Afamelanotide (Clinuvel CUV1647) gets orphan drug status in the US

Written by on July 30, 2008 in melanotan with 22 Comments

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."

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About the Author: David Pascoe started the Rosacea Support Group in October 1998. .

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22 Reader Comments

  1. Peter says:

    This has the potential to make billions in the US.
    How long does it take for an orphan-drug to hit your local pharmacy?

  2. David Pascoe says:

    Hi Peter,

    I went and hunted for more clues as to availability and updated the article above. No obvious answers, sadly.

    davidp.

  3. MD says:

    as the one-year clinical phase III trials have just been started in european countries thru summer 2008 there will be no legal availability before end of 2009.

  4. Peter says:

    Looks like 2010 @ best for the US, oh well.

  5. Mariel says:

    This would be good for me. I have the sun sensitivity of porphyria, which means I must cover up. I have HCP, which is not AS sun sensitive as erythropoetic porph, but still is sensitive. I also have had 6l skin cancers and widespread actinic keratoses. I cover up so well that I only had one cancer removed this year and two keratoses.

    Mariel

  6. Peter says:

    I have A.K., got my semi-annual treatment today. I mentioned CUV 1647 to my dermatologist as a treatment for me, she had no problem with doing the implant after she gets a little more educated in the drug once it gets blessed in the US

  7. David Pascoe says:

    I think it this will be a great treatment for sun sensitive indivduals. Lets hope for a smooth Phase III process.

  8. Peter says:

    Got my vote D.D.

  9. ray says:

    I found the link to this on a Porphyria support group site (bless you Mariel). I’ve lived all my 30 years having to accomodate the impact of erythropoietic protoporphyria. I’m not ashamed to say I welled up while reading, the potential this bears is massive. Can’t wait to investigate and learn more.

    • Melissa Fincher says:

      I have EPP as well, Ray. I too, welled up while reading. I am praying for a quick approval from the FDA on this drug.

  10. David Pascoe says:

    Just posted is an update on afamelanotide saying that it has shown promising preliminary Phase 3 results from 14 participants in Switzerland.

    See: afamelanotide positive Phase III results for erythropoietic protoporphyria

  11. Dale says:

    What an exciting prospect. I have 3 children with EPP and this drug could change their lives literally.

  12. kat says:

    I have suffered from PMLE my whole life and can not wait for this to be accepted by FDA in the US!

  13. Barbara says:

    Latest commercialization dates:

    Q1 2011 for Europe

    Much later for US. Why they are delaying in the US is beyond me but it’s ridiculous and really makes me angry. They got the green light to start US trials in Juanary 2009 and here we are 8 mos later and no trials have started in the US.

    Pathetic!

  14. Felipe says:

    Anyone please help me

    today is may 6th 2010 can i get afamelanotide anywhere in the world

    please please have mercy

  15. David Pascoe says:

    Sorry Felipe, but afamelanotide is not available anywhere as yet. Only those in the clinical trials have had access to it.

  16. Jay says:

    My son has EPP. How difficult or is it even possible to get him on the US trial list?

  17. David Pascoe says:

    Just updating my previous comment #15: afamelanotide, now known as Scenesse is available on prescription for sufferers of EPP in Italy.

    See: Afamelanotide (Scenesse) now available in Italy

    davidp.

  18. David Pascoe says:

    Hi Jay,

    Your best bet is to contact the American Porphyria Foundation, who are hoping to create a database of all porphyria patients in the US. They should also be able to advise on clinical trials and locations. Be aware that even if you should find a suitable trial location there may be age restrictions in force as well.

    http://www.porphyriafoundation.com/

    all the best,
    davidp.

  19. patricia says:

    hi is there nothing that can cure vitiligo or at least slow it down ! i have had vitiligo for 30yrs just in the same 4 places on my body but in the last two yrs it just seems to be spreading very quickly. i am on thyroxine tabs for underactive thyroid i was on 150mg a dy then two yrs ago dr`s dropped it to 75mg cold this be something to do with it spreading ? thanx

  20. jaxx1 says:

    Hi

    I to am a sufferer of severe vitiligo with very little tan left .

    Will Afamelanotide help?

    Thanks

  21. zz says:

    hi
    i am too a vitiligo suffer aswill..i thought afamelantiode can help vitilgo?am in the uk dont know when we will got this drug or we dont get it at all..doctors in uk dont care about vitiligo paients from my experiences…..from the website it says this could help the repigmtion..or am i geting it wrong?????

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