norUrso - new medicine

A new interesting medicine is being tried out, read more here:

This sounds very exciting! Does anyone have any more information on this or know someone who has tried it? Thanks!!!

Sounds very promising.

Article is from last year. The phase 2 trial is about to be completed (it was supposed to end few months ago, but these often get delayed and final reviews take time). If it was successful they can start preparing phase 3 trial. This needs lots of discussions and evidence to government agency(and financing, as full phase 3 trial can cost $100 million and still fail to get approval).

I’m hoping that this was successful and they get government approval (and financing) soon so they could start phase 3 trial next year. If things go well also in phase 3 trial, they need lots of study analysis and paperwork to apply for final approval. This would probably take several years even in best case to have this available to general public.

Lets see in few months if we get announcement about this phase 2 trial. Maybe this treatment could be available in 2018 or so.

I’m cautiously positive about this.

Wonder if we can get help from a powerful foundation like Melinda Gates Foundation?

I was 3 weeks ago on a Falk Symposium in Freiburg. Prof. Trauner presented there NorUDCA (norurso) as a possible drug for cholestatic diseases. He said that they expect to publish the phase 2 PSC results begining of 2015. All over all, he was very positive about this drug so I hope their results are ok until now (more than 120 patients recruited in phase 2 trial).
By the way, Falk received in 07/2014 for norurso an orphean drug designation from european medicines agency . This will allow them to have a less constraining (and expensive) phase 3:

Thanks for the update. They got orphan designation in the US too. Hope phase 2 went well and they can start phase 3 trial already in 2015. 2018 looks possible target year for availability if things go well. If only in Europe we can try to smuggle it to US…

As a side note, FDA regulations are awful and need for update. For example, if life saving drug increases cholesterol that is something I can live with…now FDA would probably delay it years.

I am pretty confident that one of the drugs which are now in developpment phase will be available in the next 5 years. NorUDCA (2018?) is my favorite as its mecanism of bicarbonate umbrella for cholangiocytes seems to me rationally appropriate in the case of PSC. Beside that I see LUM001 (2018?) being also very interesting - it decreases the circulation of bilary acids and has already several positive opinions on internet. I am not very convinced of OCA in case of PSC, I think the next Intercept's compound INT-767 will be more interesting for us. A union of ATRA+UDCA could also work and be the first available option as both are already available in pharmacy (more infos in 2015). And there is of course vancomycin, which shows more and more its efficacity in trials. :-)

In the next 10 years, I expect the monoclonal antibodies like simtuzumab, vedolizumab or BTT1023 to work on PSC. They will be however very expensive :-)

My theory is that PSC is not a homogenous disease but much more a common bile ducts' syndrom/reaction to several undiscovered diseases with different causes, some of them immunological and some not. Drugs like UDCA, norUDCA, LUM001, OCA or INT-767 influencing the bilary acid mecanism will act on this syndrom, protecting the cholangiocytes and will extend the life expectance, but will not cure it.

This sounds positive... lets see what the outcome of the stage 2 trial is...