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