I came across the name of this medication which is an orphan drug specifically for congenital ichthyosis. I can't seem to find any other info on it besides that.Could anyone tell me a bit more about it? Thanks

It's interesting because this was originally designed as a cancer drug. It's a retinoid, technically, much like Soriatane or Accutane and it was designed to reduce the level of retinoic acid in tumors.
They call it an orphan drug because they want to use to treat a disease that affects less than 200,000 people. According to the manufacturer, only about 40,000 people have Ichthyosis.
Apparently, instead of introducing synthetic forms of Vitamin A into your system like Accutane or Soriatane, this drug will slow your metabolism and the degradation of the retinoic acid you already have in yoru system. This will cause your body to maintain higher levels and thus, give you the results you would get with Accutane or Soriatane. It's supposed to give you less side-effects than the other drugs and not cause you to build up the retinoic acid.
It "seems" to me that the drug is available, but due to it's orphan status, the manufacturer has exclusivity on it for seven years, so there will be no generic form of it.
I tried to go to the company's website, but they seem to have a different name now, and I guess I should note that they appear to be the company that manufactures Soriatane. They don't list the drug on the new website that you are directed to, which is now Stiefel Laboratories. I guess the next step would be to ask your doctor to ask his drug rep? Or email the manufacturer at www.stiefel.com?

Hmmmm... maybe this is something I should look into also.

Thanks Glori! I asked our derm about it (in Australia) and they don't have it here. He said to do some research and then if it seems promising, we could go to the US (my whole family live in CT). We're going to be seeing Dr. leonard Milstone when we go visit my family so perhaps I'll ask him. In the meantime, I will e-mail Stiefel. It's very strange that there isn't much info on it. You would think there would be?

Sarah Helen- It does look interesting and promising although I don't think I'll get my hope up yet

Hi all, this is Flavio from Italy.
I'm President of the small italian Ichthyosis patient organisation, which is member of the fledging European Network, ENI (http://www.ichthyose.eu/). I am also one of the Directors of Eurordis, the European Rare Diseases Oraganisation (http://www.eurordis.org).

Liarazole is a medical product originally prepared by Barrier Therpeutics NV (Belgium) and it was granted "Orphan Drug Status" by the EMEA, the European Union Agency equivalent to the FDA, in 2003.
http://www.emea.europa.eu/pdfs/human/comp/opinion/118503en.pdf

In 2004 FDA also granted Liarazole the same status.
http://www.pslgroup.com/dg/2447de.htm

During 2006 and 2007 Liarazole was tested in a phase II clinical trial involving patients from several european countries, including Italy. The outcomes were very encouraging, but from a statistical standpoint EMEA judged the trial not significant enough and asked Barrier to repeat it on a bigger cohort.

This effectively cancelled the project and contributed to the final dismission of the company which was acquired by another one (I don't recall the name) and this one by Stiefel.

ENI is currently trying to revive the initiative, but without much success. The experient demonstrates, in my opinion, how narrow-minded burocrats can be, even when they work in such instiutions as EMEA, explicitly creared to help people get the drugs they need.

The trial involved 64 Lamellar Ichthyosis patients, which for such a very rare disease, is not bad at all. Getting Stiefel to do it all over again and with much more people seems difficult, but perhaps a wordlwide appeal might help.

Here you have a full report: http://www.bio-medicine.org/medicine-tec...hthyosis-319-1/

The previous attempt was done in Europe only because of the more stringent rules on clinica trials in the US, I think. And of course coordination was much simpler limiting the field of operations to one side of the Atlantic...

We might try do push things a bit more, globally.
Ciao for now, Flavio

Hello Flavio
Welcome to our ichthyosis bulletin board and thank you for the followup information concerning Liazarole. I think it would take worldwide appeal for Stiefel Corp. and Barrier Therapuetics to continue research. The time, energy, and expenses of bringing a drug to the public is so high, many companies discontinue the trials before it has a chance to succeed.
Congragulations on your re-election to the board of directors of Eurordis. Your work with the European Ichthyosis Union and their conference in Copenhagen was wonderful to see.The ENI will bring more support and awareness to ichthyosis people in the member nations and those in surrounding countries. I hope to see more of your posts and keep us informed on the progress of the Italian Ichthyosis Union and the ENI. Thanks again Flavio.
Sincerely,
Les Avakian

Hi Flavio and thank you so much for the info! I'm sorry that it isn't going to be an easy drug to bring to market though I wonder what it would take really? If it really is a great drug then I'm up for doing everything I can

Hello Everyone
In doing research into liazarole, I found another drug in Barrier Therapuetics pipeline you might be interested in researching. It is called Rambazole and is a retinoic acid metabolic blocking agent working in the same pattern as Liazarole. As Glori stated in her previous post, it blocks the degradation of retinoic acid by blocking particle P450 hydroxylases, there by increasing intra cellular levels of retinoic acid. The Barrier Therapuetics site states its rambazole is being researched for psoriasis and acne. Their Trials 2B were completed in 2007,and now are looking for a partner. I hope this is a step forward in bringing this product to market.
Sincerely,
Les

Thanks Les, I will definitely read some more info into it. Am I correct to assume that these types of "retinoic acid metabolic blocking agents" do not have the same negative side effects as oral retinoids? I'm pretty sure I read that somewhere..would be wonderful wouldn't it

Hi
I see this was a conversation from 2009 - does anyone know of any progress or new info since then?
Also hello to Brandynsmum Morgan I was wondering where in Sydney you are and if you'd be interested in chatting with me? (new mum on the central coast)

courtney