi Peter,
I went and hunted for more clues as to availability and updated the article above. No obvious answers, sadly.
davidp.
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